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Observational study of children with Hypochondroplasia

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

BioMarin Pharmaceutical · NCT06212947

This study is tracking the growth and development of children with Hypochondroplasia to see how the condition affects them as they grow up.

Quick facts

Study typeObservational
Enrollment400 (estimated)
AgesN/A to 15 Years
SexAll
SponsorBioMarin Pharmaceutical (industry)
Locations43 sites (Phoenix, Arizona and 42 other locations)
Trial IDNCT06212947 on ClinicalTrials.gov

What this trial studies

This observational study aims to assess the growth patterns and clinical progression of children diagnosed with Hypochondroplasia. By collecting growth measurements and other relevant variables over time, the study seeks to provide insights into the condition's impact on development. The study will involve multiple centers across different countries to gather a diverse set of data. Participants will be monitored until they reach the age of 15, allowing for a comprehensive understanding of the condition's trajectory.

Who should consider this trial

Good fit: Ideal candidates for this study are children aged 15 years or younger with a confirmed genetic diagnosis of Hypochondroplasia.

Not a fit: Patients with other genetic short stature conditions or those who have received investigational products within the last six months may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance understanding of Hypochondroplasia and inform better management strategies for affected children.

How similar studies have performed: While there may be limited studies specifically on Hypochondroplasia, observational studies in similar conditions have provided valuable insights and shown success in understanding growth patterns.

Eligibility criteria

Show full inclusion / exclusion 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

Where this trial is running

Phoenix, Arizona and 42 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Hypochondroplasia

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.