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

NCT ID: NCT00768287

Last Updated: 2021-04-06

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

77 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-01-31

Study Completion Date

2016-12-31

Brief Summary

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Primary Objective:

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

Key Secondary Objectives:

To evaluate the ability of IB1001 to provide coverage against bleeding under surgical circumstances; To evaluate the long-term safety and efficacy of IB1001

Detailed Description

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Conditions

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Hemophilia B

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Study Part 1: Randomized, double-blind, cross-over study of pharmacokinetics; Study Part 2: Non-randomized, open-label study of safety and efficacy; Surgical sub-study: Non-randomized, open-label study of safety and efficacy during major surgery
Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Study Part 1 only

Study Groups

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IB1001

Group Type EXPERIMENTAL

IB1001

Intervention Type BIOLOGICAL

Study Part 1: Randomized, double-blind, cross-over study with IB1001 and nonacog alfa; Study Part 2: Non-randomized, open-label evaluation of prophylaxis and on demand IB1001; Surgical Sub-study: Open-label evaluation of IB1001 during major surgery

nonacog alfa

Group Type ACTIVE_COMPARATOR

nonacog alfa

Intervention Type BIOLOGICAL

Study Part 1: Randomized, double-blind, cross-over study with IB1001 and nonacog alfa

Interventions

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IB1001

Study Part 1: Randomized, double-blind, cross-over study with IB1001 and nonacog alfa; Study Part 2: Non-randomized, open-label evaluation of prophylaxis and on demand IB1001; Surgical Sub-study: Open-label evaluation of IB1001 during major surgery

Intervention Type BIOLOGICAL

nonacog alfa

Study Part 1: Randomized, double-blind, cross-over study with IB1001 and nonacog alfa

Intervention Type BIOLOGICAL

Other Intervention Names

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Recombinant factor IX (rFIX) IXINITY Recombinant factor IX (rFIX) BeneFIX

Eligibility Criteria

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Inclusion Criteria

1. Patient must be willing to give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent, make the required study visits, and follow instructions while enrolled in the study
2. Severe (factor IX activity ≤2 U/dL) hemophilia B subjects on demand therapy with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern as above demonstrated prior to starting prophylaxis
3. Immunocompetent (CD4 count \>400/mm3) and not receiving immune modulating or chemotherapeutic agents
4. Previously treated patients with a minimum of 150 exposure days to a factor IX preparation
5. Platelet count at least 150,000/mm3
6. Liver function: alanine transaminase \[ALT\] and aspartate transaminase \[AST\] ≤2 times the upper limit of the normal range
7. Total bilirubin ≤1.5 times the upper limit of the normal range
8. Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
9. Willingness to participate in the trial for up to 12-15 months
10. European Union (EU), Israel, and Canada: Age of at least 12 years and body weight of ≥40 kilograms to participate in any PK Study or the Surgical Sub-study \[the Surgical Sub-study does not apply to the UK\]; age of at least 12 years for the prophylaxis and on demand components of the Treatment Phase and Continuation Study

United States (US): Age of at least 12 years and body weight of ≥40 kilograms to participate in any PK Study or the Surgical Sub-study; age of at least 5 years for the prophylaxis and on demand components of the Treatment Phase and Continuation Study
11. Hemoglobin ≥7 g/dL at the time of the blood draw

Exclusion Criteria

1. History of factor IX inhibitor ≥0.6 Bethesda units (BU)
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. On medications that could impact hemostasis, such as aspirin
6. 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
7. 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
Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Medexus Pharma, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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City of Hope

Duarte, California, United States

Site Status

The Hemophilia Treatment Center of Orthopaedic Hospital

Los Angeles, California, United States

Site Status

Emory University School of Medicine Pediatric Hematology

Atlanta, Georgia, United States

Site Status

Rush University Medical Center-Pediatric Hematology Oncology

Chicago, Illinois, United States

Site Status

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, United States

Site Status

University of Minnesota Center for Bleeding and Clotting Disorder

Minneapolis, Minnesota, United States

Site Status

Hemophilia Treatment Center of Las Vegas

Las Vegas, Nevada, United States

Site Status

Hemophilia and Thrombosis Center

Cincinnati, Ohio, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

University of Texas Health Science Center-Houston, Gulf States Hemophilia & Thrombophilia Center

Houston, Texas, United States

Site Status

Centre Regional de Traitement de l 'Hemophilie

Nantes, Loire-Atlantique, France

Site Status

Hopital Edouard Herriot

Lyon, , France

Site Status

Sahyadri Specialty Hospital, Deccan Gymkhana

Pune, Maharashtra, India

Site Status

Jehangir Clinical Development Centre

Pune, Maharashtra, India

Site Status

The National Hemophilia Center-Sheba MC

Tel Litwinsky, Ramat Gan, Israel

Site Status

Ospedale di Careggi

Florence, , Italy

Site Status

University of Milan

Milan, , Italy

Site Status

MTZ Clinical Research

Warsaw, , Poland

Site Status

Royal Free Hospital

London, England, United Kingdom

Site Status

Manchester Haemophilia Comprehensive Care Manchester Royal Infirmary

Manchester, England, United Kingdom

Site Status

Royal Hallamshire Hospital

Sheffield, England, United Kingdom

Site Status

Centre for Haemostasis and Thrombosis, Basingstoke and North Hampshire Foundation Trust

Basingstoke, Hampshire, United Kingdom

Site Status

University Hospital of Wales Health Park

Cardiff, Wales, United Kingdom

Site Status

Countries

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United States France India Israel Italy Poland United Kingdom

References

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Collins PW, Quon DVK, Makris M, Chowdary P, Kempton CL, Apte SJ, Ramanan MV, Hay CRM, Drobic B, Hua Y, Babinchak TJ, Gomperts ED. Pharmacokinetics, safety and efficacy of a recombinant factor IX product, trenonacog alfa in previously treated haemophilia B patients. Haemophilia. 2018 Jan;24(1):104-112. doi: 10.1111/hae.13324. Epub 2017 Aug 17.

Reference Type RESULT
PMID: 28833808 (View on PubMed)

Martinowitz U, Shapiro A, Quon DV, Escobar M, Kempton C, Collins PW, Chowdary P, Makris M, Mannucci PM, Morfini M, Valentino LA, Gomperts E, Lee M. Pharmacokinetic properties of IB1001, an investigational recombinant factor IX, in patients with haemophilia B: repeat pharmacokinetic evaluation and sialylation analysis. Haemophilia. 2012 Nov;18(6):881-7. doi: 10.1111/j.1365-2516.2012.02897.x. Epub 2012 Jul 5.

Reference Type DERIVED
PMID: 22764744 (View on PubMed)

Other Identifiers

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IB1001-01

Identifier Type: -

Identifier Source: org_study_id

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