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Long-term assessment of children with hypochondroplasia

Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL

QED Therapeutics, a BridgeBio company · NCT06410976

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

Quick facts

Study typeObservational
Enrollment150 (estimated)
Ages30 Months to 16 Years
SexAll
SponsorQED Therapeutics, a BridgeBio company (industry)
Locations25 sites (Oakland, California and 24 other locations)
Trial IDNCT06410976 on ClinicalTrials.gov

What this trial studies

This observational study involves children aged 2.5 to under 17 years diagnosed with hypochondroplasia (HCH). It aims to evaluate various aspects such as growth patterns, medical complications related to HCH, health-related quality of life, functional abilities, and cognitive functions. The study is non-interventional, meaning no medications will be administered, and it will collect data to better understand the natural history of HCH in children. The findings will help characterize the condition and its impact on affected individuals.

Who should consider this trial

Good fit: Ideal candidates are children aged 2.5 to under 17 years with a clinical diagnosis of hypochondroplasia confirmed by molecular testing.

Not a fit: Patients with other short stature conditions or those who have undergone specific treatments like growth hormone therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the growth and health challenges faced by children with hypochondroplasia, leading to improved management strategies.

How similar studies have performed: While this study focuses on a specific condition, similar observational studies have successfully characterized other skeletal dysplasias, suggesting potential for valuable findings.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 prior to screening.

Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant.

Clinically significant abnormality in any laboratory test result at screening

Current evidence of corneal or retinal disorders.

Have used any other investigational or approved product or medical device for the treatment of HCH or short stature for ≥ 30 days or with the last dose \<6 months before screening.

Have had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).

Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening.

Having had a fracture of the long bones or spine within 12 months of screening.

History and/or current evidence of extensive ectopic tissue calcification.

History of malignancy.

Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation, and/or would place the participant at high risk for poor compliance with study activities or for not completing the study.

Current participation in any other ongoing clinical study with another sponsor.

Where this trial is running

Oakland, California and 24 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, skeletal dysplasia, endochondral ossification, hypochondroplasia, HCH, shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3

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.