Long-Term Extension Study of Vosoritide to Treat Children with Hypochondroplasia
Research type
Research Study
Full title
A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children with Hypochondroplasia
IRAS ID
1011985
Contact name
Emily Graham
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
0000-000000-00
Research summary
Hypochondroplasia (HCH) is characterised by short stature, stocky build, disproportionately short arms and legs, broad, short hands and feet, mild joint laxity, scoliosis and macrocephaly. Biomarin developed vosoritide for the treatment of Achondroplasia (ACH). Vosoritide is a C-type natriuretic peptide (CNP) analog. CNP is found in the human body and is involved in bone growth regulation. Similar to ACH, HCH is also a rare form of genetic short stature and due to the common mechanism and phenotypic expression of disease (both conditions result in short stature and body disproportion), HCH and ACH are considered closely related. This study will allow for long-term assessment of safety and the effect of daily administration of vosoritide on Annualized Growth Velocity (AGV), standing height, and height Z-score in participants with Hypochondroplasia until they reach final adult height. This study is planned to involve approximately 140 participants who will receive Vosoritide (based on their weight) until they reach near final adult height. Participants are expected to visit the clinic on Study Day 1, Week 26, Week 52, and every 6 months thereafter until they reach near final adult height (NFAH, defined as evidence of growth plates closure at X-ray examination as per standard of care and AGV < 1.5 cm/year observed over a period of at least 6 month) and are at least 16 years of age for females and 18 years of age for males. Contact for study follow up will be conducted remotely via telephone. This study will collect data on medical history, concomitant medications, anthropometric measurements, Health-Related Quality of Life and functionality indicators, clinical data from blood samples, X-ray imaging, adverse events, and medical procedures. Participants will be given one single injection of medication daily. An approved caregiver will administer the study medication between clinic visits. The DMC will review and provide input on the safety data collected in the study.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
25/SC/0219
Date of REC Opinion
7 Aug 2025
REC opinion
Further Information Favourable Opinion