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

April 23, 2021 updated by: Baxalta now part of Shire

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

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.

Study Overview

Status

Completed

Conditions

Hemophilia A

Intervention / Treatment

Drug: Antihemophilic factor, recombinant, manufactured protein-free

Study Type

Interventional

Enrollment

Phase

Phase 2
Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 6 years (Child)

Accepts Healthy Volunteers

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Interventional Model: Single Group Assignment
Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM) Time Frame: 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.	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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Baxalta now part of Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 7, 2002

Primary Completion (Actual)

January 4, 2005

Study Completion (Actual)

January 4, 2005

Study Registration Dates

First Submitted

September 8, 2005

First Submitted That Met QC Criteria