A Study to Assess Children with Hypochondroplasia
Research type
Research Study
Full title
A Multicenter Multinational Observational Study of Children with Hypochondroplasia,
IRAS ID
339748
Contact name
Melita Irving
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 4 months, 0 days
Research summary
The purpose of this clinical research study is to to learn more about how participants with hypochondroplasia grow and to collect prospective and retrospective serial anthropometric measurements in younger participants with Hypochondroplasia (HCH).
This study is planned to enroll approximately 400 participants worldwide with HCH.
No investigational product will be administered to participants in this study.
Enrolled participants may be considered for enrolment in future studies sponsored by BioMarin.
After a 5-week Screening period, the maximum study duration for enrolled participants is defined by achievement of the final adult height at the ages of 16 years for females or 18 years for males.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
24/SC/0111
Date of REC Opinion
2 May 2024
REC opinion
Further Information Favourable Opinion