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

Primary Purpose

Von Willebrand Disease

Status

Recruiting

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

wilate

About this trial

This is an interventional treatment trial for Von Willebrand Disease

Eligibility Criteria

undefined - 5 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patients aged <6 years at the time of screening
Type 3 (at least four patients), severe type 2 (except 2N) or severe type 1 VWD (VWF:RCo <20%) according to medical history, requiring substitution therapy with a VWF-containing product
Minimum body weight 12.5 kg at the time of screening
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:

History or current suspicion of VWF or FVIII inhibitors
Injection of DDAVP or VWF-containing product within 72 hours prior to inclusion
Medical history of a thromboembolic event
Platelet count <100,000/µL at screening (except for VWD type 2B)
Patients receiving, or scheduled to receive, immunosuppressant drugs (other than antiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs
Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within four weeks before enrolment
Other coagulation disorders or bleeding disorders
Known hypersensitivity to any of the components of the study drug

Sites / Locations

Loma Linda University HealthRecruiting
Tulane UniversityRecruiting
Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology
University Hospital Ostrava Department for Pediatric Medicine
University Hospital Motol, Department of Paediatric Haematology and OncologyRecruiting
Universitätsklinikum Bonn Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT)
Gerinnungszentrum Rhein-Ruhr Ambulanz und Fachlaboratorium für Gerinnungserkrankungen/HämophilieRecruiting
IMSP Mother and Child Institute
PHI University Clinic for Child Diseases
FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology
Morozovskaya Children's Hospital
State Institution National Children's Specialized Clinic "OHMATDET" of Ministry of Health of Ukraine, Centre of pathology of hemostasis
Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre"Recruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

wilate treatment

Arm Description

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Area under the curve (AUC) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

AUC normalised for the administered dose (AUCnorm) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

In vivo half-life (T1/2) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

Maximum plasma concentration (Cmax) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

Time to reach maximum plasma concentration (Tmax) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

Mean residence time (MRT) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

Volume of distribution (Vd) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

Clearance (CL) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

Incremental in-vivo recovery (IVR) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

Efficacy of wilate in the prevention and treatment of spontaneous and traumatic breakthrough BEs based on their rate and the proportion of spontaneous and traumatic BEs successfully treated with wilate

Assessed by the use of a 4-point ordinal haemostatic efficacy scale (excellent - good - moderate - none)

The overall efficacy of wilate in perioperative prophylaxis against excessive bleeding as assessed at the end of the postoperative period by the responsible treating investigator

Assessed by the use of a 4-point ordinal haemostatic efficacy scale (excellent - good - moderate - none)

wilate consumption data for prophylactic treatment, for on-demand treatment and during surgical prophylaxis

Incidence of VWF and FVIII inhibitors

Incidence of thromboembolic events

Joint Health Status determination by the use of the Hemophilia Joint Health Score, given the patient's age and constitutional development allow this assessment

Safety and tolerability of wilate assessed by monitoring adverse events (AEs) throughout the study

Full Information

NCT ID

NCT04953884

First Posted

June 18, 2021

Last Updated

August 23, 2023

Sponsor

Octapharma

1. Study Identification

Unique Protocol Identification Number

NCT04953884

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2023

Overall Recruitment Status

Recruiting

Study Start Date

July 28, 2021 (Actual)

Primary Completion Date

October 2023 (Anticipated)

Study Completion Date

October 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Octapharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Von Willebrand Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

wilate treatment

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

wilate

Intervention Description

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

Primary Outcome Measure Information:

Title

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

Time Frame

Up to 12 months of treatment

Secondary Outcome Measure Information:

Title

Area under the curve (AUC) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time

Time Frame