Vosoritide for Hypochondroplasia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety and effectiveness of vosoritide, an injectable medication, for children with hypochondroplasia (HCH), a genetic condition that causes short stature. The trial will include two groups: one receiving vosoritide and the other a placebo (a non-active treatment) to compare results over a year. Children under 36 months with confirmed HCH and specific growth challenges are suitable candidates for this trial. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to early understanding of its impact.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it does mention that certain medications are prohibited. It's best to discuss your current medications with the trial team to see if they are allowed.
Is there any evidence suggesting that vosoritide is likely to be safe for humans?
A previous study found that vosoritide was generally well-tolerated by children with hypochondroplasia, meaning it didn't cause unexpected problems. Researchers identified no new major safety issues. Another report indicated that the safety profile of vosoritide matched what is already known from its use in treating achondroplasia, a related condition. This means the side effects were expected and manageable. This information suggests that vosoritide is safe for use in humans, particularly in children with certain growth conditions.12345
Why do researchers think this study treatment might be promising for hypochondroplasia?
Vosoritide is unique because it directly targets the root cause of hypochondroplasia by acting as a C-type natriuretic peptide (CNP) analog. Unlike standard treatments that primarily focus on managing symptoms, vosoritide works by promoting bone growth at the growth plates, potentially addressing the underlying growth deficiency. Researchers are excited about vosoritide because it offers a new approach that could improve growth outcomes and overall quality of life for individuals with hypochondroplasia.
What evidence suggests that vosoritide might be an effective treatment for hypochondroplasia?
Research has shown that vosoritide, which participants in this trial may receive, can help children with hypochondroplasia grow faster. In studies, children who received vosoritide grew more than those who did not receive the treatment. Vosoritide also reduced the outward curve of the shin bone, a common issue in some bone conditions. Real-world data supports that children treated with vosoritide for 24 months experienced significant height increases. These findings suggest that vosoritide can positively impact growth and bone development in children with hypochondroplasia.12678
Are You a Good Fit for This Trial?
This trial is for very young children, from birth up to less than 3 years old, who have been diagnosed with Hypochondroplasia (HCH), a genetic bone growth disorder. Specific eligibility details are not provided, but typically participants must meet certain health criteria and not be involved in other studies.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive daily subcutaneous injections of vosoritide or placebo for 52 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Vosoritide
Find a Clinic Near You
Who Is Running the Clinical Trial?
BioMarin Pharmaceutical
Lead Sponsor
Alexander Hardy
BioMarin Pharmaceutical
Chief Executive Officer since 2023
MBA from INSEAD
Greg Friberg
BioMarin Pharmaceutical
Chief Medical Officer
MD from New York Medical College
ICON Clinical Research
Industry Sponsor