Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A - A Continuation of Baxter Study 060101

NCT00189982 | Completed Phase 2

• 1 other identifier: 060401
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101.

Conditions

Hemophilia A

Keywords

Factor VIII Deficiency

Outcome Measures

Primary Outcomes (1)

• Assessment of safety, as measured by the incidence, causality, and severity of adverse experiences

  Throughout the study period of approximately 23 months.

Secondary Outcomes (2)

• Assessment of the hemostatic efficacy in the treatment of bleeding episodes;

  At least 50 exposure days or a total treatment time of 6 months, whichever came first.

• assessment of the hemostatic efficacy in surgical or invasive procedures

  From day of surgery until time of discharge from hospital or clinic (up to approximately 2 weeks post surgery).

Interventions

Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM) DRUG

Treatment regimens were determined by the investigator, and may have been any combination of standard prophylaxis (25 to 50 IU/kg body weight, 3 to 4 times per week), investigator-determined prophylaxis, and/or on-demand treatment (dose selected by investigator). Once the treatment regimen was established, the regimen was to be maintained for the duration of the study, unless clinical reasons necessitated a change. The treatment of bleeding episodes and perioperative management was at the discretion of the investigator and consistent with the institution's standard of care.

Eligibility Criteria

• Age: Up to 6 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subject must have participated and completed participation in Baxter's clinical study 060101
• Subject or parent/legally authorized representative has provided written informed consent

You may not qualify if:

• Subjects who have withdrawn from Baxter's Clinical Study 060101 prior to the termination of the study

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (1)

Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 8, 2005
• First Posted: September 19, 2005
• Study Start: December 17, 2004
• Primary Completion: November 10, 2006
• Study Completion: November 10, 2006
• Last Updated: May 5, 2021

Record last verified: 2021-05

Locations

CA (1)