Overview

This trial is active, not recruiting.

Condition hemophilia b
Treatment recombinant factor ix product, ib1001
Phase phase 3
Sponsor Cangene Corporation
Start date November 2010
End date April 2016
Trial size 22 participants
Trial identifier NCT01271868, IB1001-02

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Other)
Age less than 6 years old
recombinant factor ix product, ib1001
On-Demand or Prophylaxis
(Other)
Age between 6 to 12 years old
recombinant factor ix product, ib1001
On-Demand or Prophylaxis

Primary Outcomes

Measure
Pharmacokinetics, Safety and Efficacy
time frame: 50 exposure days

Eligibility Criteria

Male or female participants up to 12 years old.

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

Additional Information

Official title Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
Description Primary Objective(s): 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. Acute effects are defined as adverse events (AEs) which occur within the first 72 hours after the administration of a study product. Secondary Objectives: 1. To obtain tolerance and compliance information on pediatric subject response to an intravenously delivered recombinant factor IX product , IB1001 2. To evaluate the safety of IB1001 during the 50 exposure days treatment course Investigators/Study Centers: This international study will be conducted at clinical sites located in the United States (USA), Europe, Mexico and India Phase of Development: Phase 3/4 Planned Sample Size: Up to 22 pediatric subjects (<12 years of age) will be enrolled in order to have 20 subjects complete the study. Study Duration: The study duration will be approximately 6 months per subject to include a minimum of 50 exposures to IB1001. Study Design: The study is a non-randomized, open label design that includes an initial pharmacokinetics evaluation in all participants, followed by six months (50 exposure days) of treatment.
Trial information was received from ClinicalTrials.gov and was last updated in June 2013.
Information provided to ClinicalTrials.gov by Cangene Corporation.