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

NCT01271868 | Terminated Phase 3 Results DMC

• 1 other identifier: IB1001-02
• Study Type: interventional
• Target: 9
• Locations: 3 countries
• Sites: 10

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.

Conditions

Hemophilia B

Keywords

Recombinant Factor IX

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (7)

• Area Under the Curve (0-inf)

  Pre-infusion to 72 hours following infusion

• Terminal Half-life

  Pre-infusion to 72 hours following infusion

• Concentration (Max)

  Pre-infusion to 72 hours following infusion

• Incremental Recovery

  Pre-infusion to 72 hours following infusion

• Clearance

  Pre-infusion to 72 hours following infusion

Study Arms (1)

IB1001

EXPERIMENTAL

Biological: IB1001

Interventions

IB1001 BIOLOGICAL

Also known as: Recombinant factor IX, IXINITY

IB1001

Eligibility Criteria

• Age: Up to 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Medexus Pharma, Inc.: lead

Study Sites (10)

RUSH University Medical Center

Chicago, Illinois, 60612, United States

Location

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, 46260, United States

Location

Gulf States Hemophilia & Thrombophilia Center

Houston, Texas, 77030, United States

Location

AMRI Hospital, Institute of Haematology & Transfusion Medicine

Kolkata, 700073, India

Location

Christian Medical College and Hospital

Ludhiana, 141008, India

Location

Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises

Pune, 411001, India

Location

Sahyadri Speciality Hospital

Pune, 411004, India

Location

Bhailal Amin General Hospital

Vadodara, 390003, India

Location

Christian Medical College

Vellore, 632004, India

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia B

Interventions

IB1001

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Limitations and Caveats

Early termination leading to a small number of subjects analyzed

Results Point of Contact

• Title: Dr. David Schaaf
• Organization: Aptevo Therapeutics

schaafd@apvo.com

206-859-6655

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 6, 2011
• First Posted: January 7, 2011
• Study Start: November 1, 2010
• Primary Completion: December 1, 2016
• Study Completion: December 1, 2016
• Last Updated: March 15, 2021
• Results First Posted: April 11, 2019

Record last verified: 2021-03

Locations

US (3); IN (6); GB (1)