Phase 3 study to evaluate the Safety and Efficacy of Vosoritide in Children with Hypochondroplasia

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children with Hypochondroplasia

• IRAS ID

  1009842

• Contact name

  Tabitha Santoso

• Contact email

  Tabitha.santoso@bmrn.com

• Sponsor organisation

  BioMarin Pharmaceutical, Inc.

• Clinicaltrials.gov Identifier

  NCT06455059

• 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 (111-303) will allow for long-term assessment of the effect of daily administration of vosoritide on Annualized Growth Velocity (AGV), standing height, and height Z-score in participants with Hypochondroplasia.

  This study is planned to involve approximately 80 participants who will receive Vosoritide (based on their weight) or placebo daily by subcutaneous (SC) injection for 52 weeks. Participants are expected to visit the clinic on Study Day 1, Day 2 and approximately every 3 months thereafter. The other visits 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.

  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

  London - Brighton & Sussex Research Ethics Committee

• REC reference

  24/LO/0684

• Date of REC Opinion

  22 Oct 2024

• REC opinion

  Further Information Favourable Opinion