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

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00157040
• Other study ID #: 060101
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: June 7, 2002
• Est. completion date: January 4, 2005

Study information

• Verified date: April 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 50
• Est. completion date: January 4, 2005
• Est. primary completion date: January 4, 2005
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 6 Years

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Antihemophilic factor, recombinant, manufactured protein-free

Locations

Country	Name	City	State
Canada	Hospital for Sick Children, Division of Hematology/Oncology	Toronto	Ontario
Puerto Rico	University Pediatric Hospital	San Juan
United States	University of Michigan Hemophilia Treatment Center	Ann Arbor	Michigan
United States	Emory University, Department of Pediatrics	Atlanta	Georgia
United States	Children´s Memorial Hospital	Chicago	Illinois
United States	Children´s Hospital of Michigan	Detroit	Michigan
United States	University of Texas Health Science Center	Houston	Texas
United States	Indiana Hemophilia and Thrombosis Center	Indianapolis	Indiana
United States	University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics	Iowa City	Iowa
United States	Children´s Hospital Los Angeles	Los Angeles	California
United States	Comprehensive Bleeding Disorders Center	Peoria	Illinois
United States	Children´s Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Baxalta now part of Shire

Countries where clinical trial is conducted

United States,  Canada,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM)	Terminal phase half-life of rAHF-PFM was to be determined using the biphasic linear regression model.	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