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

Phase 2

Completed

• Conditions: Hemophilia A

• Registration Number: NCT00157040
• Lead Sponsor: Baxalta now part of Shire

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2002-06-07
• Primary Completion: 2005-01-04
• Study Completion: 2005-01-04
• Study First Submitted: 2005-09-08
• Study First Submitted QC: 2005-09-08
• Study First Posted: 2005-09-12
• Status Verified: 2021-04
• Last Update Submitted: 2021-04-23
• Last Update Posted: 2021-04-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 50

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (12)

Children´s Hospital of Michigan; 🇺🇸 Detroit, Michigan, United States

University of Texas Health Science Center; 🇺🇸 Houston, Texas, United States

Emory University, Department of Pediatrics; 🇺🇸 Atlanta, Georgia, United States

Children´s Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Hospital for Sick Children, Division of Hematology/Oncology; 🇨🇦 Toronto, Ontario, Canada

Comprehensive Bleeding Disorders Center; 🇺🇸 Peoria, Illinois, United States

Indiana Hemophilia and Thrombosis Center; 🇺🇸 Indianapolis, Indiana, United States

University of Michigan Hemophilia Treatment Center; 🇺🇸 Ann Arbor, Michigan, United States

Children´s Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

University Pediatric Hospital; 🇵🇷 San Juan, Puerto Rico

Children´s Memorial Hospital; 🇺🇸 Chicago, Illinois, United States

University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics; 🇺🇸 Iowa City, Iowa, United States