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

Primary Purpose

Hemophilia B

Status

Withdrawn

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

IB1001

About this trial

This is an interventional supportive care trial for Hemophilia B focused on measuring 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

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

IB1001

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Number of bleeding episodes divided by number of months of observation

Full Information

NCT ID

NCT02048111

First Posted

November 14, 2013

Last Updated

August 19, 2016

Sponsor

Cangene Corporation

1. Study Identification

Unique Protocol Identification Number

NCT02048111

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2016

Overall Recruitment Status

Withdrawn

Why Stopped

The study was withdrawn from the IND.

Study Start Date

March 2014 (undefined)

Primary Completion Date

October 2015 (Anticipated)

Study Completion Date

November 2015 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Cangene Corporation

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Supportive Care

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

IB1001

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

IB1001

Intervention Description

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.

Primary Outcome Measure Information:

Title

Number of study subjects with adverse events

Description

Time Frame

Within 6 months

Secondary Outcome Measure Information:

Title

Number of bleeding episodes divided by number of months of observation

Time Frame

Within 12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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

Facility Information:

City

Chicago

State/Province

Illinois

Country

United States

City

Manchester

Country

United Kingdom

12. IPD Sharing Statement

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

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