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ACTIVE NOT RECRUITING
NCT06455059
PHASE3

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Sponsor: BioMarin Pharmaceutical

View on ClinicalTrials.gov

Summary

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia

Key Details

Gender

All

Age Range

3 Years - 17 Years

Study Type

INTERVENTIONAL

Enrollment

80

Start Date

2024-06-17

Completion Date

2026-08-01

Last Updated

2026-01-20

Healthy Volunteers

No

Interventions

DRUG

Vosoritide

Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.

DRUG

Placebo

Subcutaneous injection of recommended dose of placebo

Locations (23)

Nemours Alfred I. DuPont Hospital for Children

Wilmington, Delaware, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Murdoch Children's Research Institute

Parkville, Victoria, Australia

University of Alberta Stollery Children's Hospital

Edmonton, Alberta, Canada

SickKids - The Hospital for Sick Children

Toronto, Ontario, Canada

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, Canada

Hospices Civils de Lyon - Hopital Femme Mere Enfant

Lyon, France

Hopital de la Timone

Marseille, France

Hopital Necker-Enfants Malade

Paris, France

CHU de Toulouse

Toulouse, France

Uniklinik Koln

Cologne, Germany

Univeristatskinderklinik Magdeburg

Magdeburg, Germany

Instituto Giannina Gaslini

Genoa, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, Italy

Ospedale Pediatrico Bambino Gesu

Rome, Italy

Osaka University Hosptial

Osaka, Japan

Tokushima University Hospital

Tokushima, Japan

Institute of Science Tokyo Hospital

Tokyo, Japan

Tottori University Hospital

Tottori, Japan

Vithas Hospital San Jose

Vitoria-Gasteiz, Spain

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom