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RECRUITING
NCT06998524
PHASE3

A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease

Sponsor: Hoffmann-La Roche

View on ClinicalTrials.gov

Summary

This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD). Participants on prior standard of care (SOC) on-demand therapy will be assessed via a randomized comparison (Arm A - emicizumab prophylaxis and Arm B - continuation of SOC on-demand therapy), while participants on prior SOC prophylactic therapy (Arm C - emicizumab prophylaxis) will be assessed via intra-participant analysis with data obtained from the preceding non-interventional study (NIS), WP45335 (NCT06883240).

Official title: A Phase III, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Prophylaxis in Patients With Type 3 Von Willebrand Disease

Key Details

Gender

All

Age Range

1 Month - Any

Study Type

INTERVENTIONAL

Enrollment

75

Start Date

2025-06-27

Completion Date

2029-03-30

Last Updated

2026-03-19

Healthy Volunteers

No

Interventions

DRUG

Emicizumab

Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W). During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.

DRUG

von Willebrand Factor (VWF) Concentrates

Used according to local labeling or local treatment guidelines.

DRUG

Factor VIII (FVIII) Concentrates

Used according to local labeling or local treatment guidelines.

DRUG

von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates

Used according to local labeling or local treatment guidelines.

DRUG

Bypassing Agents

Used according to local labeling or local treatment guidelines.

Locations (27)

UC Davis

Sacramento, California, United States

University of Florida

Gainesville, Florida, United States

University of Minnesota Medical Center

Minneapolis, Minnesota, United States

Washington University School of Medicine

St Louis, Missouri, United States

UZ Leuven Gasthuisberg

Leuven, Belgium

The Hospital for Sick Children

Toronto, Ontario, Canada

McGill University Health Center

Montreal, Quebec, Canada

IPS SURA Industriales Medellín

Medellín, Colombia

Hopital Claude Huriez - CHU Lille

Lille, France

Groupe Hospitalier Necker Enfants Malades

Paris, France

Universitätsklinikum Bonn

Bonn, Germany

Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

Duisburg, Germany

Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin

Frankfurt/M., Germany

Universita' Degli Studi La Sapienza-Ist.Di Ematologia

Rome, Lazio, Italy

IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, Lombardy, Italy

AOU Careggi

Florence, Tuscany, Italy

Kurume University Hospital

Fukuoka, Japan

Nagoya University Hospital

Nagoya, Japan

Erasmus MC

Rotterdam, Netherlands

Instytut Hematologii i Transfuzjologii

Warsaw, Poland

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, South Africa

Hospital Universitario la Paz

Madrid, Spain

Hospital Universitario Virgen del Rocio

Seville, Spain

Sahlgrenska Universitetssjukhuset

Gothenburg, Sweden

St Thomas' Hospital

London, United Kingdom

Great Ormond Street Hospital

London, United Kingdom

Manchester Royal Infirmary

Manchester, United Kingdom