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Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)

Primary Purpose

Hereditary Angioedema (HAE)

Status
Completed
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
CINRYZE 500 U
CINRYZE 1000 U
Sponsored by
Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Angioedema (HAE)

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents.
  2. Be ≥2 years of age.
  3. Meet the following minimum body weight criteria:

    • Subjects 2 to 5 years of age must weigh at least 12.5 kg; and
    • Subjects 6 years of age and above must weigh at least 25 kg.
  4. Have a confirmed diagnosis of Type I or Type II HAE. NOTE: Diagnosis may be based on historical data including family history, clinical symptoms (characteristic attacks), or documentation of low level of C1 INH protein and/or C1 INH activity.
  5. Have a history of at least one angioedema attack per month (on average) during the 3 consecutive months immediately before enrollment.
  6. Agree to adhere to the protocol-defined schedule of assessments and procedures.
  7. Agree to avoid his/her known angioedema attack triggers during the study to the best of his/her ability.
  8. If a female of reproductive age, be postmenopausal (≥12 months following cessation of menstruation), surgically sterile, or following an acceptable method of birth control (and agree to continue its use through 1 month after the last dose of study drug):

    • Non-hormonal methods (eg, abstinence, barrier control) for at least 1 complete menstrual cycle before the Screening Visit.
    • Stable doses of estrogen and/or progestin containing products for at least 2 months before the Screening Visit.
  9. If a male of reproductive age, be surgically sterile or agree to follow an acceptable method of birth control (eg, abstinence, barrier control) from the Screening Visit through 2 months after the last dose of study drug.
  10. If an adult, be informed of the nature of the study and provide written informed consent before any study-specific procedures are performed.

OR If a child or minor (<20 years of age), have a parent/legal guardian who is informed of the nature of the study provide written informed consent (ie, permission) for the child to participate in the study before any study-specific procedures are performed. Assent will be obtained from children ≥14 years of age.

Exclusion Criteria:

  1. Have a history of hypercoagulability (abnormal blood clotting).
  2. Have a diagnosis of acquired angioedema or be known to have C1 INH antibodies.
  3. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE) or other blood products.
  4. Have received C1 INH therapy or any blood products within 3 days before the first dose of study drug.
  5. Have had signs or symptoms of an angioedema attack within 2 days before the first dose of study drug.
  6. Have any change (start, stop, or change in dose) in androgen therapy (eg, danazol, oxandrolone, stanozolol, testosterone), tranexamic acid, epsilon-aminocaproic acid (EACA), or other antifibrinolytics within 14 days before the first dose of study drug.
  7. If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (eg, estrogen/progestin containing products) within 2 months before the first dose of study drug.
  8. Be pregnant or breastfeeding.
  9. Have received an investigational drug other than those required for prevention or treatment of angioedema attacks within 30 days before the first dose of study drug.
  10. Have, as determined by the Investigator and/or the Sponsor's Medical Monitor, any surgical or medical condition that could interfere with the administration of study drug or interpretation of study results.

Sites / Locations

  • Toyohashi Municipal Hospital
  • Gunma University Hospital
  • Kobe University Hospital
  • Heart Life Hospital
  • Naha City Hospital
  • Shiman University Hospital
  • Asahi General Hospital
  • Adachi kyosai Hospital
  • Hiroshima University Hospital
  • Tomakomai City Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Subjects 2 to 5 years of age

Subjects 6 years of age and older

Arm Description

500 U of CINRYZE will be administered by IV infusion twice weekly for 12 weeks

1000 U of CINRYZE will be administered by IV infusion twice weekly for 12 weeks.

Outcomes

Primary Outcome Measures

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered an investigational product and that did not necessarily have a causal relationship with the treatment. TEAEs were defined as all AEs that started during the treatment period and up to 7 days after the last dose of investigational product, or AEs that were seen at baseline but worsened in frequency and/or severity during the treatment period and up to 7 days after the last dose of investigational product.
Number of Participants With Clinically Significant Abnormalities in Physical Examination Reported as Adverse Events (AEs)
Physical examinations included measurement of body weight and height. Clinically significant abnormalities related to physical examination as determined by investigator were recorded and reported as AE.
Number of Participants With Potentially Clinically Important (PCI) Vital Signs Reported as Adverse Events (AEs)
Vital sign assessments included systolic blood pressure (SBP), diastolic blood pressure (DBP) and pulse rate. Investigator used both absolute values and change from baseline values to determine if the vital sign was potentially clinically important. Criteria for the potential clinical importance of both absolute and change from baseline values were pre-specified as: SBP (less than [<] 90 millimeter of mercury [mmHg]; greater than or equal to [>=] 140 mmHg), DBP (< 60 mmHg; >=90 mmHg) and pulse (less than or equal to [<=] 50 beats per minute [bpm]; >= 100 bpm. A participant's vital sign had to meet both the absolute and change from baseline criteria to be considered as potentially clinically important.
Number of Participants With Potentially Clinically Important (PCI) Clinical Laboratory Assessments Reported as Adverse Events (AEs)
Number of participants with potentially clinically important (PCI) clinical laboratory assessments reported as adverse events were reported.
Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1
C1 INH antigen concentration in plasma was determined using an automated nephelometric assay.
Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 12
C1 INH antigen concentration in plasma was determined using an automated nephelometric assay.
Concentration of Plasma Complement C4 at Week 1
Concentration of plasma complement C4 was reported.
Concentration of Plasma Complement C4 at Week 12
Concentration of plasma complement C4 was reported.
Concentration of Plasma Complement C1q at Week 1
Concentration of plasma complement C1q was reported.
Normalized Number of Angioedema Attacks (NNA) Per Month
Angioedema attack was defined as any participant-reported (or caregiver-reported) indication of swelling or pain at any location following a report of no swelling or pain on the previous day (that is, there must have been a full symptom-free calendar day preceding the onset of symptoms for an attack to be considered a new attack). NNA was calculated as the overall number of angioedema attacks recorded during the period divided by the number of days in the period and multiplied by 30.4.Number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency as compared to the historical data where, NNA was the number of angioedema attacks during 3 months prior to study drug administration. Historical data was obtained from medical or angioedema history electronic case report forms (eCRF).
Number of Participants With Angioedema Attacks in Different Anatomic Locations
Anatomic locations where there was a presence of pain or swelling of any level of severity; mild, moderate or severe at any day during the attack were reported. Mild: the attack symptoms were noticeable but were easily tolerated by the participant and did not interfere with the participant's daily activities. Moderate: the attack symptoms interfered with the participant's ability to attend work/school or participate in family life and social/recreational activities and severe: the attack symptoms significantly limited the participant's ability to attend work/school or participate in family life and social/recreational activities. Number of participants with angioedema attacks in different anatomic locations in treatment period was compared to NNA for historical data. Historical data was based on the typical location of angioedema attacks in the 3 months prior to study drug administration. Here, H refers to historical and T refers to treatment.
Average Severity (Intensity) of Angioedema Attacks
All attacks in each therapy period were assigned a value of 1 (mild), 2 (moderate), or 3 (severe). Attack severity was considered the highest value assigned by the participant to any swelling location on any day during the attack. The average severity was derived by dividing the cumulative severity score by the total number of attacks. Average severity was set to 0 if there was no attack in a period. Average severity of angioedema attacks in treatment period compared to the NNA of angioedema attacks for historical data was reported. Historical data was based on the typical severity of angioedema attacks in the 3 months prior to study drug administration. Historical data was obtained from medical or angioedema history electronic case report forms (eCRF).
Average Duration of Angioedema Attacks
Average duration of attacks was calculated by dividing the cumulative duration of attacks by the total number of attacks during the treatment period. Historical data was based on the typical severity of angioedema attacks in the 3 months prior to study drug administration. Average duration of angioedema attacks in treatment period was compared to the NNA for historical data. Historical data was obtained from medical or angioedema history eCRF.
Normalized Number of Angioedema Attacks (NNA) Per Month Treated With Rescue Medication
The normalized number of angioedema attacks was calculated as the overall number of angioedema attacks recorded during the period divided by the number of days in the period and multiplied by 30.4. NNA treated with rescue medications were reported for CINRYZE, non-CINRYZE C1-INH or not treated with C1-INH (including attacks treated with any medications other than C1-INH or untreated attacks). CINRYZE was only considered as a rescue medication when treated for breakthrough attack treatment. For historical data, only medications taken prior to the start of drug study drug administration and had an indication of "hereditary angioedema (HAE) management - acute treatment" selected on the prior and concomitant medications and therapy were considered as rescue medications. Historical data was obtained from medical or angioedema history eCRF.
Number of Participants Achieving Clinical Responder Rate Relative to Historical Data
Number of participants achieving at least 50 percent (%), 70% or 90% reduction in NNA relative to NNA for historical data was reported.
Change From Baseline in Angioedema Quality of Life (AE-QoL) in Treatment Period
Angioedema quality of life (AE-QoL) questionnaire was a self-administered validated angioedema disease-specific quality of life instrument. It consisted of 17 specific questions that were associated with work, physical activity, free time, social relations, and diet. Each of the 17 items had a 5-point response scale ranging from 1 (Never) to 5 (Very Often). The questionnaire was scored according to the developers' guidelines to produce a total score and 4 domain scores (functioning, fatigue/mood, fear/shame, nutrition). Raw domain scores (mean of the item scores within each scale) and the raw total score (mean of all item scores) were rescaled using linear transformations into final percentage scores ranging 0 to 100, based on the maximum possible score, where the higher the score the greater the QoL impairment.
Number of Participants With Breakthrough Angioedema Attacks
A breakthrough attack was defined as an angioedema attack that occurs during long-term prevention therapy with CINRYZE (that is, between first study drug and last study drug dose). Number of participants with 1, 2, 3 or more angioedema attacks and who achieved initial improvement and complete resolution were also reported. Breakthrough angioedema attacks assessed by CINRYZE treatment, non-CINRYZE C1 INH treatment and untreated with C1-INH were reported. Here BAA refers to breakthrough angioedema attacks.
Time From Attack Onset to Initial Improvement and Complete Resolution
Time to initial improvement (TII) was calculated from the time of study drug administration to initial symptom improvement. Time to complete resolution was defined as the time from the onset of attack to complete resolution of all symptoms. Time to initial improvement and time to complete resolution as assessed by CINRYZE, non-CINRYZE and untreated were reported.
Time From Onset of Attack to Time Treated by CINRYZE
The median time from onset of attack to time treated with CINRYZE was reported.
Time From Treatment With CINRYZE to Initial Improvement
Time to initial improvement was calculated from the time of study drug administration to initial symptom improvement. Median time from treatment with CINRYZE to initial improvement was reported.

Secondary Outcome Measures

Full Information

First Posted
August 9, 2016
Last Updated
May 13, 2021
Sponsor
Shire
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1. Study Identification

Unique Protocol Identification Number
NCT02865720
Brief Title
Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
Official Title
A Phase 3, Open-label, Single-period Study to Evaluate the Safety and Treatment Effect of Intravenous Administration of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
September 8, 2016 (Actual)
Primary Completion Date
June 23, 2017 (Actual)
Study Completion Date
June 23, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire

4. Oversight

5. Study Description

Brief Summary
The purpose of this study is to determine if an investigational treatment is safe and well tolerated when administered by intravenous (IV) infusion in Japanese subjects with HAE.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema (HAE)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
8 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Subjects 2 to 5 years of age
Arm Type
Experimental
Arm Description
500 U of CINRYZE will be administered by IV infusion twice weekly for 12 weeks
Arm Title
Subjects 6 years of age and older
Arm Type
Experimental
Arm Description
1000 U of CINRYZE will be administered by IV infusion twice weekly for 12 weeks.
Intervention Type
Drug
Intervention Name(s)
CINRYZE 500 U
Intervention Description
IV infusion administered twice weekly
Intervention Type
Drug
Intervention Name(s)
CINRYZE 1000 U
Intervention Description
IV infusion administered twice weekly
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Description
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered an investigational product and that did not necessarily have a causal relationship with the treatment. TEAEs were defined as all AEs that started during the treatment period and up to 7 days after the last dose of investigational product, or AEs that were seen at baseline but worsened in frequency and/or severity during the treatment period and up to 7 days after the last dose of investigational product.
Time Frame
From start of study drug administration up to Week 12
Title
Number of Participants With Clinically Significant Abnormalities in Physical Examination Reported as Adverse Events (AEs)
Description
Physical examinations included measurement of body weight and height. Clinically significant abnormalities related to physical examination as determined by investigator were recorded and reported as AE.
Time Frame
From start of study drug administration up to Week 12
Title
Number of Participants With Potentially Clinically Important (PCI) Vital Signs Reported as Adverse Events (AEs)
Description
Vital sign assessments included systolic blood pressure (SBP), diastolic blood pressure (DBP) and pulse rate. Investigator used both absolute values and change from baseline values to determine if the vital sign was potentially clinically important. Criteria for the potential clinical importance of both absolute and change from baseline values were pre-specified as: SBP (less than [<] 90 millimeter of mercury [mmHg]; greater than or equal to [>=] 140 mmHg), DBP (< 60 mmHg; >=90 mmHg) and pulse (less than or equal to [<=] 50 beats per minute [bpm]; >= 100 bpm. A participant's vital sign had to meet both the absolute and change from baseline criteria to be considered as potentially clinically important.
Time Frame
Baseline up to Week 12
Title
Number of Participants With Potentially Clinically Important (PCI) Clinical Laboratory Assessments Reported as Adverse Events (AEs)
Description
Number of participants with potentially clinically important (PCI) clinical laboratory assessments reported as adverse events were reported.
Time Frame
Baseline up to Week 12
Title
Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1
Description
C1 INH antigen concentration in plasma was determined using an automated nephelometric assay.
Time Frame
Week 1: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 hours (h) post-dose
Title
Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 12
Description
C1 INH antigen concentration in plasma was determined using an automated nephelometric assay.
Time Frame
Week 12: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose
Title
Concentration of Plasma Complement C4 at Week 1
Description
Concentration of plasma complement C4 was reported.
Time Frame
Week 1: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose
Title
Concentration of Plasma Complement C4 at Week 12
Description
Concentration of plasma complement C4 was reported.
Time Frame
Week 12: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose
Title
Concentration of Plasma Complement C1q at Week 1
Description
Concentration of plasma complement C1q was reported.
Time Frame
Baseline (Week 1)
Title
Normalized Number of Angioedema Attacks (NNA) Per Month
Description
Angioedema attack was defined as any participant-reported (or caregiver-reported) indication of swelling or pain at any location following a report of no swelling or pain on the previous day (that is, there must have been a full symptom-free calendar day preceding the onset of symptoms for an attack to be considered a new attack). NNA was calculated as the overall number of angioedema attacks recorded during the period divided by the number of days in the period and multiplied by 30.4.Number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency as compared to the historical data where, NNA was the number of angioedema attacks during 3 months prior to study drug administration. Historical data was obtained from medical or angioedema history electronic case report forms (eCRF).
Time Frame
Baseline up to Week 12
Title
Number of Participants With Angioedema Attacks in Different Anatomic Locations
Description
Anatomic locations where there was a presence of pain or swelling of any level of severity; mild, moderate or severe at any day during the attack were reported. Mild: the attack symptoms were noticeable but were easily tolerated by the participant and did not interfere with the participant's daily activities. Moderate: the attack symptoms interfered with the participant's ability to attend work/school or participate in family life and social/recreational activities and severe: the attack symptoms significantly limited the participant's ability to attend work/school or participate in family life and social/recreational activities. Number of participants with angioedema attacks in different anatomic locations in treatment period was compared to NNA for historical data. Historical data was based on the typical location of angioedema attacks in the 3 months prior to study drug administration. Here, H refers to historical and T refers to treatment.
Time Frame
Baseline up to Week 12
Title
Average Severity (Intensity) of Angioedema Attacks
Description
All attacks in each therapy period were assigned a value of 1 (mild), 2 (moderate), or 3 (severe). Attack severity was considered the highest value assigned by the participant to any swelling location on any day during the attack. The average severity was derived by dividing the cumulative severity score by the total number of attacks. Average severity was set to 0 if there was no attack in a period. Average severity of angioedema attacks in treatment period compared to the NNA of angioedema attacks for historical data was reported. Historical data was based on the typical severity of angioedema attacks in the 3 months prior to study drug administration. Historical data was obtained from medical or angioedema history electronic case report forms (eCRF).
Time Frame
Baseline up to Week 12
Title
Average Duration of Angioedema Attacks
Description
Average duration of attacks was calculated by dividing the cumulative duration of attacks by the total number of attacks during the treatment period. Historical data was based on the typical severity of angioedema attacks in the 3 months prior to study drug administration. Average duration of angioedema attacks in treatment period was compared to the NNA for historical data. Historical data was obtained from medical or angioedema history eCRF.
Time Frame
Baseline up to Week 12
Title
Normalized Number of Angioedema Attacks (NNA) Per Month Treated With Rescue Medication
Description
The normalized number of angioedema attacks was calculated as the overall number of angioedema attacks recorded during the period divided by the number of days in the period and multiplied by 30.4. NNA treated with rescue medications were reported for CINRYZE, non-CINRYZE C1-INH or not treated with C1-INH (including attacks treated with any medications other than C1-INH or untreated attacks). CINRYZE was only considered as a rescue medication when treated for breakthrough attack treatment. For historical data, only medications taken prior to the start of drug study drug administration and had an indication of "hereditary angioedema (HAE) management - acute treatment" selected on the prior and concomitant medications and therapy were considered as rescue medications. Historical data was obtained from medical or angioedema history eCRF.
Time Frame
Baseline up to Week 12
Title
Number of Participants Achieving Clinical Responder Rate Relative to Historical Data
Description
Number of participants achieving at least 50 percent (%), 70% or 90% reduction in NNA relative to NNA for historical data was reported.
Time Frame
Baseline up to Week 12
Title
Change From Baseline in Angioedema Quality of Life (AE-QoL) in Treatment Period
Description
Angioedema quality of life (AE-QoL) questionnaire was a self-administered validated angioedema disease-specific quality of life instrument. It consisted of 17 specific questions that were associated with work, physical activity, free time, social relations, and diet. Each of the 17 items had a 5-point response scale ranging from 1 (Never) to 5 (Very Often). The questionnaire was scored according to the developers' guidelines to produce a total score and 4 domain scores (functioning, fatigue/mood, fear/shame, nutrition). Raw domain scores (mean of the item scores within each scale) and the raw total score (mean of all item scores) were rescaled using linear transformations into final percentage scores ranging 0 to 100, based on the maximum possible score, where the higher the score the greater the QoL impairment.
Time Frame
Baseline, Week 12
Title
Number of Participants With Breakthrough Angioedema Attacks
Description
A breakthrough attack was defined as an angioedema attack that occurs during long-term prevention therapy with CINRYZE (that is, between first study drug and last study drug dose). Number of participants with 1, 2, 3 or more angioedema attacks and who achieved initial improvement and complete resolution were also reported. Breakthrough angioedema attacks assessed by CINRYZE treatment, non-CINRYZE C1 INH treatment and untreated with C1-INH were reported. Here BAA refers to breakthrough angioedema attacks.
Time Frame
Baseline up to Week 12
Title
Time From Attack Onset to Initial Improvement and Complete Resolution
Description
Time to initial improvement (TII) was calculated from the time of study drug administration to initial symptom improvement. Time to complete resolution was defined as the time from the onset of attack to complete resolution of all symptoms. Time to initial improvement and time to complete resolution as assessed by CINRYZE, non-CINRYZE and untreated were reported.
Time Frame
Baseline up to Week 12
Title
Time From Onset of Attack to Time Treated by CINRYZE
Description
The median time from onset of attack to time treated with CINRYZE was reported.
Time Frame
Baseline up to Week 12
Title
Time From Treatment With CINRYZE to Initial Improvement
Description
Time to initial improvement was calculated from the time of study drug administration to initial symptom improvement. Median time from treatment with CINRYZE to initial improvement was reported.
Time Frame
Baseline up to Week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents. Be ≥2 years of age. Meet the following minimum body weight criteria: Subjects 2 to 5 years of age must weigh at least 12.5 kg; and Subjects 6 years of age and above must weigh at least 25 kg. Have a confirmed diagnosis of Type I or Type II HAE. NOTE: Diagnosis may be based on historical data including family history, clinical symptoms (characteristic attacks), or documentation of low level of C1 INH protein and/or C1 INH activity. Have a history of at least one angioedema attack per month (on average) during the 3 consecutive months immediately before enrollment. Agree to adhere to the protocol-defined schedule of assessments and procedures. Agree to avoid his/her known angioedema attack triggers during the study to the best of his/her ability. If a female of reproductive age, be postmenopausal (≥12 months following cessation of menstruation), surgically sterile, or following an acceptable method of birth control (and agree to continue its use through 1 month after the last dose of study drug): Non-hormonal methods (eg, abstinence, barrier control) for at least 1 complete menstrual cycle before the Screening Visit. Stable doses of estrogen and/or progestin containing products for at least 2 months before the Screening Visit. If a male of reproductive age, be surgically sterile or agree to follow an acceptable method of birth control (eg, abstinence, barrier control) from the Screening Visit through 2 months after the last dose of study drug. If an adult, be informed of the nature of the study and provide written informed consent before any study-specific procedures are performed. OR If a child or minor (<20 years of age), have a parent/legal guardian who is informed of the nature of the study provide written informed consent (ie, permission) for the child to participate in the study before any study-specific procedures are performed. Assent will be obtained from children ≥14 years of age. Exclusion Criteria: Have a history of hypercoagulability (abnormal blood clotting). Have a diagnosis of acquired angioedema or be known to have C1 INH antibodies. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE) or other blood products. Have received C1 INH therapy or any blood products within 3 days before the first dose of study drug. Have had signs or symptoms of an angioedema attack within 2 days before the first dose of study drug. Have any change (start, stop, or change in dose) in androgen therapy (eg, danazol, oxandrolone, stanozolol, testosterone), tranexamic acid, epsilon-aminocaproic acid (EACA), or other antifibrinolytics within 14 days before the first dose of study drug. If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (eg, estrogen/progestin containing products) within 2 months before the first dose of study drug. Be pregnant or breastfeeding. Have received an investigational drug other than those required for prevention or treatment of angioedema attacks within 30 days before the first dose of study drug. Have, as determined by the Investigator and/or the Sponsor's Medical Monitor, any surgical or medical condition that could interfere with the administration of study drug or interpretation of study results.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Toyohashi Municipal Hospital
City
Toyohashi
State/Province
Aiti
ZIP/Postal Code
441-8570
Country
Japan
Facility Name
Gunma University Hospital
City
Maebashi
State/Province
Gunma
ZIP/Postal Code
371-8511
Country
Japan
Facility Name
Kobe University Hospital
City
Kobe
State/Province
Hyogo
ZIP/Postal Code
650-0017
Country
Japan
Facility Name
Heart Life Hospital
City
Nakagusuku
State/Province
Nakagami
ZIP/Postal Code
901-2417
Country
Japan
Facility Name
Naha City Hospital
City
Naha
State/Province
Okinawa
ZIP/Postal Code
902-8511
Country
Japan
Facility Name
Shiman University Hospital
City
Izumo
State/Province
Shimane
ZIP/Postal Code
693-8501
Country
Japan
Facility Name
Asahi General Hospital
City
Asahi
State/Province
Tiba
ZIP/Postal Code
289-2511
Country
Japan
Facility Name
Adachi kyosai Hospital
City
Adachi
State/Province
Tokyo
ZIP/Postal Code
120-0022
Country
Japan
Facility Name
Hiroshima University Hospital
City
Hiroshima
ZIP/Postal Code
734-8551
Country
Japan
Facility Name
Tomakomai City Hospital
City
Tomakomai
ZIP/Postal Code
053-8567
Country
Japan

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/

Learn more about this trial

Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)

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