Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A

• Status: Completed
• Trial end date: 2018-03-29
• Target enrollment: 0
• Participant gender: Male

Summary

The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Octapharma
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

1. Severe hemophilia A (<1% FVIII:C) according to medical history

2. Male patients aged ≥12 years

3. Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)

4. Immunocompetence (CD4+ count >200/µL)

5. Good documentation of the historical bleeding rate (at least for the 6 months preceding study start)

6. Voluntarily given, fully informed written and signed consent obtained by the patient (or parent/legal guardian in case of adolescents) before any study-related procedures are conducted

Whenever possible, the interval between the Screening Visit and the PK or Non-PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/µL for patients to be enrolled (i.e., exclusion criterion no. 4).

Exclusion Criteria:

1. Any coagulation disorders other than hemophilia A

2. History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory anti-bodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory

3. Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)

4. Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs

5. Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment