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

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.

Study Overview

• Status: Terminated
• Conditions: Hemophilia B
• Intervention / Treatment: Biological: IB1001

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 3

Contacts and Locations

Study Locations

• India
  • Kolkata, India, 700073
    • AMRI Hospital, Institute of Haematology & Transfusion Medicine
  • Ludhiana, India, 141008
    • Christian Medical College and Hospital
  • Pune, India, 411004
    • Sahyadri Speciality Hospital
  • Pune, India, 411001
    • Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises
  • Vadodara, India, 390003
    • Bhailal Amin General Hospital
  • Vellore, India, 632004
    • Christian medical college
• United Kingdom
  • London, United Kingdom, WC1N 3JH
    • Great Ormond Street Hospital for Children
• United States
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Indiana Hemophilia & Thrombosis Center
  • Texas
    • Houston, Texas, United States, 77030
      • Gulf States Hemophilia & Thrombophilia Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 10 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IB1001	Biological: IB1001; Other Names: Recombinant factor IX; IXINITY

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Outcome Measure	Measure Description	Time Frame
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	Pre-infusion to 72 hours following infusion
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	Pre-infusion to 72 hours following infusion
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	Pre-infusion to 72 hours following infusion
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	Pre-infusion to 72 hours following infusion
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	Pre-infusion to 72 hours following infusion
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	Pre-infusion to 72 hours following infusion
Annualized Bleed Rate	Measure was assessed during the Treatment Study	Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 41.7+/- 11.7 Months

Collaborators and Investigators

• Sponsor: Medexus Pharma, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2010
• Primary Completion (Actual): December 1, 2016
• Study Completion (Actual): December 1, 2016

Study Registration Dates

• First Submitted: January 6, 2011
• First Submitted That Met QC Criteria: January 6, 2011
• First Posted (Estimate): January 7, 2011

Study Record Updates

• Last Update Posted (Actual): March 15, 2021
• Last Update Submitted That Met QC Criteria: March 11, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Keywords: Recombinant Factor IX
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: IB1001-02