Status:
COMPLETED
Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
Lead Sponsor:
Octapharma
Conditions:
Severe Hemophilia A
Eligibility:
MALE
1-11 years
Phase:
PHASE3
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 ...
Eligibility Criteria
Inclusion
- Severe haemophilia A (\<1% FVIII:C) according to medical history
- Male patients aged 1 to \<12 years
- Previous treatment with a FVIII concentrate for at least 50 exposure days (EDs)
- Immunocompetence (CD4+ count \>200/μL)
- 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
- Any coagulation disorders other than haemophilia A
- 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
- Severe liver or kidney diseases (alanine aminotransferase \[ALAT\] and aspartate transaminase \[ASAT\] levels \>5 times of upper limit of normal, creatinine \>120 μmol/L)
- 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
Key Trial Info
Start Date :
November 22 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 3 2018
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT03376516
Start Date
November 22 2017
End Date
November 3 2018
Last Update
January 19 2021
Active Locations (3)
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1
Kirov SSC Hematology and Transfusiology
Kirov, Russia
2
"National Children's Specialized Clinic "OKHMATDYT"
Kyiv, Ukraine
3
"Western Ukrainian Specialized Children's Medical Center"
Lviv, Ukraine