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

NCT02048111 | Withdrawn Phase 3 DMC

• 1 other identifier: IB1001-04
• Study Type: interventional
• Target: N/A
• Locations: 2 countries
• Sites: 2

Brief Summary

To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of IB1001 in subjects with hemophilia B.

Conditions

Hemophilia B

Keywords

Hemophilia B; Hemophilia A; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked

Outcome Measures

Primary Outcomes (1)

• Number of study subjects with adverse events

  Information on adverse events is collected after each infusion of study drug by a study subject. Assessment of adverse events is then performed by an investigator after 5 infusions of study drug, 1 month, 2 months, 3 months and 6 months of study drug treatment.

  Within 6 months

Secondary Outcomes (1)

• Number of bleeding episodes divided by number of months of observation

  Within 12 months

Study Arms (1)

IB1001

EXPERIMENTAL

Biological: IB1001

Interventions

IB1001 BIOLOGICAL

Prophylaxis (during Treatment and Continuation phases): 40 - 75 IU/kg twice weekly. The starting dose for prophylaxis may be based on previous recombinant factor IX product use. The recommended starting prophylaxis dose is 40 - 60 IU/kg twice weekly, however, the investigator may prescribe up to 75 IU/kg twice weekly at their discretion (after clinically assessing the subject) and discretion of the subject. The dose or the frequency of IB1001 prophylaxis may be adjusted at the discretion of the investigator.

IB1001

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age of at least 12 years
• Body Mass Index of ≤ 29, with a minimum body weight of 40 kg
• Written Institutional Review Board (IRB)/ Ethics Committee (EC)-approved informed consent form (ICF)
• Willingness to make the required study visits, and follow instructions while enrolled in the study (up to 12 months)
• Severe (factor IX activity ≤2 U/dL) hemophilia B patients with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months or in the event the subject is on prophylaxis, a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months prior to being placed on prophylaxis
• Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of the PK and Treatment/Continuation Phase of the study
• Previously treated patients with a minimum of 150 exposure days to a factor IX preparation
• Willingness to adhere to the 5-day washout of any factor IX replacement therapy prior to PK evaluations
• Immunocompetent (CD4 count \>400/mm3) and not receiving immune modulating or chemotherapeutic agents
• 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
• Hemoglobin ≥7 g/dL at the time of the blood draw

You may not qualify if:

• History of factor IX inhibitor ≥0.6 BU (Bethesda units)
• 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
• Previous use of IB1001
• Use of medications that could impact hemostasis, such as aspirin
• Hypersensitivity to the active substance or to any of the excipients in the investigational products
• Known allergic reaction to hamster proteins
• 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

• Cangene Corporation: lead

Study Sites (2)

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia B; Hemophilia A; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked

Interventions

IB1001

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

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

Study Record Dates

• First Submitted: November 14, 2013
• First Posted: January 29, 2014
• Study Start: March 1, 2014
• Primary Completion: October 1, 2015
• Study Completion: November 1, 2015
• Last Updated: August 23, 2016

Record last verified: 2016-08

Locations

GB (1); US (1)