A Study of Vosoritide Versus Placebo in Children with Hypochondroplasia Aged 0 to < 36 Months
Research type
Research Study
Full title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children with Hypochondroplasia, Aged 0 to < 36 Months
IRAS ID
1012089
Contact name
Emily Graham
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
0000-000000-00
Research summary
Updated after REC recommendation
9. Lay Summary of the study
Hypochondroplasia (HCH) is a genetic condition that causes short stature, stocky build, disproportionately short arms and legs, broad, short hands and feet, mild joint laxity, scoliosis and macrocephaly. There is no cure for this condition but there are some treatments available to help with this condition. Biomarin (the sponsor) developed vosoritide for the treatment of Achondroplasia (ACH). Similar to HCH, ACH is also a rare form of genetic short stature, and so both of these conditions are considered as closely related. Vosoritide is a type of hormone involved in bone growth regulation. This study is investigating the effects of Vosoritide in children with HCH.
This is a Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children with Hypochondroplasia, Aged 0 to < 36 Months.
This study is planned to involve approximately 60 participants with a genetically diagnosed HCH. Participants will receive the study treatment daily for 52 weeks by subcutaneous injections. The dose of the study medication will be based on participant’s weight. Study participants will visit the study site at the time points specified in the parental/guardian information sheet. Other visits will be conducted remotely via telephone. This study will collect data on medical history, concomitant medications, physical examinations, Health-Related Quality of Life and functionality indicators, clinical data from blood samples, X-ray imaging, adverse events, and medical procedures.
The participants will be given one single injection of medication daily. An approved caregiver will administer the study medication between clinic visits.The Data Monitoring Committee (DMC) will review and provide input on the safety data collected in the study. The Data Monitoring Committee data review will occur every 6 months or as needed.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
25/YH/0155
Date of REC Opinion
3 Oct 2025
REC opinion
Further Information Favourable Opinion