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Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A (pathfinder™2)

Primary Purpose

Congenital Bleeding Disorder, Haemophilia A

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

turoctocog alfa pegol

About this trial

This is an interventional treatment trial for Congenital Bleeding Disorder

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria: - Male patients with severe congenital haemophilia A (FVIII activity below 1%, according to medical records) - Documented history of at least 150 EDs (exposure days) to other FVIII products - At least 12 years and body weight at least 35 kg (except for Croatia, France, Russia, Israel and the Netherlands where the lower age limit will be 18 years) Exclusion Criteria: - Previous participation in this trial defined as withdrawal after administration N8-GP - Any history of FVIII inhibitors - FVIII inhibitors above or equal to 0.6 BU/mL at screening - HIV (human immunodeficiency virus) positive, defined by medical records with CD4+ (T-lymphocyte subtype) count below or equal to 200/mcL or a viral load of more than 400000 copies/mL. If the data is not available in medical records within last 6 months, CD4+ will be measured at the screening visit - Congenital or acquired coagulation disorders other than haemophilia A - Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) as defined by available medical records - Platelet count below 50,000 platelets/mcL (laboratory value at the screening visit) - ALAT (alanine aminotransferase) above 3 times the upper limit of normal reference ranges at central laboratory - Creatinine level equal to or greater than 1.5 times above upper normal limit (according to central laboratory reference ranges) - Ongoing immune modulating or chemotherapeutic medication

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Prophylaxis

On-demand

Arm Description

Outcomes

Primary Outcome Measures

The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 19 Months

All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors.

Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 19 Months

Annualised bleeding rate (ABR) is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP.

The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 25 Months

Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 25 Months

ABR is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP.

Incidence Rate of FVIII-inhibitors ≥0.6 BU: At Approximately 80 Months

Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 80 Months

Annualised bleeding rate (ABR) is the number of bleeding episodes per year reported during the prophylactic treatment with N8-GP.

Secondary Outcome Measures

Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 19 Months

Haemostatic effect of N8-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by participant and/or parent(s)/caregiver within approximately 8 hours after a single injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hrs after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hrs after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms.

Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 25 Months

Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 80 Months

Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 19 Months

The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported.

Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 25 Months

The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported.

Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 80 Months

The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported.

Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 19 Months

The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported.

Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 25 Months

The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported.

Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 80 Months

The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported.

Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 19 Months

Number of infusions are presented as average dose used during propphylaxis and on-demand treatment.

Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 25 Months

Number of infusions are presented as average dose used during prophylaxis and on-demand treatment.

Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 80 Months

Number of infusions are presented as average dose used during prophylaxis and on-demand treatment.

Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 19 Months

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported.

Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 25 Months

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported.

Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 80 Months

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported.

Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 19 Months

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported.

Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 25 Months

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported.

Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 80 Months

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported.

Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 19 Months

Recovery and trough levels of FVIII:C was reported for all participants at Visit 3 (Week 4) and end of main phase (approx. 19 months). The data was reported for all participants who received prophylaxis treatment. Chromogenic assay was performed with N8-GP product specific standard (PSS) as a calibrator.

Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 25 Months

Recovery and trough levels of FVIII:C was reported for all participants at the end of extension phase 1 study (approx. 25 months). The data was reported for all participants who received prophylaxis treatment. Chromogenic assay was performed with N8-GP product specific standard (PSS) as a calibrator.

Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 80 Months

Since patients were allowed to change prophylaxis regimen at any time during the extension phase part 2, and since the visit intervals were different for the 2 prophylaxis treatment regimens (Q4D and Q7D), FVIII activity data are reported only as incremental recovery at this timepoint.

Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Patients 13-16 Years Old) After Approx 19 and 25 Months

Reported results are change from baseline (Month 0) measured at end of main phase (approx Month 19) and change from Month 19 at end of Extension 1 (approx Month 25) of the study. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia.

Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Patients 13-16 Years Old): After Approx 80 Months

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point.

Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Parents of Patients 13-16 Years Old): After Approx 19 and 25 Months

Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The questionnaire was completed by parents of the patients in the 13-16 years old age bracket. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia.

Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Parents of Patients 13-16 Years Old): After Approx 80 Months

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. The questionnaire was completed by parents of the patients in the 13-16 years old age bracket. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point.

Patient Reported Outcomes - Change in HAEM-A-QOL (>=17 Years) Total Scores: After Approx 19 and 25 Months

Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The HAEM-A-QOL (for adults (>=17 years)) assessment included questions on questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia.

Patient Reported Outcomes - Change in HAEM-A-QOL (>=17 Years) Total Scores: After Approximately 80 Months

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The HAEM-A-QOL (for adults (>=17 years)) assessment included questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia.

Patient Reported Outcomes - Change in HEMO-SAT (Patients) Scores: After Approx 19 and 25 Months

Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The HEMO-SAT (Hematology-satisfaction) assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by patients). Adults completing the questionnaire could achieve a score from 0 to 100, with lower scores reflecting greater treatment satisfaction. The scale range for each of the 6 domains was 0-100 with lower scores reflecting greater treatment satisfaction. A decrease in the score would mean improvement.

Patient Reported Outcomes - Change in HEMO-SAT (Patients) Scores: After Approximately 80 Months

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by patients). Adults completing the questionnaire could achieve a score from 0 to 100, with lower scores reflecting greater treatment satisfaction. The scale range for each of the 6 domains was 0-100 with lower scores reflecting greater treatment satisfaction. A decrease in the score would mean improvement.

Patient Reported Outcomes - Change in HEMO-SAT Scores (Parents): After Approx 19 and 25 Months

The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by parents). Adults completing the questionnaire could achieve a score from 0 to 100, with lower scores reflecting greater treatment satisfaction. The scale range for each of the 6 domains was 0-100 with lower scores reflecting greater treatment satisfaction. A decrease in the score would mean improvement.

Patient Reported Outcomes - Change in HEMO-SAT (Parents) Scores: After Approximately 80 Months

Reported results are from Visit 1 (Month 0), and change from visit 1 upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Max. no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = max. number of participants contributed to the analysis for each time point. The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction. Adults completing the questionnaire could achieve a score from 0 to 100, with lower scores reflecting greater treatment satisfaction. The scale range for each of the 6 domains was 0-100 with lower scores reflecting greater treatment satisfaction. A decrease in the score would mean improvement.

Patient Reported Outcomes - Change in EQ-5D-VAS Scores: After Approx 19 and 25 Months

Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The European quality of life visual analogue scale (EQ5D-VAS) records the patient's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS can be used as a quantitative measure of health outcome that reflect the patient's own judgement. EQ-5D-VAS: range 0 to 100. A higher score indicates better self reported health status. A positive change indicates an improvement.

Patient Reported Outcomes - Change in EQ-5D-VAS Scores: After Approximately 80 Months

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The EQ5D-VAS records the patient's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS can be used as a quantitative measure of health outcome that reflect the patient's own judgement. EQ-5D-VAS: range 0 to 100. A higher score indicates better self reported health status. A positive change indicates an improvement.

Patient Reported Outcomes - Change in European Quality of Life Utility Index: After Approx 19 and 25 Months

Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The European quality of life utility index comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels where 1 indicates better health state (no problems) and 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; a positive change indicates an improvement.

Patient Reported Outcomes - Change in European Quality of Life Utility Index Scores: After Approximately 80 Months

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It is possible that a patient answers more than one questionnaire in a single time interval. Overall units analysed = Max no. of questionnaires answered by participants for this endpoint. Overall no. of participants analysed = max no. of participants analysed at each time point. This utility index has 5 dimensions: mobility, self-care, usual activities, pain/discomfort & anxiety/depression. Each dimension has 3 levels where 1 indicates better health state (no problems) and 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; a positive change indicates an improvement.

Number of Hospital Admissions During the Trial

The number of hospital admissions that took place in the study were reported.

Number of Days at the Hospital During the Trial

The mean number of days that participants spent at the hospital during the study were reported.

Number of Admissions to the Emergency Room (ER) During the Trial

The number of admissions to the ER that took place in the study were reported for each group.

Number of Days Missing School or Work

The mean number of days that participants missed to go to school or work were reported.

Number of Days Using Mobility Aid

The mean number of days that participants used any aids for mobility during the study were reported.

Number of Participants Using Pain Medication

The number of participants using pain medication during the main plus extension phase 1 of the study (approximately 25 months) and during extension phase 2(approximately 80 months) were reported.

Number of Bleeds Using Pain Medication

The mean number of bleeds using pain medication in the main phase of the study (approximately 19 months) were reported.

Number of Adverse Events Reported During the Trial Period: After Approximately 19 Months

All presented adverse events (AEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Number of Adverse Events Reported During the Trial Period: After Approximately 25 Months

All presented adverse events (AEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Number of Adverse Events Reported During the Trial Period: After Approximately 80 Months

The number of adverse events observed during the study after approximately 80 months was reported.

Number of Serious Adverse Events Reported During the Trial Period: After Approximately 19 Months

All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Number of Serious Adverse Events Reported During the Trial Period: After Approximately 25 Months

All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Number of Serious Adverse Events Reported During the Trial Period: After Approximately 80 Months

All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Change in Blood Pressure: After Approximately 19 Months

The mean change in the systolic and diastolic blood pressure values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0).

Change in Blood Pressure: After Approximately 25 Months

The mean change in the systolic and diastolic blood pressure values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19).

Change in Blood Pressure: After Approximately 80 Months

Change in the blood pressure was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa.

Change in Pulse: After Approximately 19 Months

The mean change in the pulse values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0).

Change in Pulse: After Approximately 25 Months

The mean change in the pulse values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19).

Change in Pulse: After Approximately 80 Months

Change in pulse was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa.

Change in Body Temperature: After Approximately 19 Months

The mean change in the body temperature values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0).

Change in Body Temperature: After Approximately 25 Months

The mean change in the body temperature (C) values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19).

Change in Body Temperature: After Approximately 80 Months

Change in body temperature was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa.

Change in Respiratory Rate: After Approximately 19 Months

The mean change in the respiratory rate values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0).

Change in Respiratory Rate: After Approximately 25 Months

The mean change in the respiratory rate (breaths/min) values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19).

Change in Respiratory Rate: After Approximately 80 Months

Change in respiratory rate was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa.

FVIII Activity 30 Min Post -Injection (C30min)

FVIII plasma activity was measured after 30 mins of injection. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator

Incremental Recovery (Single Dose and Steady State)

Incremental recovery was defined as the dose-normalised activity recorded 30 min after end of injection. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator.

Trough Level (Single Dose and Steady State)

Trough level was defined as the plasma FVIII activity recorded immediately before next dose is given. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator.

Area Under the Curve (AUC0-inf)

Area under the plasma activity versus time profile from time zero to infinity (AUC0-inf) was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. It is the measure of total plasma exposure. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator.

Area Under the Curve (AUC0-t)

Area under the plasma activity versus time profile from time zero to the last measurable activity (AUC0-t) was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator.

Terminal Half Life (t1/2)

t½ = ln(2) / λz, where λz is the terminal elimination rate constant. The terminal elimination rate constant was estimated using linear regression on the terminal part of the log(activity) versus time profile. This was measured at Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator.

Clearance (CL)

Total plasma clearance (CL) of drug after intravenous administration was reported. Clearance was calculated using the formula CL= Dose / AUC(0-inf). This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator.

Mean Residence Time (MRT)

MRT = AUMC/AUC(0-inf), where AUMC is the area under the first moment curve, i.e. the area under the curve t∙C(t), calculated with the same method as AUC(0-inf) (linear trapezoidal method + extrapolated area). This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator.

Volume of Distribution at Steady State (Vss)

Apparent volume of distribution at steady state is a product of the mean residence time and clearance and was calculated using the formula - Vss = CL x MRT. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator.

Full Information

NCT ID

NCT01480180

First Posted

November 23, 2011

Last Updated

November 3, 2020

Sponsor

Novo Nordisk A/S

1. Study Identification

Unique Protocol Identification Number

NCT01480180

Brief Title

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

Acronym

pathfinder™2

Official Title

A Multi-national Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Patients With Haemophilia A

Study Type

Interventional

2. Study Status

Record Verification Date

November 2020

Overall Recruitment Status

Completed

Study Start Date

January 30, 2012 (Actual)

Primary Completion Date

December 10, 2018 (Actual)

Study Completion Date

December 10, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novo Nordisk A/S

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

This trial is conducted globally. The aim of the trial is to evaluate the safety and efficacy, including pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in subjects with Haemophilia A.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Congenital Bleeding Disorder, Haemophilia A

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

186 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Prophylaxis

Arm Type

Experimental

Arm Title

On-demand

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

turoctocog alfa pegol

Other Intervention Name(s)

NNC 0129-0000-1003

Intervention Description

Administered i.v.

Primary Outcome Measure Information:

Title

The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 19 Months

Description

Time Frame

After approximately 19 months

Title

Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 19 Months

Description

Annualised bleeding rate (ABR) is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP.

Time Frame

After approximately 19 months

Title

The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 25 Months

Description

Time Frame

After approximately 25 months

Title

Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 25 Months

Description

ABR is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP.

Time Frame

After approximately 25 months

Title

Incidence Rate of FVIII-inhibitors ≥0.6 BU: At Approximately 80 Months

Description

Time Frame

At approximately 80 months

Title

Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 80 Months

Description

Annualised bleeding rate (ABR) is the number of bleeding episodes per year reported during the prophylactic treatment with N8-GP.

Time Frame

After approximately 80 months

Secondary Outcome Measure Information:

Title

Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 19 Months

Description

Time Frame

After approximately 19 months

Title

Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 25 Months

Description

Time Frame

After approximately 25 months

Title

Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 80 Months

Description

Time Frame

After approximately 80 months

Title

Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 19 Months

Description

The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported.

Time Frame

After approximately 19 months

Title

Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 25 Months

Description

The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported.

Time Frame

After approximately 25 months

Title

Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 80 Months

Description

The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported.

Time Frame

After approximately 80 months

Title

Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 19 Months

Description

The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported.

Time Frame

After approximately 19 months

Title

Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 25 Months

Description

The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported.

Time Frame

After approximately 25 months

Title

Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 80 Months

Description

The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported.

Time Frame

After approximately 80 months

Title

Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 19 Months

Description

Number of infusions are presented as average dose used during propphylaxis and on-demand treatment.

Time Frame

After approximately 19 months

Title

Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 25 Months

Description

Number of infusions are presented as average dose used during prophylaxis and on-demand treatment.

Time Frame

After approximately 25 months

Title

Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 80 Months

Description

Number of infusions are presented as average dose used during prophylaxis and on-demand treatment.

Time Frame

After approximately 80 months

Title

Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 19 Months

Description

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported.

Time Frame

After approximately 19 months

Title

Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 25 Months

Description

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported.

Time Frame

After approximately 25 months

Title

Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 80 Months

Description

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported.

Time Frame

After approximately 80 months

Title

Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 19 Months

Description

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported.

Time Frame

After approximately 19 months

Title

Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 25 Months

Description

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported.

Time Frame

After approximately 25 months

Title

Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 80 Months

Description

The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported.

Time Frame

After approximately 80 months

Title

Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 19 Months

Description

Time Frame

After approximately 19 months

Title

Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 25 Months

Description

Time Frame

After approximately 25 months

Title

Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 80 Months

Description

Time Frame

After approximately 80 months

Title

Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Patients 13-16 Years Old) After Approx 19 and 25 Months

Description

Time Frame

After approx 19 and 25 months

Title

Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Patients 13-16 Years Old): After Approx 80 Months

Description

Time Frame

2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs

Title

Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Parents of Patients 13-16 Years Old): After Approx 19 and 25 Months

Description

Time Frame

After approx 19 and 25 months

Title

Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Parents of Patients 13-16 Years Old): After Approx 80 Months

Description

Time Frame

2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs

Title

Patient Reported Outcomes - Change in HAEM-A-QOL (>=17 Years) Total Scores: After Approx 19 and 25 Months

Description

Time Frame

After approx 19 and 25 months

Title

Patient Reported Outcomes - Change in HAEM-A-QOL (>=17 Years) Total Scores: After Approximately 80 Months

Description

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The HAEM-A-QOL (for adults (>=17 years)) assessment included questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia.

Time Frame

2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs

Title

Patient Reported Outcomes - Change in HEMO-SAT (Patients) Scores: After Approx 19 and 25 Months

Description

Time Frame

After approx 19 and 25 months

Title

Patient Reported Outcomes - Change in HEMO-SAT (Patients) Scores: After Approximately 80 Months

Description

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by patients). Adults completing the questionnaire could achieve a score from 0 to 100, with lower scores reflecting greater treatment satisfaction. The scale range for each of the 6 domains was 0-100 with lower scores reflecting greater treatment satisfaction. A decrease in the score would mean improvement.

Time Frame

1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs

Title

Patient Reported Outcomes - Change in HEMO-SAT Scores (Parents): After Approx 19 and 25 Months

Description

Time Frame

After approx 19 and 25 months

Title

Patient Reported Outcomes - Change in HEMO-SAT (Parents) Scores: After Approximately 80 Months

Description

Time Frame

2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs

Title

Patient Reported Outcomes - Change in EQ-5D-VAS Scores: After Approx 19 and 25 Months

Description

Time Frame

After approx 19 and 25 months

Title

Patient Reported Outcomes - Change in EQ-5D-VAS Scores: After Approximately 80 Months

Description

Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The EQ5D-VAS records the patient's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS can be used as a quantitative measure of health outcome that reflect the patient's own judgement. EQ-5D-VAS: range 0 to 100. A higher score indicates better self reported health status. A positive change indicates an improvement.

Time Frame

1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs

Title

Patient Reported Outcomes - Change in European Quality of Life Utility Index: After Approx 19 and 25 Months

Description

Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The European quality of life utility index comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels where 1 indicates better health state (no problems) and 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; a positive change indicates an improvement.

Time Frame

After approx 19 and 25 months

Title

Patient Reported Outcomes - Change in European Quality of Life Utility Index Scores: After Approximately 80 Months

Description

Time Frame

1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs

Title

Number of Hospital Admissions During the Trial

Description

The number of hospital admissions that took place in the study were reported.

Time Frame

After approx 19, 25 and 80 months

Title

Number of Days at the Hospital During the Trial

Description

The mean number of days that participants spent at the hospital during the study were reported.

Time Frame

After approx 19, 25 and 80 months

Title

Number of Admissions to the Emergency Room (ER) During the Trial

Description

The number of admissions to the ER that took place in the study were reported for each group.

Time Frame

After approx 19, 25 and 80 months

Title

Number of Days Missing School or Work

Description

The mean number of days that participants missed to go to school or work were reported.

Time Frame

Approx 19, 25 and 80 months

Title

Number of Days Using Mobility Aid

Description

The mean number of days that participants used any aids for mobility during the study were reported.

Time Frame

Approx 19, 25 and 80 months

Title

Number of Participants Using Pain Medication

Description

The number of participants using pain medication during the main plus extension phase 1 of the study (approximately 25 months) and during extension phase 2(approximately 80 months) were reported.

Time Frame

After approx 25 and 80 months

Title

Number of Bleeds Using Pain Medication

Description

The mean number of bleeds using pain medication in the main phase of the study (approximately 19 months) were reported.

Time Frame

After approx 19 months

Title

Number of Adverse Events Reported During the Trial Period: After Approximately 19 Months

Description

All presented adverse events (AEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Time Frame

After approx 19 months

Title

Number of Adverse Events Reported During the Trial Period: After Approximately 25 Months

Description

All presented adverse events (AEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Time Frame

After approx. 25 months

Title

Number of Adverse Events Reported During the Trial Period: After Approximately 80 Months

Description

The number of adverse events observed during the study after approximately 80 months was reported.

Time Frame

After approximately 80 months

Title

Number of Serious Adverse Events Reported During the Trial Period: After Approximately 19 Months

Description

All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Time Frame

After approximately 19 months

Title

Number of Serious Adverse Events Reported During the Trial Period: After Approximately 25 Months

Description

All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Time Frame

After approximately 25 months

Title

Number of Serious Adverse Events Reported During the Trial Period: After Approximately 80 Months

Description

All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.

Time Frame

After approximately 80 months

Title

Change in Blood Pressure: After Approximately 19 Months

Description

Time Frame

After approximately 19 months

Title

Change in Blood Pressure: After Approximately 25 Months

Description

Time Frame

After approximately 19 and 25 months

Title

Change in Blood Pressure: After Approximately 80 Months

Description

Change in the blood pressure was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa.

Time Frame

After approximately 80 months

Title

Change in Pulse: After Approximately 19 Months

Description

Time Frame

After approximately 19 months

Title

Change in Pulse: After Approximately 25 Months

Description

Time Frame

After approximately 25 months

Title

Change in Pulse: After Approximately 80 Months

Description

Change in pulse was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa.

Time Frame

After approximately 80 months

Title

Change in Body Temperature: After Approximately 19 Months

Description

Time Frame

After approximately 19 months

Title

Change in Body Temperature: After Approximately 25 Months

Description

Time Frame

After approximately 25 months

Title

Change in Body Temperature: After Approximately 80 Months

Description

Change in body temperature was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa.

Time Frame

After approximately 80 months

Title

Change in Respiratory Rate: After Approximately 19 Months

Description

Time Frame

After approximately 19 months

Title

Change in Respiratory Rate: After Approximately 25 Months

Description

Time Frame

After approximately 25 months

Title

Change in Respiratory Rate: After Approximately 80 Months

Description

Change in respiratory rate was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa.

Time Frame

After approximately 80 months

Title

FVIII Activity 30 Min Post -Injection (C30min)

Description

Time Frame

Week 0, week 28

Title

Incremental Recovery (Single Dose and Steady State)

Description

Time Frame

Week 0, week 28

Title

Trough Level (Single Dose and Steady State)

Description

Time Frame

Week 0, week 28

Title

Area Under the Curve (AUC0-inf)

Description

Time Frame

Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28

Title

Area Under the Curve (AUC0-t)

Description

Time Frame

Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28

Title

Terminal Half Life (t1/2)

Description

Time Frame

Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28

Title

Clearance (CL)

Description

Time Frame

Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28

Title

Mean Residence Time (MRT)

Description

Time Frame

Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28

Title

Volume of Distribution at Steady State (Vss)

Description

Time Frame

Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Global Clinical Registry (GCR, 1452)

Organizational Affiliation

Novo Nordisk A/S

Official's Role

Study Director

Facility Information:

Facility Name

Novo Nordisk Investigational Site

City

Phoenix

State/Province

Arizona

ZIP/Postal Code

85016

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Long Beach

State/Province

California

ZIP/Postal Code

90806

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Sacramento

State/Province

California

ZIP/Postal Code

95817

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Torrance

State/Province

California

ZIP/Postal Code

90502-2004

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Washington

State/Province

District of Columbia

ZIP/Postal Code

20007

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Washington

State/Province

District of Columbia

ZIP/Postal Code

20010-2978

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Orlando

State/Province

Florida

ZIP/Postal Code

32827

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Tampa

State/Province

Florida

ZIP/Postal Code

33607

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Augusta

State/Province

Georgia

ZIP/Postal Code

30912

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Boise

State/Province

Idaho

ZIP/Postal Code

83712

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

New Orleans

State/Province

Louisiana

ZIP/Postal Code

70118-5720

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Detroit

State/Province

Michigan

ZIP/Postal Code

48201

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

East Lansing

State/Province

Michigan

ZIP/Postal Code

48823

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55404

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Omaha

State/Province

Nebraska

ZIP/Postal Code

68198-5456

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

New Brunswick

State/Province

New Jersey

ZIP/Postal Code

08901

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Newark

State/Province

New Jersey

ZIP/Postal Code

07102

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

452289

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Dayton

State/Province

Ohio

ZIP/Postal Code

45404

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19134

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Charleston

State/Province

South Carolina

ZIP/Postal Code

29425

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Nashville

State/Province

Tennessee

ZIP/Postal Code

37232-9830

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Charlottesville

State/Province

Virginia

ZIP/Postal Code

22908

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Norfolk

State/Province

Virginia

ZIP/Postal Code

23507

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Spokane

State/Province

Washington

ZIP/Postal Code

99204

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Camperdown

State/Province

New South Wales

ZIP/Postal Code

2050

Country

Australia

Facility Name

Novo Nordisk Investigational Site

City

South Brisbane

State/Province

Queensland

ZIP/Postal Code

4101

Country

Australia

Facility Name

Novo Nordisk Investigational Site

City

Parkville

State/Province

Victoria

ZIP/Postal Code

3052

Country

Australia

Facility Name

Novo Nordisk Investigational Site

City

Campinas

State/Province

Sao Paulo

ZIP/Postal Code

13081-970

Country

Brazil

Facility Name

Novo Nordisk Investigational Site

City

Sofia

ZIP/Postal Code

1756

Country

Bulgaria

Facility Name

Novo Nordisk Investigational Site

City

Split

ZIP/Postal Code

21 000

Country

Croatia

Facility Name

Novo Nordisk Investigational Site

City

Zagreb

ZIP/Postal Code

10 000

Country

Croatia

Facility Name

Novo Nordisk Investigational Site

City

København Ø

ZIP/Postal Code

2100

Country

Denmark

Facility Name

Novo Nordisk Investigational Site

City

Århus N

ZIP/Postal Code

8200

Country

Denmark

Facility Name

Novo Nordisk Investigational Site

City

Bron Cedex

ZIP/Postal Code

69677

Country

France

Facility Name

Novo Nordisk Investigational Site

City

Kremlin-Bicêtre

ZIP/Postal Code

94270

Country

France

Facility Name

Novo Nordisk Investigational Site

City

Nantes Cedex 1

ZIP/Postal Code

44093

Country

France

Facility Name

Novo Nordisk Investigational Site

City

Paris

ZIP/Postal Code

75015

Country

France

Facility Name

Novo Nordisk Investigational Site

City

Berlin

ZIP/Postal Code

10249

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Bonn

ZIP/Postal Code

53127

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Frankfurt/M.

ZIP/Postal Code

60590

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Hannover

ZIP/Postal Code

30625

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Homburg

ZIP/Postal Code

66421

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

München

ZIP/Postal Code

80336

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Budapest

ZIP/Postal Code

H-1134

Country

Hungary

Facility Name

Novo Nordisk Investigational Site

City

Debrecen

ZIP/Postal Code

4012

Country

Hungary

Facility Name

Novo Nordisk Investigational Site

City

Tel-Hashomer

ZIP/Postal Code

52621

Country

Israel

Facility Name

Novo Nordisk Investigational Site

City

Firenze

ZIP/Postal Code

50134

Country

Italy

Facility Name

Novo Nordisk Investigational Site

City

Milano

ZIP/Postal Code

20124

Country

Italy

Facility Name

Novo Nordisk Investigational Site

City

Udine

ZIP/Postal Code

33100

Country

Italy

Facility Name

Novo Nordisk Investigational Site

City

Vicenza

ZIP/Postal Code

36100

Country

Italy

Facility Name

Novo Nordisk Investigational Site

City

Aichi

ZIP/Postal Code

466-8560

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Hiroshima

ZIP/Postal Code

734-8551

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Kitakyusyu, Fukuoka

ZIP/Postal Code

807 8555

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Nara

ZIP/Postal Code

634-8522

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Saitama

ZIP/Postal Code

350-0225

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Shimotsuke-shi, Tochigi

ZIP/Postal Code

329 0498

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Shizuoka

ZIP/Postal Code

420-8660

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Tokyo

ZIP/Postal Code

108-8639

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Tokyo

ZIP/Postal Code

160-0023

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Tokyo

ZIP/Postal Code

167-0035

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Yokohama-shi, Kanagawa

ZIP/Postal Code

241-0811

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Daejeon

ZIP/Postal Code

302-799

Country

Korea, Republic of

Facility Name

Novo Nordisk Investigational Site

City

Kuala Lumpur

ZIP/Postal Code

50400

Country

Malaysia

Facility Name

Novo Nordisk Investigational Site

City

Selangor Darul Ehsan

ZIP/Postal Code

68000

Country

Malaysia

Facility Name

Novo Nordisk Investigational Site

City

Groningen

ZIP/Postal Code

9713 GZ

Country

Netherlands

Facility Name

Novo Nordisk Investigational Site

City

Rotterdam

ZIP/Postal Code

3015 CE

Country

Netherlands

Facility Name

Novo Nordisk Investigational Site

City

Oslo

ZIP/Postal Code

0027

Country

Norway

Facility Name

Novo Nordisk Investigational Site

City

San Juan

ZIP/Postal Code

00935

Country

Puerto Rico

Facility Name

Novo Nordisk Investigational Site

City

Saint-Petersburg

ZIP/Postal Code

191065

Country

Russian Federation

Facility Name

Novo Nordisk Investigational Site

City

Madrid

ZIP/Postal Code

28046

Country

Spain

Facility Name

Novo Nordisk Investigational Site

City

Málaga

ZIP/Postal Code

29010

Country

Spain

Facility Name

Novo Nordisk Investigational Site

City

Malmö

ZIP/Postal Code

205 02

Country

Sweden

Facility Name

Novo Nordisk Investigational Site

City

Genève 14

ZIP/Postal Code

1211

Country

Switzerland

Facility Name

Novo Nordisk Investigational Site

City

Lausanne

ZIP/Postal Code

1011

Country

Switzerland

Facility Name

Novo Nordisk Investigational Site

City

Zürich

ZIP/Postal Code

8091

Country

Switzerland

Facility Name

Novo Nordisk Investigational Site

City

Changhua

ZIP/Postal Code

500

Country

Taiwan

Facility Name

Novo Nordisk Investigational Site

City

Taipei

ZIP/Postal Code

100

Country

Taiwan

Facility Name

Novo Nordisk Investigational Site

City

Adana

ZIP/Postal Code

01130

Country

Turkey

Facility Name

Novo Nordisk Investigational Site

City

Bornova-IZMIR

ZIP/Postal Code

35100

Country

Turkey

Facility Name

Novo Nordisk Investigational Site

City

Samsun

ZIP/Postal Code

55319

Country

Turkey

Facility Name

Novo Nordisk Investigational Site

City

Basingstoke

ZIP/Postal Code

RG24 9NA

Country

United Kingdom

Facility Name

Novo Nordisk Investigational Site

City

Cardiff

ZIP/Postal Code

CF14 4XW

Country

United Kingdom

Facility Name

Novo Nordisk Investigational Site

City

London

ZIP/Postal Code

NW3 2QG

Country

United Kingdom

Facility Name

Novo Nordisk Investigational Site

City

London

ZIP/Postal Code

SE1 7EH

Country

United Kingdom

Facility Name

Novo Nordisk Investigational Site

City

Oxford

ZIP/Postal Code

OX3 7LJ

Country

United Kingdom

Facility Name

Novo Nordisk Investigational Site

City

Sheffield

ZIP/Postal Code

S10 2JF

Country

United Kingdom

12. IPD Sharing Statement

Citations:

Citation

Giangrande P, Chowdary P, Enhrenforth S, Hanabusa H, Leebeek FW, Lentz SR, Nemes L, Poulsen LH, Santagostino E, You CW, Clausen WHO, Oldenburg J and on behalf of for the pathfinder™2 Investigators. Clinical evaluation of novel recombinant glycopegylated FVIII (turoctocog alfa pegol, N8-GP): efficacy and safety in previously treated patients with severe hemophilia A - results of pathfinder™2 international trial. Journal of Thrombosis and Haemostasis (Abstracts) 2015; 13 (Supplement S2): 1-997 [OR212]

Results Reference

result

PubMed Identifier

27904904

Citation

Giangrande P, Andreeva T, Chowdary P, Ehrenforth S, Hanabusa H, Leebeek FW, Lentz SR, Nemes L, Poulsen LH, Santagostino E, You CW, Clausen WH, Jonsson PG, Oldenburg J; Pathfinder2 Investigators. Clinical evaluation of glycoPEGylated recombinant FVIII: Efficacy and safety in severe haemophilia A. Thromb Haemost. 2017 Jan 26;117(2):252-261. doi: 10.1160/TH16-06-0444. Epub 2016 Dec 1. Erratum In: Thromb Haemost. 2017 Jun;117(6):1163.

Results Reference

result

PubMed Identifier

32544297

Citation

Giangrande P, Abdul Karim F, Nemes L, You CW, Landorph A, Geybels MS, Curry N. Long-term safety and efficacy of N8-GP in previously treated adults and adolescents with hemophilia A: Final results from pathfinder2. J Thromb Haemost. 2020 Sep;18 Suppl 1(Suppl 1):5-14. doi: 10.1111/jth.14959.

Results Reference

result

Links:

URL

http://novonordisk-trials.com

Description

Clinical Trials at Novo Nordisk

Learn more about this trial

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

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Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A (pathfinder™2)

Congenital Bleeding Disorder, Haemophilia A

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial