Clinical trial

Clinical Study to Investigate the Efficacy, Pharmacokinetics, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease Patients Under the Age of 6 Years

Name

WIL-33

Description

The WIL-33 study aims to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as screening von Willebrand factor ristocetin cofactor activity \[VWF:RCo\] \<20%) under the age of 6 years, over a period of 12 months.

Trial arms

Trial start

2021-07-28

Estimated PCD

2024-12-01

Trial end

2024-12-01

Status

Active (not recruiting)

Phase

Early phase I

Treatment

wilate

wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with von Willebrand disease (VWD) and/or haemophilia A

Arms:

wilate treatment

Size

Primary endpoint

Total annualised bleeding rate (tABR) during prophylactic treatment with wilate.

Up to 12 months of treatment

Eligibility criteria

Inclusion Criteria: 1. Patients aged \<6 years at the time of screening 2. Type 3 (at least four patients), severe type 2 (except 2N) or severe type 1 VWD (any of which with VWF:RCo \<20%) according to medical history, requiring substitution therapy with a VWF-containing product 3. Minimum body weight 12.5 kg at the time of screening 4. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patient's parent(s)/ legal guardian(s)) Exclusion Criteria: 1. History or current suspicion of VWF or FVIII inhibitors 2. Injection of DDAVP or VWF-containing product within 72 hours prior to inclusion 3. Medical history of a thromboembolic event 4. Platelet count \<100,000/µL at screening (except for VWD type 2B) 5. Patients receiving, or scheduled to receive, immunosuppressant drugs (other than antiretroviral chemotherapy), such as prednisone (equivalent to \>10 mg/day), or similar drugs 6. Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within four weeks before enrolment 7. Other coagulation disorders or bleeding disorders 8. Known hypersensitivity to any of the components of the study drug

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ACTUAL'}}

Updated at

2024-03-12

1 organization

1 product

1 indication

Organization

Octapharma

Product

wilate

Indication

von Willebrand disease