Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B - Full Text View

Purpose

The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B.

Condition	Intervention	Phase
Hemophilia B	Drug: Recombinant factor IX Product, IB1001	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Study of Inspiration's Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia

Drug Information available for: Factor IX

U.S. FDA Resources

Further study details as provided by Inspiration Biopharmaceuticals, Inc.:

Primary Outcome Measures:

Pharmacokinetics, Safety and Efficacy [ Time Frame: 50 exposure days ] [ Designated as safety issue: Yes ]
To evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B. Acute effects are defined as adverse events (AEs) which occur within the first 72 hours after the administration of a study product.

Estimated Enrollment:	22
Study Start Date:	November 2010
Estimated Primary Completion Date:	April 2016 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Age group 1 Age less than 6 years old	Drug: Recombinant factor IX Product, IB1001 On-Demand or Prophylaxis
Age group 2 Age between 6 to 12 years old	Drug: Recombinant factor IX Product, IB1001 On-Demand or Prophylaxis

Detailed Description:

Primary Objective(s):

To evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B. Acute effects are defined as adverse events (AEs) which occur within the first 72 hours after the administration of a study product.

Secondary Objectives:

To obtain tolerance and compliance information on pediatric subject response to an intravenously delivered recombinant factor IX product , IB1001
To evaluate the safety of IB1001 during the 50 exposure days treatment course

Investigators/Study Centers:

This international study will be conducted at clinical sites located in the United States (USA), Europe, Mexico and India

Phase of Development: Phase 3/4

Planned Sample Size:

Up to 22 pediatric subjects (<12 years of age) will be enrolled in order to have 20 subjects complete the study.

Study Duration:

The study duration will be approximately 6 months per subject to include a minimum of 50 exposures to IB1001.

Study Design:

The study is a non-randomized, open label design that includes an initial pharmacokinetics evaluation in all participants, followed by six months (50 exposure days) of treatment.

Eligibility

Ages Eligible for Study:	up to 12 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Subject's parent or legal guardian must give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to make the required study visits and follow instructions while enrolled in the study. For subjects ≥7 years of age, assent will be obtained if required by the institution. For subjects < 7 years of age, legal assent is not reasonable to obtain.
Severe (factor IX activity ≤2 IU/dL) hemophilia B subjects currently on-demand therapy with a minimum of 2 bleeding episodes requiring factor IX therapy over the preceding 6 months or 4 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern similar to that above demonstrated prior to starting prophylaxis
Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
Previously treated patients with a minimum of 50 exposure days to a factor IX preparation
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
Willingness to participate in the trial for approximately 6 months (50 exposures)
Age ≤12 years
Hemoglobin ≥7 g/dL at the time of the blood draw

Exclusion Criteria:

History of factor IX inhibitor ≥0.6 Bethesda units (BU)
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
On medications that could impact hemostasis, such as aspirin
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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01271868

Locations

United States, Illinois
RUSH University Medical Center
Chicago, Illinois, United States, 60612
United States, Indiana
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, United States, 46260
United States, Texas
Gulf States Hemophilia & Thrombophilia Center
Houston, Texas, United States, 77030
India
AMRI Hospital, Institute of Haematology & Transfusion Medicine
Kolkata, India, 700073
Christian Medical College and Hospital
Ludhiana, India, 141008
Sahyadri Speciality Hospital
Pune, India, 411004
Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises
Pune, India, 411001
Bhailal Amin General Hospital
Vadodara, India, 390003
Christian Medical College
Vellore, India, 632004
United Kingdom
Great Ormond Street Hospital for Children
London, United Kingdom, WC1N 3JH

Sponsors and Collaborators

Inspiration Biopharmaceuticals, Inc.

More Information

No publications provided

Responsible Party:	Inspiration Biopharmaceuticals, Inc.
ClinicalTrials.gov Identifier:	NCT01271868 History of Changes
Other Study ID Numbers:	IB1001-02
Study First Received:	January 6, 2011
Last Updated:	April 24, 2013
Health Authority:	United States: Food and Drug Administration

Keywords provided by Inspiration Biopharmaceuticals, Inc.:

Recombinant Factor IX

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 May 16, 2013