A Multicenter Multinational Observational Study of Children With Hypochondroplasia

a study on Hypochondroplasia

Summary

Eligibility
for people ages up to 15 years (full criteria)
Location
at UCSF
Dates
study started
completion around

Description

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.

Keywords

Hypochondroplasia, Dwarfism, Lordosis, Congenital Limb Deformities

Eligibility

You can join if…

Open to people ages up to 15 years

  • Participants must be ≤ 15 years old at the time of signing the informed consent
  • Participants must have genetic confirmation of Hypochondroplasia diagnosis

You CAN'T join if...

  • 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

Locations

  • UCSF Benioff Children's Hospital accepting new patients
    Oakland California 94609 United States
  • Cedars-Sinai Medical Center not yet accepting patients
    Los Angeles California 90048 United States
  • Phoenix Children's Hospital accepting new patients
    Phoenix Arizona 85016 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
ID
NCT06212947
Study Type
Observational
Participants
Expecting 400 study participants
Last Updated