Overview

This trial is active, not recruiting.

Condition hemophilia a
Treatments moroctocog alfa ( af-cc), laboratory tests
Phase phase 4
Sponsor Pfizer
Start date December 2009
End date May 2016
Trial size 50 participants
Trial identifier NCT00914459, 2008-008435-29, 3082B2-4433, B1831005

Summary

The study will be investigating pharmacokinetics, safety and efficacy in patients less than 12 years of age with severe hemophilia A that have been previously treated with Factor VIII products ( including blood products).

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Masking open label
Primary purpose treatment
Arm
(Other)
Open Label
moroctocog alfa ( af-cc) ReFacto AF
Dosing is at the discretion of the Investigator
laboratory tests
Factor VIII PK samples, Hematology, Chemistry and Coagulation testing, FactorVIII Inhibitor and Anti Factor VIII antibody

Primary Outcomes

Measure
Incidence of clinically significant FVIII inhibitor development.
time frame: 2 years

Secondary Outcomes

Measure
Annualized bleeding rates (ABRs) in subjects receiving treatment with ReFacto AF.
time frame: 2 years
Responses to the first on-demand treatment with ReFacto AF for all new bleeds (4 point scale of assessment) as assessed by the parent/legal representative.
time frame: 2 years
Number of ReFacto AF infusions to treat each new bleed.
time frame: 2 years
Number of bleeds within 48 hours of a preventive/prophylaxis dose of ReFacto AF.
time frame: 2 years
Average infusion dose and total factor consumption, and the incidence of less-than-expected therapeutic effect (LETE).
time frame: 2 years
Incidence of SAEs and nonserious AEs.
time frame: 2 years

Eligibility Criteria

Male participants up to 11 years old.

Inclusion Criteria: - Male subjects less than 12 years of age with a documented history of severe hemophilia A (FVIII:C less than 1%). - Subjects who are less than 6 years of age must have had at least 50 Exposure Days (EDs) to prior FVIII products (including blood products). - Subjects who are equal to or greater than 6 years of age must have had greater than 150 EDs to prior FVIII products (including blood products). Exclusion Criteria: - For laboratory assessments, any measured Bethesda inhibitor titer equal to or greater than 0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer greater than ULN for the testing laboratory at the time of screening. - Any other bleeding disorder in addition to hemophilia A. - Treatment with any investigational drug or device within 30 days before the time of signing the parental informed consent/assent form. - Major surgery planned to occur during the course of the study. - 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). - Regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids), or currently receiving immune tolerance induction (ITI) for inhibitor treatment. - The subject is receiving treatment for HIV or hepatitis infection (unless the subject is on a stable antiviral regimen [i.e., consistent treatment regimen for at least 3 months before the parental informed consent/assent form is signed]). - Platelet count less than 100,000/µL. - Prothrombin time (PT) equal to or greater than 1.25 x ULN, or international normalized ratio (INR) equal to or greater than 1.5. - Known hypersensitivity to hamster protein.

Additional Information

Official title A Non-randomized, Open-label Study To Evaluate The Pharmacokinetics, Safety And Efficacy Of Refacto Af In Previously Treated Pediatric Subjects Less Than Twelve Years Of Age With Severe Hemophilia A (Fviii:c <1%).
Trial information was received from ClinicalTrials.gov and was last updated in February 2016.
Information provided to ClinicalTrials.gov by Pfizer.