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

Hemophilia B Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01271868
• Other study ID #: IB1001-02
• Status: Terminated
• Phase: Phase 3
• Start date: November 2010
• Est. completion date: December 2016

Study information

• Verified date: March 2021
• Source: Medexus Pharma, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 9
• Est. completion date: December 2016
• Est. primary completion date: December 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Biological:
• IB1001

Locations

Country	Name	City	State
India	AMRI Hospital, Institute of Haematology & Transfusion Medicine	Kolkata
India	Christian Medical College and Hospital	Ludhiana
India	Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises	Pune
India	Sahyadri Speciality Hospital	Pune
India	Bhailal Amin General Hospital	Vadodara
India	Christian Medical College	Vellore
United Kingdom	Great Ormond Street Hospital for Children	London
United States	RUSH University Medical Center	Chicago	Illinois
United States	Gulf States Hemophilia & Thrombophilia Center	Houston	Texas
United States	Indiana Hemophilia & Thrombosis Center	Indianapolis	Indiana

Sponsors (1)

Lead Sponsor	Collaborator
Medexus Pharma, Inc.

Countries where clinical trial is conducted

United States,  India,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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