Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A

NCT ID: NCT00157040

Last Updated: 2021-04-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2002-06-07

Study Completion Date

2005-01-04

Brief Summary

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The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts. Part 1 of the study is a pharmacokinetic evaluation, and Part 2 is an evaluation of efficacy and safety.

Detailed Description

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Conditions

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Hemophilia A

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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Antihemophilic factor, recombinant, manufactured protein-free

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Subject is less than 6 years of age
* Severe or moderately-severe hemophilia A as defined by a baseline factor VIII level \<= 2%, documented at screening or on the basis of historical data (e.g., at hemophilia diagnosis)
* Documented medical history of at least 50 exposure days for treatment with all other factor VIII products
* Subject's parent or legally authorized representative has provided informed consent

Exclusion Criteria

* Detectable inhibitor to factor VIII measured in the screening sample by the local or central hemostasis laboratory
* History of inhibitor to factor VIII at any time prior to screening
* Subject has any one of the following laboratory abnormalities at the time of screening:

1. platelet count \< 100,000/mm3
2. hemoglobin concentration \< 10 g/dL (100 g/L)
3. serum creatinine \> 1.5 times the ULN for age
4. total bilirubin \> 2 times the ULN for age
* Subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., platelet dysfunction secondary to uremia, liver failure, von Willebrand's Disease)
* Subject has known hypersensitivity to RECOMBINATE rAHF
* Subject is currently participating in another investigational drug study or has participated in any clinical study involving an investigational drug within 30 days of study entry
* Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
Maximum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Baxalta now part of Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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Children´s Hospital Los Angeles

Los Angeles, California, United States

Site Status

Emory University, Department of Pediatrics

Atlanta, Georgia, United States

Site Status

Children´s Memorial Hospital

Chicago, Illinois, United States

Site Status

Comprehensive Bleeding Disorders Center

Peoria, Illinois, United States

Site Status

Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, United States

Site Status

University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics

Iowa City, Iowa, United States

Site Status

University of Michigan Hemophilia Treatment Center

Ann Arbor, Michigan, United States

Site Status

Children´s Hospital of Michigan

Detroit, Michigan, United States

Site Status

Children´s Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

University of Texas Health Science Center

Houston, Texas, United States

Site Status

Hospital for Sick Children, Division of Hematology/Oncology

Toronto, Ontario, Canada

Site Status

University Pediatric Hospital

San Juan, , Puerto Rico

Site Status

Countries

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United States Canada Puerto Rico

References

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Shapiro A, Gruppo R, Pabinger I, Collins PW, Hay CR, Schroth P, Casey K, Patrone L, Ehrlich H, Ewenstein BM. Integrated analysis of safety and efficacy of a plasma- and albumin-free recombinant factor VIII (rAHF-PFM) from six clinical studies in patients with hemophilia A. Expert Opin Biol Ther. 2009 Mar;9(3):273-83. doi: 10.1517/14712590902729392.

Reference Type DERIVED
PMID: 19216617 (View on PubMed)

Other Identifiers

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060101

Identifier Type: -

Identifier Source: org_study_id

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