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Growth Hormone Analog

Vosoritide for Dwarfism

Phase 3
Waitlist Available
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participants must be ≥ 3 to < 18 years of age at enrollment
A confirmed genetic diagnosis of HCH
Must not have
Have an unstable condition likely to require surgical intervention during the study
Evidence of decreased growth velocity and/or growth plate closure
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial

Summary

"This trial aims to test if vosoritide can be used as a treatment for children with hypochondroplasia."

Who is the study for?
This trial is for children with hypochondroplasia, a form of short-limbed dwarfism. Participants should have conditions like dwarfism, lordosis (curved lower back), or congenital limb deformities related to HCH.
What is being tested?
The study is testing Vosoritide's effectiveness compared to a placebo in treating children with HCH. It's a Phase 3 trial, which means it's looking at how well the drug works and monitoring its safety.
What are the potential side effects?
While specific side effects are not listed here, common ones for new treatments can include reactions at the injection site, headaches, nausea, and potential allergic responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 3 and 17 years old.
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I have a confirmed genetic diagnosis of HCH.
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My height is much shorter than average for my age and sex.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I might need surgery for an unstable condition during the study.
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My growth has slowed or stopped earlier than expected.
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I am not taking any medications that are not allowed in the study.
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I am planning to undergo surgery to lengthen my limb.
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I have been treated with growth hormone, IGF-1, or steroids.
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I have or might have cancer.
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I am not planning any bone surgery during the study, except for tooth extraction.
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I have never used experimental treatments or devices for HCH or short stature.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: vosoritide injection with vial and syringeExperimental Treatment1 Intervention
Group II: Placebo injection with vial and syringePlacebo Group1 Intervention

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
159 Previous Clinical Trials
114,994 Total Patients Enrolled
1 Trials studying Hypochondroplasia
400 Patients Enrolled for Hypochondroplasia
Medical Director, MDStudy DirectorBioMarin Pharmaceutical
80 Previous Clinical Trials
16,436 Total Patients Enrolled
1 Trials studying Hypochondroplasia
400 Patients Enrolled for Hypochondroplasia
~53 spots leftby Aug 2026
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