Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

PHASE3Enrolling by invitationINTERVENTIONAL
Enrollment

140

Participants

Timeline

Start Date

June 20, 2025

Primary Completion Date

December 31, 2040

Study Completion Date

December 31, 2040

Conditions
Hypochondroplasia
Interventions
DRUG

Vosoritide

Open-label administration of vosoritide using weight-band dosing

Trial Locations (23)

3052

Murdoch Children's Research Institute, Parkville

19803

Nemours Children's Hospital, Delaware (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