Brief Title
Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
Official Title
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A
Brief 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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Total Annualized Bleeding Rate (TABR)
Secondary Outcome
Spontaneous Annualized Bleeding Rate (SABR)
Condition
Severe Hemophilia A
Intervention
Wilate
Study Arms / Comparison Groups
All patients
Description: All patients will receive Wilate for prophylactic treatment
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
57
Start Date
December 2016
Completion Date
March 29, 2018
Primary Completion Date
March 29, 2018
Eligibility 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
Gender
Male
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Cristina Solomon, MD, ,
Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT02954575
Organization ID
WIL-27
Responsible Party
Sponsor
Study Sponsor
Octapharma
Study Sponsor
Cristina Solomon, MD, Study Director, Octapharma
Verification Date
December 2020