Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

80

Participants

Timeline

Start Date

June 6, 2024

Primary Completion Date

August 1, 2026

Study Completion Date

August 1, 2026

Conditions
Hypochondroplasia
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

Trial Locations (23)

3052

Murdoch Children's Research Institute, Parkville

19803

Nemours Alfred I. DuPont Hospital for Children, Wilmington

20010

Children's National Medical Center, Washington D.C.

53226

Medical College of Wisconsin, Milwaukee

60611

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

Unknown

University of Alberta Stollery Children's Hospital, Edmonton

SickKids - The Hospital for Sick Children, Toronto

Centre Hospitalier Universitaire Sainte-Justine, Montreal

Hospices Civils de Lyon - Hopital Femme Mere Enfant, Lyon

Hopital de la Timone, Marseille

Hopital Necker-Enfants Malade, Paris

CHU de Toulouse, Toulouse

Uniklinik Koln, Cologne

Univeristatskinderklinik Magdeburg, Magdeburg

Instituto Giannina Gaslini, Genoa

Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome

Ospedale Pediatrico Bambino Gesu, Rome

Osaka University Hosptial, Osaka

Tokushima University Hospital, Tokushima

Institute of Science Tokyo Hospital, Tokyo

Tottori University Hospital, Tottori

Vithas Hospital San Jose, Vitoria-Gasteiz

Great Ormond Street Hospital for Children NHS Foundation Trust, London

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY