Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII

Pfizer

Status and phase: Completed; Phase 3

Conditions: Hemophilia A

Treatments: Biological: Moroctocog alfa (AF-CC)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00543439

3082B2-313

B1831001 (Other Identifier)

2006-005575-17 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 66 patients

Sex: Male

Ages: 6 months to 15 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

---

66 participants in 2 patient groups

1

Experimental group

Description:

On-Demand therapy for 6 months, followed by Routine Prophylaxis treatment for 1 year.

Treatment:

Biological: Moroctocog alfa (AF-CC)

Biological: Moroctocog alfa (AF-CC)

2

Experimental group

Description:

Routine Prophylaxis Crossover

Treatment:

Biological: Moroctocog alfa (AF-CC)

Biological: Moroctocog alfa (AF-CC)