Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL
QED Therapeutics, a BridgeBio company
Summary
This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).
Description
The objective is to evaluate growth, HCH-related medical complications, health-related quality of life, functional abilities and cognitive functions of study participants. Data collected will contribute to the characterization of the natural history of children with HCH. No study medication will be administered.
Eligibility
- Age range
- 2–16 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Signed informed consent. Aged 2.5 to \<17 years at study entry. Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test. Participants are ambulatory and able to stand without assistance. Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. Exclusion Criteria: Have ACH or short stature condition other than HCH. In females, having had their menarche. Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months…