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RecruitingNCT06212947

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Trial Parameters

ConditionHypochondroplasia
SponsorBioMarin Pharmaceutical
Study TypeOBSERVATIONAL
PhaseN/A
Enrollment400
SexALL
Min AgeN/A
Max Age15 Years
Start Date2023-11-27
Completion2043-12-31

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Brief Summary

This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest.

Eligibility Criteria

Inclusion Criteria: * Participants must be ≤ 15 years old at the time of signing the informed consent * Participants must have genetic confirmation of Hypochondroplasia diagnosis Exclusion Criteria: * Have a diagnosis of another genetic short stature condition other than Hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature * Received an investigational product or medical device within 6 months before the Screening visit

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