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

August 19, 2016 updated by: Cangene Corporation

Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients With Severe Hemophilia B

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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)

Study Type

Interventional

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: SUPPORTIVE_CARE
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of study subjects with adverse events
Time Frame: Within 6 months
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 Outcome Measures

Outcome Measure
Time Frame
Number of bleeding episodes divided by number of months of observation
Time Frame: Within 12 months
Within 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cangene Corporation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2014

Primary Completion (ANTICIPATED)

October 1, 2015

Study Completion (ANTICIPATED)

November 1, 2015

Study Registration Dates

First Submitted

November 14, 2013

First Submitted That Met QC Criteria

January 27, 2014

First Posted (ESTIMATE)

January 29, 2014

Study Record Updates

Last Update Posted (ESTIMATE)

August 23, 2016

Last Update Submitted That Met QC Criteria

August 19, 2016

Last Verified

August 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IB1001-04

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hemophilia B

Clinical Trials on IB1001

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Malawi

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe