Trial record 6 of 1632 for:    Open Studies | "Genetic Diseases, Inborn"

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

This study is not yet open for participant recruitment.
Verified January 2014 by Cangene Corporation
Sponsor:
Information provided by (Responsible Party):
Cangene Corporation
ClinicalTrials.gov Identifier:
NCT02048111
First received: November 14, 2013
Last updated: January 27, 2014
Last verified: January 2014
  Purpose

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.


Condition Intervention Phase
Hemophilia B
Biological: IB1001
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
Official Title: Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients With Severe Hemophilia B

Resource links provided by NLM:


Further study details as provided by Cangene Corporation:

Primary Outcome Measures:
  • Number of study subjects with adverse events [ Time Frame: Within 6 months ] [ Designated as safety issue: Yes ]
    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.


Secondary Outcome Measures:
  • Number of bleeding episodes divided by number of months of observation [ Time Frame: Within 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 18
Study Start Date: March 2014
Estimated Study Completion Date: November 2015
Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: IB1001 Biological: IB1001

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.


Detailed Description:

Primary Objectives:

  • to evaluate safety of IB1001 within the first 50 exposure days,
  • to determine IB1001 pharmacokinetics (PK), and
  • to assess efficacy of IB1001 prophylaxis with respect to breakthrough bleeding and with respect to control of hemorrhaging in subjects with severe hemophilia B within the first 50 exposure days

Secondary Objectives:

  • to evaluate long-term safety of IB1001; and
  • to evaluate long term efficacy of IB1001.

Exploratory Objectives:

  • to evaluate markers of thrombogenicity during the first 24 hours post-infusion [thrombogenicity markers will include at a minimum D-dimer test; however should there be a clinical reason (e.g., three consecutive elevations in D-dimer levels, a possible clinical thrombogenic episode), sufficient samples will be collected to also evaluate levels of fragment 1+2 (F1+2) and thrombin-antithrombin III complex (TAT)]
  • to evaluate IB1001 immunogenicity response (development of inhibitory and non-inhibitory factor IX binding antibodies and antibodies to host cell proteins)
  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age of at least 12 years
  2. Body Mass Index of ≤ 29, with a minimum body weight of 40 kg
  3. Written Institutional Review Board (IRB)/ Ethics Committee (EC)-approved informed consent form (ICF)
  4. Willingness to make the required study visits, and follow instructions while enrolled in the study (up to 12 months)
  5. 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
  6. Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of the PK and Treatment/Continuation Phase of the study
  7. Previously treated patients with a minimum of 150 exposure days to a factor IX preparation
  8. Willingness to adhere to the 5-day washout of any factor IX replacement therapy prior to PK evaluations
  9. Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
  10. Platelet count at least 150,000/mm3
  11. Liver function: alanine transaminase (ALT) and aspartate transaminase (AST) ≤2 times the upper limit of the normal range
  12. Total bilirubin ≤1.5 times the upper limit of the normal range
  13. Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
  14. Hemoglobin ≥7 g/dL at the time of the blood draw

Exclusion Criteria:

  1. History of factor IX inhibitor ≥0.6 BU (Bethesda units)
  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. Previous use of IB1001
  6. Use of medications that could impact hemostasis, such as aspirin
  7. Hypersensitivity to the active substance or to any of the excipients in the investigational products
  8. Known allergic reaction to hamster proteins
  9. 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
  10. 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT02048111

Contacts
Contact: Fran Yadao, Manager Clinical Operations fyadao@cangene.com

Locations
United States, Illinois
Not yet recruiting
Chicago, Illinois, United States
United Kingdom
Not yet recruiting
Manchester, United Kingdom
Sponsors and Collaborators
Cangene Corporation
  More Information

No publications provided

Responsible Party: Cangene Corporation
ClinicalTrials.gov Identifier: NCT02048111     History of Changes
Other Study ID Numbers: IB1001-04
Study First Received: November 14, 2013
Last Updated: January 27, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Cangene Corporation:
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

Additional relevant MeSH terms:
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

ClinicalTrials.gov processed this record on April 16, 2014