Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease VWD) Patients <6 Years of Age

NCT ID: NCT04953884

Last Updated: 2024-12-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-07-28
• Study Completion Date: 2024-12-18

Brief Summary

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.

Conditions

Von Willebrand Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

wilate treatment

PK: Single dose of 80 IU/kg body weight (BW). Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at the recommended dose of over 12 months. Minor haemorrhage: loading dose 30-50 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours to achieve von Willebrand factor activity (VWF:Ac) and FVIII:C trough levels of \>30%. Major haemorrhage: loading dose 50-80 IU/kg BW followed by a maintenance dose of 30-50 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of \>50%. Minor surgery: loading dose of 40-60 IU/kg BW followed by a maintenance dose of 20-30 IU/kg BW every 12- 24 hours for up to 3 days, to achieve VWF:Ac peak levels of 50% after loading dose and trough levels \>30% during maintenance. Major surgery: loading dose of 60-80 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours for up to 6 days or longer, to achieve VWF:Ac peak levels of 100% after loading dose and trough levels \>50% during maintenance

Group Type: EXPERIMENTAL

wilate

Intervention Type: DRUG

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

Interventions

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

Intervention Type: DRUG

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

• Maximum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Octapharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Tulane University

New Orleans, Louisiana, United States

University Hospital Ostrava Department for Pediatric Medicine

Ostrava, , Czechia

University Hospital Motol, Department of Paediatric Haematology and Oncology

Prague, , Czechia

Gerinnungszentrum Rhein-Ruhr Ambulanz und Fachlaboratorium für Gerinnungserkrankungen/Hämophilie

Duisburg, , Germany

IMSP Mother and Child Institute

Chisinau, , Moldova

PHI University Clinic for Child Diseases

Skopje, , North Macedonia

FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology

Moscow, , Russia

Morozovskaya Children's Hospital

Moscow, , Russia

Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre"

Lviv, , Ukraine

Countries

Belarus; United States; Czechia; Germany; Moldova; North Macedonia; Russia; Ukraine

Other Identifiers

WIL-33

Identifier Type: -

Identifier Source: org_study_id