Study to Investigate the product Wilate in Patients with Severe Hemophilia A

Phase 1

• Conditions: Severe hemophilia A (<1% FVIII:C); MedDRA version: 20.0Level: LLTClassification code 10060613Term: Hemophilia A (Factor VIII)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-003681-34-PL
• Lead Sponsor: Octapharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-11-08
• Last Update Posted: 15 October 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 55

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 pre-ceding study start)
6. Voluntarily given, fully informed written and signed consent obtained by the pa-tient (or parent/legal guardian in case of adolescents) before any study-related pro-cedures 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., inclusion criterion no. 4).
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified