Status:Not yet recruiting
Keywords:Prophylaxis
, Hemophilia
, Children
IRB Number:00050690
Specialty:Pediatric Hematology and Oncology
Sub Specialty:Hemophilia
Primary Objective
- To demonstrate that moroctocog alfa (AF-CC) prophylaxis reduces annualized bleeding rates relative to on-demand therapy.
Secondary Objectives
- To assess the effect of a high- versus low-frequency dosing schedule on the efficacy of moroctocog alfa (AF-CC) prophylaxis.
- To characterize the pharmacokinetics of moroctocog alfa (AF-CC) in children younger than 6 years of age with hemophilia A.
- To describe moroctocog alfa (AF-CC) efficacy and safety in children, including
characterization of the incidence of “less than expected therapeutic effect”.
Principle Investigator: Hassan Yaish
Principle Department: Pediatric Administration
Co Investigator:
Name:Jeanette Davis
Phone:801-587-7525
Email:jeanette.davis@hsc.utah.edu
1. Male subjects with moderately severe to severe hemophilia A (FVIII:C ≤ 2%) by both the local laboratory and the central laboratory at screening.
2. A negative FVIII inhibitor by both the local laboratory and the central laboratory at screening (for local laboratory, a Bethesda inhibitor titer less than the upper limit of normal [ULN] for the laboratory performing the assay and is not reported to be ≥ 0.6 Bethesda Units [BU]/mL; for central laboratory, Bethesda inhibitor titer < 0.6 BU/mL by Nijmegen assay).
3. A medical history negative for a past FVIII inhibitor (see Protocol Definitions).
4. Age < 6 years at time of screening visit (study visit 1).
5. Previous experience of FVIII therapy (≥ 20 exposure days to any FVIII replacement product).
6. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 5.0 × ULN, and bilirubin ≤ 2 mg/dL (< 35 μmol/L).
7. Serum albumin ≥ the lower limit of normal (LLN).
8. Serum creatinine ≤ 1.25 × ULN.
9. Platelet count ≥ 100,000/μL.
10. Absolute CD4 count > 400/μL.
11. Prothrombin time (PT) ≤ 1.25 × ULN, or international normalized ratio (INR) ≤ 1.5
12. The subject is not receiving treatment for HIV or hepatitis infection, or the subject is on a stable antiviral regimen at the time of signing the consent/assent form (i.e., stable dosing for at least 3 months before signing the consent/assent form).
13. The subject is able to comply with the mandatory 72-hour FVIII washout preceding each FVIII:C and FVIII inhibitor assessment during the study.
Additional criteria for subjects participating in the PK:
1. Male subjects as described immediately above except they must have a FVIII:C of 1% confirmed by the central laboratory screening test
2. Age < 6 years at time of PK assessment (study visit 2).
3. The subject’s size is sufficient to permit PK-related phlebotomy.
4. The subject is able to comply with the procedures conducted during the PK assessment, including a mandatory 72-hour washout period preceding the PK assessments.
1. A history of FVIII inhibitor (clinical or laboratory-based assessment). For laboratory assessments, any measured Bethesda inhibitor titer ≥ 0.6 BU/mL, regardless of the laboratory normal range, or any measured Bethesda inhibitor titer greater than the ULN for the laboratory performing the assay.
2. Presence of a bleeding disorder in addition to hemophilia A.
3. Treatment with any investigational drug or device within 30 days before the time of signing the consent/assent form.
4. Major or orthopedic surgery planned to occur within the period following the signing of the consent/assent form to the Final Study Contact (ie, during the course of the study).
5. Regular (eg, daily, every other day) use of antifibrinolytic agents or medications known to influence platelet function such as aspirin or certain nonsteroidal antiinflammatory drugs (NSAIDs).
6. Regular, concomitant therapy with immunomodulating drugs (eg, intravenous immunoglobulin [IVIG], routine systemic corticosteroids).
7. Known hypersensitivity to hamster protein.
8. 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 to assure study eligibility; inability to properly store study drug; expectation of poor compliance in study related documentation).
9. Unwilling or unable to follow the terms of the protocol.
