A Multicenter Multinational Observational Study of Children With Hypochondroplasia

BioMarin Pharmaceutical

Status: Enrolling

Conditions: Hypochondroplasia

Study type: Observational

Funder types: Industry

Identifiers

NCT06212947

111-902

Details and patient eligibility

About

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

Enrollment: 400 estimated patients

Sex: All

Ages: Under 15 years old

Volunteers: No Healthy Volunteers

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

Trial design

400 participants in 1 patient group

Children with Hypochondroplasia

Description:

Children with Hypochondroplasia