Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

Phase 3

Terminated

• Conditions: Hemophilia B
• Interventions: Biological: IB1001

• Registration Number: NCT01271868
• Lead Sponsor: Medexus Pharma, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-11
• Study First Submitted: 2011-01-06
• Study First Submitted QC: 2011-01-06
• Study First Posted: 2011-01-07
• Primary Completion: 2016-12
• Study Completion: 2016-12
• Disposition First Submitted: 2019-01-10
• Disposition First Submitted QC: 2019-01-10
• Disposition First Posted: 2019-02-08
• Results First Submitted: 2019-02-28
• Results First Submitted QC: 2019-03-20
• Results First Posted: 2019-04-11
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-11
• Last Update Posted: 2021-03-15

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

1. 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.
2. 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
3. Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
4. Previously treated patients with a minimum of 50 exposure days to a factor IX preparation
5. Platelet count at least 150,000/mm3
6. Liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times the upper limit of the normal range
7. Total bilirubin ≤1.5 times the upper limit of the normal range
8. Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
9. Willingness to participate in the trial for approximately 6 months (50 exposures)
10. Age ≤12 years
11. Hemoglobin ≥7 g/dL at the time of the blood draw

Exclusion Criteria

1. History of factor IX inhibitor ≥0.6 Bethesda units (BU)
2. Existence of another coagulation disorder
3. Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation (DIC)
4. Use of an investigational drug within 30 days prior to study entry
5. On medications that could impact hemostasis, such as aspirin
6. 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
7. 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
IB1001	IB1001	-

Primary Outcome Measures

Name	Time	Method
Number of Infusions Required for Bleed Control	Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 39.7 ± 12.4 months

Secondary Outcome Measures

Name	Time	Method
Area Under the Curve (0-inf)	Pre-infusion to 72 hours following infusion	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	Pre-infusion to 72 hours following infusion	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)	Pre-infusion to 72 hours following infusion	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	Pre-infusion to 72 hours following infusion	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	Pre-infusion to 72 hours following infusion	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)	Pre-infusion to 72 hours following infusion	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	Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 41.7+/- 11.7 Months	Measure was assessed during the Treatment Study

Trial Locations

Locations (10)

Gulf States Hemophilia & Thrombophilia Center; 🇺🇸 Houston, Texas, United States

RUSH University Medical Center; 🇺🇸 Chicago, Illinois, United States

Christian Medical College and Hospital; 🇮🇳 Ludhiana, India

AMRI Hospital, Institute of Haematology & Transfusion Medicine; 🇮🇳 Kolkata, India

Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises; 🇮🇳 Pune, India

Bhailal Amin General Hospital; 🇮🇳 Vadodara, India

Great Ormond Street Hospital for Children; 🇬🇧 London, United Kingdom

Christian Medical College; 🇮🇳 Vellore, India

Sahyadri Speciality Hospital; 🇮🇳 Pune, India

Indiana Hemophilia & Thrombosis Center; 🇺🇸 Indianapolis, Indiana, United States