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Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B

Primary Purpose

Hemophilia B

Status

Completed

Phase

Phase 4

Locations

India

Study Type

Interventional

Intervention

Nonacog alfa

About this trial

This is an interventional treatment trial for Hemophilia B

Eligibility Criteria

12 Years - 65 Years (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Male subjects ≥12 years to ≤65 years with a diagnosis of congenital moderately-severe to severe hemophilia B (FIX activity ≤2%).
Documented history of at least 50 exposure days (EDs) to FIX-containing products.
Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study. For minors under the age of legal consent in India, assent of the participating child needs to be documented for the age range 12 to 18 in addition to the parental informed consent.

Exclusion Criteria:

Prior history of inhibitor to FIX or positive inhibitor testing (≥0.6 BU/mL) during Screening. Clinical signs or symptoms of decreased response to FIX.
Known hypersensitivity to the active substance or any of the excipients.
Known allergic reaction to hamster proteins.
Presence of any bleeding disorder in addition to hemophilia B.
Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation.
Planned surgery within 6 months from the start of the study.
Unsuitable to participate in study for any other reason as assessed by the investigator; including any disorder, except for conditions associated with hemophilia B, which in the investigator's opinion might jeopardize subject's safety or compliance with the protocol.
Subjects (or a legally acceptable representative) is not able to understand study documents and study procedure.
Immunocompromised subjects due to human immunodeficiency virus (HIV) infection (defined as viral load above or equal to 100,000 copies/mL; and for HIV+ subjects: cluster of differentiation 4 positive (CD4+) lymphocyte count below or equal to 200/μL). HIV status and CD4+ lymphocyte count results may be obtained at screening or from available medical records; results must be not older than 6 months prior to screening.
Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, subjects who have been previously enrolled into the study, or subjects who are Pfizer employees directly involved in the conduct of the study.
Planned use of any non-study medication for treatment of hemophilia (eg, other factor replacement agents, bypassing agents, or non-factor treatments [such as anti-tissue factor pathway inhibitors]).

Sites / Locations

Nirmal Hospital Pvt Ltd.
K.J. Somaiya Hospital and Research Centre
Sahyadri Clinical Research & Development Centre
Christian Medical College and Hospital
Sahyadri Super Specialty Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment Arm

Arm Description

At least twenty five eligible male subjects will be enrolled in the treatment arm to receive Nonacog alfa until 16 exposure days (EDs) or a period of up to 8 weeks on treatment had occurred (whichever occurs first).

Outcomes

Primary Outcome Measures

Percentage of Participants Who Developed Factor IX (FIX) Inhibitors

FIX inhibitor development was defined as an inhibitor titer >= 0.6 Bethesda units per milliliter (BU/mL) confirmed by central laboratory testing during the course of the study.

Secondary Outcome Measures

Number of Participants With Serious Adverse Events (SAEs) and Medically Important Events (MIEs)

An adverse events (AE) is any untoward medical occurrence in clinical investigation participant administered a product or medical device; event need not necessarily to have a causal relationship with treatment or usage. SAEs: an AE resulting in any of following outcomes/deemed significant for any other reason: death; initial/prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. MIEs: any confirmed FIX inhibitor development (titer greater than or equal to 0.6 BU/mL confirmed by central laboratory testing), thrombotic events (any event associated with the formation of a blood clot including catheter-associated thrombi and thrombotic complications) or hypersensitivity reactions (hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgment).

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs are events between first dose of study drug and up to 116 days that were absent before treatment or that worsened relative to pretreatment state.

Mean Annualized Bleeding Rate (ABR)

ABR: number of bleeding episodes per year. ABR for each participant = number of bleeds during treatment interval duration (TID)/(TID/365.25). Number of bleeds for each participant for ABR included all new bleeds (with unique start date and time) requiring treatment with nonacog alfa during TID. TID=date of Visit 4 (3 to 10 days after last dose) - date of Visit 2 (Day 1) +1. For participants who had Visit 4 beyond 3 (+7) days after final dose due to COVID-19 pandemic, date of final dose was used in place of date of Visit 4. For discontinued participants, date of final dose/last study visit date (whichever occurred later) was used to replace Visit 4.

Mean Annualized Total Factor Consumption (TFC) Per Participant

The TFC per participant was the sum of the total amount (in international units [IU]) infused for each infusion for each participant. Annualized TFC of nonacog alfa was derived for each participant by using the following formula; Annualized TFC = (TFC / treatment interval duration)*365.25. Treatment interval duration was calculated as the number of days beginning on Visit 2 ("Day 1", provided an infusion was given) up to Visit 4 (any 1 day between Day 52 to Day 60).

Mean Annualized Total Factor Consumption (TFC) by Weight Per Participant

The total amount in international units (IU) per kilogram infused for each infusion recorded were summed to calculate the total factor consumption for each participant. Annualized TFC = (TFC / treatment interval duration)*365.25. Treatment interval duration was calculated as Date of Visit 4 - Date of Visit 2 +1. If Date of Visit 4 - Last dosing date was > 10 days then Date of Visit 4 was replaced with Last dosing date. If Date of Visit 4 was missing (due to discontinuation or any other reason), then Date of Visit 4 was replaced with last dosing date or last subject visit date, whichever was greater. International unit per kilogram= IU/kg.

Mean Number of Nonacog Alfa Infusions Used to Treat Each Bleed

Number of nonacog alfa infusions used to treat each bleed in participants was calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). On demand treatment was defined as treatment used to treat a bleeding episode.

Full Information

NCT ID

NCT04286412

First Posted

February 12, 2020

Last Updated

June 29, 2022

Sponsor

Pfizer

1. Study Identification

Unique Protocol Identification Number

NCT04286412

Brief Title

Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B

Official Title

A SINGLE COUNTRY, MULTICENTER, OPEN-LABEL AND NON-RANDOMIZED CLINICAL TRIAL WITH NONACOG ALFA PROPHYLAXIS AND TREATMENT OF BLEEDING EPISODES IN PREVIOUSLY TREATED PATIENTS WITH MODERATELY-SEVERE TO SEVERE HEMOPHILIA B FOR A DURATION OF 8 WEEKS.

Study Type

Interventional

2. Study Status

Record Verification Date

June 2022

Overall Recruitment Status

Completed

Study Start Date

February 10, 2020 (Actual)

Primary Completion Date

September 24, 2020 (Actual)

Study Completion Date

September 24, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.

6. Conditions and Keywords

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

Hemophilia B

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

25 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Treatment Arm

Arm Type

Experimental

Arm Description

Intervention Type

Biological

Intervention Name(s)

Nonacog alfa

Other Intervention Name(s)

BeneFIX

Intervention Description

Nonacog alfa is indicated in India for the treatment and prophylaxis of bleeding in patients with hemophilia B (congenital factor IX deficiency).

Primary Outcome Measure Information:

Title

Percentage of Participants Who Developed Factor IX (FIX) Inhibitors

Description

FIX inhibitor development was defined as an inhibitor titer >= 0.6 Bethesda units per milliliter (BU/mL) confirmed by central laboratory testing during the course of the study.

Time Frame

At Visit 4 (any 1 day between Day 52 to Day 60)

Secondary Outcome Measure Information:

Title

Number of Participants With Serious Adverse Events (SAEs) and Medically Important Events (MIEs)

Description

Time Frame

Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)

Title

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Description

Time Frame

Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)

Title

Mean Annualized Bleeding Rate (ABR)

Description

Time Frame

Up to 88 Days

Title

Mean Annualized Total Factor Consumption (TFC) Per Participant

Description

Time Frame

Up to 88 Days

Title

Mean Annualized Total Factor Consumption (TFC) by Weight Per Participant

Description

Time Frame

Up to 88 days

Title

Mean Number of Nonacog Alfa Infusions Used to Treat Each Bleed

Description

Time Frame

Up to 88 days

10. Eligibility

Sex

Male

Gender Based

Yes

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male subjects ≥12 years to ≤65 years with a diagnosis of congenital moderately-severe to severe hemophilia B (FIX activity ≤2%). Documented history of at least 50 exposure days (EDs) to FIX-containing products. Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study. For minors under the age of legal consent in India, assent of the participating child needs to be documented for the age range 12 to 18 in addition to the parental informed consent. Exclusion Criteria: Prior history of inhibitor to FIX or positive inhibitor testing (≥0.6 BU/mL) during Screening. Clinical signs or symptoms of decreased response to FIX. Known hypersensitivity to the active substance or any of the excipients. Known allergic reaction to hamster proteins. Presence of any bleeding disorder in addition to hemophilia B. Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation. Planned surgery within 6 months from the start of the study. Unsuitable to participate in study for any other reason as assessed by the investigator; including any disorder, except for conditions associated with hemophilia B, which in the investigator's opinion might jeopardize subject's safety or compliance with the protocol. Subjects (or a legally acceptable representative) is not able to understand study documents and study procedure. Immunocompromised subjects due to human immunodeficiency virus (HIV) infection (defined as viral load above or equal to 100,000 copies/mL; and for HIV+ subjects: cluster of differentiation 4 positive (CD4+) lymphocyte count below or equal to 200/μL). HIV status and CD4+ lymphocyte count results may be obtained at screening or from available medical records; results must be not older than 6 months prior to screening. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, subjects who have been previously enrolled into the study, or subjects who are Pfizer employees directly involved in the conduct of the study. Planned use of any non-study medication for treatment of hemophilia (eg, other factor replacement agents, bypassing agents, or non-factor treatments [such as anti-tissue factor pathway inhibitors]).

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Pfizer CT.gov Call Center

Organizational Affiliation

Pfizer

Official's Role

Study Director

Facility Information:

Facility Name

Nirmal Hospital Pvt Ltd.

City

Surat

State/Province

Gujarat

ZIP/Postal Code

395002

Country

India

Facility Name

K.J. Somaiya Hospital and Research Centre

City

Mumbai

State/Province

Maharashtra

ZIP/Postal Code

400022

Country

India

Facility Name

Sahyadri Clinical Research & Development Centre

City

Pune

State/Province

Maharashtra

ZIP/Postal Code

411004

Country

India

Facility Name

Christian Medical College and Hospital

City

Ludhiana

State/Province

Punjab

ZIP/Postal Code

141008

Country

India

Facility Name

Sahyadri Super Specialty Hospital

City

Pune

ZIP/Postal Code

411004

Country

India

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

IPD Sharing URL

https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

Links:

URL

https://pmiform.com/clinical-trial-info-request?StudyID=B1821059

Description

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Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B

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