A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

PHASE2RecruitingINTERVENTIONAL
Enrollment

60

Participants

Timeline

Start Date

July 30, 2025

Primary Completion Date

June 30, 2028

Study Completion Date

June 30, 2028

Conditions
Hypochondroplasia
Interventions
DRUG

Vosoritide

The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH

DRUG

Placebo

Subcutaneous injection of recommended dose of placebo

Trial Locations (19)

3052

NOT_YET_RECRUITING

Royal Children's Hospital Melbourne, Parkville

16147

NOT_YET_RECRUITING

IRCCS Istituto Giannina Gaslini, Genova

20010

RECRUITING

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

21205

NOT_YET_RECRUITING

The Johns Hopkins University School of Medicine, Baltimore

39120

NOT_YET_RECRUITING

Universitätskinderklinik Magdeburg, Magdeburg

50937

NOT_YET_RECRUITING

Uniklinik Köln, Cologne

54956

NOT_YET_RECRUITING

Children's Wisconsin - Fox Valley Hospital, Neenah

55454

NOT_YET_RECRUITING

University of Minneasota Masonic Children's Hospital, Minneapolis

60611

NOT_YET_RECRUITING

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

65211

NOT_YET_RECRUITING

University of Missouri, Columbia

66421

NOT_YET_RECRUITING

Universitätsklinikum des Saarlandes, Homburg

69500

NOT_YET_RECRUITING

Hospices Civils de Lyon - Hôpital Femme Mère Enfant, Bron

85016

NOT_YET_RECRUITING

Phoenix Children's Hospital - Thomas Campus (Main), Phoenix

90048

NOT_YET_RECRUITING

Cedars-Sinai Medical Center, Los Angeles

94270

NOT_YET_RECRUITING

Hôpital Bicêtre, Le Kremlin-Bicêtre

94609

NOT_YET_RECRUITING

Benioff Children's Hospital - Oakland, Oakland

00168

NOT_YET_RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Roma

W1W 5DT

NOT_YET_RECRUITING

Myriad Trials, London

WC1N3BH

NOT_YET_RECRUITING

Great Ormond Street Hospital, London

Sponsors
All Listed Sponsors
collaborator

ICON Clinical Research

INDUSTRY

lead

BioMarin Pharmaceutical

INDUSTRY