Back to resultsStudy of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
Primary Purpose
Hemophilia B
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
IB1001
Sponsored by
About this trial
This is an interventional treatment trial for Hemophilia B focused on measuring Recombinant Factor IX
Eligibility Criteria
Inclusion Criteria:
- Subject's parent or legal guardian must give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to make the required study visits and follow instructions while enrolled in the study. For subjects ≥7 years of age, assent will be obtained if required by the institution. For subjects < 7 years of age, legal assent is not reasonable to obtain.
- Severe (factor IX activity ≤2 IU/dL) hemophilia B subjects currently on-demand therapy with a minimum of 2 bleeding episodes requiring factor IX therapy over the preceding 6 months or 4 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern similar to that above demonstrated prior to starting prophylaxis
- Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
- Previously treated patients with a minimum of 50 exposure days to a factor IX preparation
- Platelet count at least 150,000/mm3
- Liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times the upper limit of the normal range
- Total bilirubin ≤1.5 times the upper limit of the normal range
- Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
- Willingness to participate in the trial for approximately 6 months (50 exposures)
- Age ≤12 years
- Hemoglobin ≥7 g/dL at the time of the blood draw
Exclusion Criteria:
- History of factor IX inhibitor ≥0.6 Bethesda units (BU)
- Existence of another coagulation disorder
- Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation (DIC)
- Use of an investigational drug within 30 days prior to study entry
- On medications that could impact hemostasis, such as aspirin
- History of poor compliance, a serious medical or social condition, or any other circumstance that, in the opinion of the investigator, would interfere with participation or compliance with the study protocol
- History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subject's ability to treat bleeding episodes with a factor IX product
Sites / Locations
- RUSH University Medical Center
- Indiana Hemophilia & Thrombosis Center
- Gulf States Hemophilia & Thrombophilia Center
- AMRI Hospital, Institute of Haematology & Transfusion Medicine
- Christian Medical College and Hospital
- Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises
- Sahyadri Speciality Hospital
- Bhailal Amin General Hospital
- Christian Medical College
- Great Ormond Street Hospital for Children
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
IB1001
Arm Description
Outcomes
Primary Outcome Measures
Number of Infusions Required for Bleed Control
Secondary Outcome Measures
Area Under the Curve (0-inf)
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Terminal Half-life
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Concentration (Max)
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Incremental Recovery
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Clearance
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Volume of Distribution (Steady State)
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Annualized Bleed Rate
Measure was assessed during the Treatment Study
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01271868
Brief Title
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
Official Title
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
Study Type
Interventional
2. Study Status
Record Verification Date
March 2021
Overall Recruitment Status
Terminated
Study Start Date
November 2010 (Actual)
Primary Completion Date
December 2016 (Actual)
Study Completion Date
December 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Medexus Pharma, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric 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
Keywords
Recombinant Factor IX
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)
8. Arms, Groups, and Interventions
Arm Title
IB1001
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
IB1001
Other Intervention Name(s)
Recombinant factor IX, IXINITY
Primary Outcome Measure Information:
Title
Number of Infusions Required for Bleed Control
Time Frame
Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 39.7 ± 12.4 months
Secondary Outcome Measure Information:
Title
Area Under the Curve (0-inf)
Description
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Time Frame
Pre-infusion to 72 hours following infusion
Title
Terminal Half-life
Description
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Time Frame
Pre-infusion to 72 hours following infusion
Title
Concentration (Max)
Description
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Time Frame
Pre-infusion to 72 hours following infusion
Title
Incremental Recovery
Description
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Time Frame
Pre-infusion to 72 hours following infusion
Title
Clearance
Description
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Time Frame
Pre-infusion to 72 hours following infusion
Title
Volume of Distribution (Steady State)
Description
Factor IX levels were assessed at the following time points:
Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours
Time Frame
Pre-infusion to 72 hours following infusion
Title
Annualized Bleed Rate
Description
Measure was assessed during the Treatment Study
Time Frame
Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 41.7+/- 11.7 Months
10. Eligibility
Sex
All
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subject's parent or legal guardian must give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to make the required study visits and follow instructions while enrolled in the study. For subjects ≥7 years of age, assent will be obtained if required by the institution. For subjects < 7 years of age, legal assent is not reasonable to obtain.
Severe (factor IX activity ≤2 IU/dL) hemophilia B subjects currently on-demand therapy with a minimum of 2 bleeding episodes requiring factor IX therapy over the preceding 6 months or 4 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern similar to that above demonstrated prior to starting prophylaxis
Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
Previously treated patients with a minimum of 50 exposure days to a factor IX preparation
Platelet count at least 150,000/mm3
Liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times the upper limit of the normal range
Total bilirubin ≤1.5 times the upper limit of the normal range
Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
Willingness to participate in the trial for approximately 6 months (50 exposures)
Age ≤12 years
Hemoglobin ≥7 g/dL at the time of the blood draw
Exclusion Criteria:
History of factor IX inhibitor ≥0.6 Bethesda units (BU)
Existence of another coagulation disorder
Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation (DIC)
Use of an investigational drug within 30 days prior to study entry
On medications that could impact hemostasis, such as aspirin
History of poor compliance, a serious medical or social condition, or any other circumstance that, in the opinion of the investigator, would interfere with participation or compliance with the study protocol
History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subject's ability to treat bleeding episodes with a factor IX product
Facility Information:
Facility Name
RUSH University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Indiana Hemophilia & Thrombosis Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
Gulf States Hemophilia & Thrombophilia Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
AMRI Hospital, Institute of Haematology & Transfusion Medicine
City
Kolkata
ZIP/Postal Code
700073
Country
India
Facility Name
Christian Medical College and Hospital
City
Ludhiana
ZIP/Postal Code
141008
Country
India
Facility Name
Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises
City
Pune
ZIP/Postal Code
411001
Country
India
Facility Name
Sahyadri Speciality Hospital
City
Pune
ZIP/Postal Code
411004
Country
India
Facility Name
Bhailal Amin General Hospital
City
Vadodara
ZIP/Postal Code
390003
Country
India
Facility Name
Christian Medical College
City
Vellore
ZIP/Postal Code
632004
Country
India
Facility Name
Great Ormond Street Hospital for Children
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
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