Overview

This trial is active, not recruiting.

Condition hemophilia a
Treatments moroctocog alfa (af-cc)
Phase phase 3
Sponsor Pfizer
Start date December 2007
End date April 2018
Trial size 80 participants
Trial identifier NCT00543439, 2006-005575-17, 3082B2-313, B1831001

Summary

The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately severe hemophilia A.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
On-Demand therapy for 6 months, followed by Routine Prophylaxis treatment for 1 year.
moroctocog alfa (af-cc) Xyntha
On-demand therapy for 6 months, followed by routine prophylaxis 25 IU/kg, administered every other day for 1 year.
(Experimental)
Routine Prophylaxis Crossover
moroctocog alfa (af-cc) Xyntha
Routine prophylaxis crossover: 45 IU/kg, administered 2 times a week for 1 year followed by 25 IU/kg administered every other day for 1 year, or, 25 IU/kg, administered every other day for 1 year, followed by 45 IU/kg, administered 2 times a week for 1 year.

Primary Outcomes

Measure
The annualized bleed rate is the primary endpoint for testing the primary objective of the study (comparing prophylaxis to on-demand therapy)
time frame: End of Study

Secondary Outcomes

Measure
The annualized bleed rate is the endpoint for testing one of the secondary objectives (comparing high vs low frequency prophylaxis regimens).
time frame: End of Study
Number of moroctocog alfa (AF-CC) infusions per bleed
time frame: End of Study
Response of bleed to moroctocog alfa (AF-CC) treatment (4-point scale of assessment)
time frame: End of Study
Time interval between bleed onset and prior moroctocog alfa (AF-CC) prophylaxis dose
time frame: End of Study
Incidence of prophylaxis regimen escalation
time frame: End of Study
Incidence of Less than Expected Therapeutic Effect
time frame: End of Study
Consumption of moroctocog alfa (AF-CC)
time frame: End of Study
Compliance with assigned prophylaxis regimen
time frame: End of Study
Incidence of Adverse Events
time frame: End of Study
Incidence of confirmed FVIII inhibitor development
time frame: End of Study

Eligibility Criteria

Male participants from 6 months up to 15 years old.

Inclusion Criteria: - Male subjects, aged less than 6 years, with moderately severe to severe hemophilia A. - A negative FVIII inhibitor titer at screening, and a medical history negative for a past FVIII inhibitor. - At least 20 exposure days to any FVIII replacement product. - Adequate hepatic and renal function - CD4 count > 400 cells/uL, and if receiving antiviral therapy must be on a stable regimen Additional criteria for subjects participating in the PK assessment: - Male subjects as described immediately above except they must have a FVIII Activity of less than or equal to 1% confirmed by the central laboratory screening test - Age < 6 years at time of PK assessment. - The subject's size is sufficient to permit PK-related phlebotomy. - The subject is able to comply with the procedures conducted during the PK assessment, including a mandatory 72-hour washout period preceding the PK assessment. Exclusion Criteria: - A history of FVIII inhibitor. - Presence of a bleeding disorder in addition to hemophilia A. - Treatment with any investigational drug or device within 30 days before the time of signing the informed consent form. - Major or orthopedic surgery planned to occur during the course of the study. - Regular (e.g., daily, every other day) use of antifibrinolytic agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDs), or regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids). - Known hypersensitivity to hamster protein.

Additional Information

Official title An Open-label Study To Evaluate Prophylaxis Treatment, And To Characterize The Efficacy, Safety, And Pharmacokinetics Of B-domain Deleted Recombinant Factor Viii Albumin Free (Moroctocog Alfa [Af-cc]) In Children With Hemophilia A
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Pfizer.