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

Sponsor
Baxalta now part of Shire (Industry)
Overall Status
Completed
CT.gov ID
NCT00157040
Collaborator
(none)
50
Enrollment
12
Locations
30.9
Actual Duration (Months)
4.2
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Antihemophilic factor, recombinant, manufactured protein-free
 Phase 2/Phase 3

Study Design

Study Type:
Interventional
Allocation:
Non-Randomized
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Recombinant Antihemophilic Factor (rAHF-PFM) Manufactured and Formulated Without Added Human or Animal Proteins: Evaluation of Pharmacokinetics, Immunogenicity, Efficacy and Safety in Previously Treated Pediatric Patients With Hemophilia A
Actual Study Start Date :
Jun 7, 2002
Actual Primary Completion Date :
Jan 4, 2005
Actual Study Completion Date :
Jan 4, 2005

Outcome Measures

Primary Outcome Measures

  1. The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM) [Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion]

    Terminal phase half-life of rAHF-PFM was to be determined using the biphasic linear regression model.

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A to 6 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Children´s Hospital Los Angeles Los Angeles California United States 90027
2 Emory University, Department of Pediatrics Atlanta Georgia United States 30322
3 Children´s Memorial Hospital Chicago Illinois United States 60614
4 Comprehensive Bleeding Disorders Center Peoria Illinois United States 61614
5 Indiana Hemophilia and Thrombosis Center Indianapolis Indiana United States 46260
6 University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics Iowa City Iowa United States 52242
7 University of Michigan Hemophilia Treatment Center Ann Arbor Michigan United States 48109
8 Children´s Hospital of Michigan Detroit Michigan United States 48201
9 Children´s Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
10 University of Texas Health Science Center Houston Texas United States 77030
11 Hospital for Sick Children, Division of Hematology/Oncology Toronto Ontario Canada M5G 1X8
12 University Pediatric Hospital San Juan Puerto Rico 00927

Sponsors and Collaborators

  • Baxalta now part of Shire

Investigators

  • Study Director: Study Director, Takeda

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Baxalta now part of Shire
ClinicalTrials.gov Identifier:
NCT00157040
Other Study ID Numbers:
  • 060101
First Posted:
Sep 12, 2005
Last Update Posted:
Apr 27, 2021
Last Verified:
Apr 1, 2021
Keywords provided by Baxalta now part of Shire
Factor VIII Deficiency
Additional relevant MeSH terms:
Hemophilia A
Factor VIII

Study Results

No Results Posted as of Apr 27, 2021