The main purpose of this study is to gather information regarding the safety of ReFacto AF, and how well it works (effectiveness) in children with severe Hemophilia A under the age of 12. Approximately 50 subjects, will take part in this study. 25 subjects will be 0-6 years of age and 25 subjects will be 6 to 12 years of age.

Phase 1

• Conditions: Severe hemophilia A (FVIII:C <1%) in male subjects <12 years of age.; MedDRA version: 17.0Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2008-008435-29-BG
• Lead Sponsor: Wyeth Pharmaceuticals Inc. Acting through its division Wyeth Research, a Pfizer Company

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-06-24
• Last Update Posted: 29 January 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 50

Inclusion Criteria

1) Male subjects <12 years of age with a documented history of severe hemophilia A (FVIII:C <1%).
a) Subjects who are <6 years of age must have had at least 50 exposure days to prior FVIII products (including blood products).
b) Subjects who are =6 years of age must have had greater than 150 exposure days to prior FVIII products (including blood products)

Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1) For laboratory assessments, any measured Bethesda inhibitor titer =0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer > ULN for the testing laboratory at the time of screening.
2) Any other bleeding disorder in addition to hemophilia A.
3) Treatment with any investigational drug or device within 30 days before the time of signing the parental informed consent/assent form.
4) Major surgery planned to occur during the course of the study.
5) Regular (e.g., daily; every other day) use of agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDS).
6) Regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids), or currently receiving immune tolerance induction (ITI) for inhibitor treatment.
7) The subject is receiving treatment for HIV or hepatitis infection (exceptions may be made for subjects on a stable antiviral regimen [i.e., consistent treatment regimen for at least 3 months before signing the parental informed consent/assent form]).
8) Platelet count <100,000/µL.
9) Prothrombin time (PT) =1.25 x ULN, or international normalized ratio (INR) =1.5.
10) Known hypersensitivity to hamster protein.
11) Any condition(s) that compromises the subject’s ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation (these conditions include, but are not limited to, inadequate medical history available to assure study eligibility; inability to properly store study medication; expectation of poor compliance in study related documentation; poor venous access), in the opinion of the investigator.
12) Unwilling or unable to follow the terms of the protocol.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified