Observational study of children with Hypochondroplasia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Phoenix Children's Hospital
  Phoenix, Arizona, United States Recruiting
• UCSF Benioff Children's Hospital
  Oakland, California, United States Recruiting
• Nemours Alfred I. Dupont Hospital for Children
  Wilmington, Delaware, United States Recruiting
• Children's National Health System
  Washington D.C., District of Columbia, United States Recruiting
• Ann & Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting

Study contacts

• Study coordinator: Trial Specialist
• Email: medinfo@bmrn.com
• Phone: 1-800-983-4587

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Measuring limb movement in people with FGFR3-related skeletal dysplasia

  This project will try using Syde® wearable movement recordings to see which measurements reliably track limb movement in people aged 3 and older with achondroplasia or hypochondroplasia.

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• Daily vosoritide versus placebo for infants and toddlers with hypochondroplasia

  This trial will test whether daily vosoritide injections for 52 weeks help infants and toddlers under 36 months with genetically confirmed hypochondroplasia grow better and stay healthier.

  Recruiting at 17 sites Phase 2 BioMarin Pharmaceutical · Phoenix, Arizona and 25 other locations · NCT07126262

• Long-term assessment of children with hypochondroplasia

  This study looks at how children with hypochondroplasia grow and develop over time to better understand their health and quality of life.

  Recruiting at 25 sites QED Therapeutics, a BridgeBio company · Oakland, California and 24 other locations · NCT06410976

• Registry for patients with achondroplasia and hypochondroplasia

  This study is collecting health information from people with achondroplasia and hypochondroplasia to help understand these conditions better and improve future treatments.

  Recruiting Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico · Milan · NCT05328050

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.