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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children with Hypochondroplasia, Aged 0 to < 36 Months

Status: Recruiting

The purpose of this study is to learn the effects, good or bad, of the study drug, vosoritide, for treating children who have hypochondroplasia. This condition affects the growth of bone and cartilage and in which kids who have it are shorter than other kids of the same age. In this study, your child will get either the study drug or placebo, which is an inactive medicine.

I'm interested

Sex: Male or Female
Age Group: Up to 18 years old
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• 0 months to less than 36 months old
• confirmed genetic diagnosis of Hypochondroplasia (HCH)
• participant's weight must be ≥ 3 kg (6.6 pounds)
• see link to clinicaltrials.gov for complete inclusion criteria
Exclusion Criteria:

• short stature condition other than HCH
• have an unstable medical condition
• see link to clinicaltrials.gov for complete exclusion criteria
Interventions:

Drug: Placebo, Drug: Vosoritide

Conditions:

Diabetes & Endocrine, Rare Diseases

Keywords:

HCH, Hypochondroplasia

Study Contact: Brad Miller - mille685@umn.edu
Principal Investigator: Brad Miller, MD, PhD
Phase: PHASE2
IRB Number: STUDY00026654
See this study on ClinicalTrials.gov

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