Brief Title
Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
Official Title
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
Brief Summary
A prospective, non-controlled, international, multi-centre phase 3 study to investigate the pharmacokinetics, efficacy, safety, and immunogenicity of Wilate in previously treated children with severe haemophilia A
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C
Secondary Outcome
Total Annualized Bleeding Rate (TABR)
Condition
Severe Hemophilia A
Intervention
Wilate
Study Arms / Comparison Groups
All patients
Description: All patients will receive Wilate for prophylactic treatment. Patients will also receive Wilate for treatment of breakthrough bleeding events as required
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
11
Start Date
November 22, 2017
Completion Date
November 3, 2018
Primary Completion Date
November 3, 2018
Eligibility Criteria
Inclusion Criteria: 1. Severe haemophilia A (<1% FVIII:C) according to medical history 2. Male patients aged 1 to <12 years 3. Previous treatment with a FVIII concentrate for at least 50 exposure days (EDs) 4. Immunocompetence (CD4+ count >200/μL) 5. Voluntarily given, fully informed written and signed consent obtained by the patient's parent(s) or legal guardian and, depending on the children's developmental stage and intellectual capacity, informed assent by the patients before any study-related procedures are performed The interval between the Screening Visit and the 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). Exclusion Criteria: 1. Any coagulation disorders other than haemophilia A 2. History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory antibodies (≥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 antiretroviral chemotherapy), such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
Gender
Male
Ages
1 Year - 11 Years
Accepts Healthy Volunteers
No
Contacts
Cristina Solomon, MD, ,
Location Countries
Russian Federation
Location Countries
Russian Federation
Administrative Informations
NCT ID
NCT03376516
Organization ID
WIL-30
Responsible Party
Sponsor
Study Sponsor
Octapharma
Study Sponsor
Cristina Solomon, MD, Study Director, Octapharma
Verification Date
December 2020