Recruiting

Observational Study

Sponsor:

QED Therapeutics, a BridgeBio company

Code:

NCT06410976

Conditions

Hypochondroplasia

Eligibility Criteria

Sex: All

Age: 2 - 16

Healthy Volunteers: Not accepted

Study Details

Brief summary:

This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).

Conditions

Hypochondroplasia

Study ID

NCT06410976

Start date

Jun 5, 2024

Status verified date

May, 2026

Completion date

Oct, 2026

Anticipated

Primary completion date

Oct, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 16

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

150 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

  • Annualized height velocity [ Time Frame: up to 3 years ]

Central Contacts and Locations

Central contacts

Locations

UCSF Benioff Children's Hospital

Recruiting

Oakland, California, United States, 94609

Childrens Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Children's National Hospital

Recruiting

Washington D.C., District of Columbia, United States, 20010

Johns Hopkins School of Medicine

Recruiting

Baltimore, Maryland, United States, 21287

University of Missouri

Recruiting

Columbia, Missouri, United States, 65201

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Vanderbilt University Medical Center

Recruiting

Nashville, Tennessee, United States, 37232

University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

Recruiting

Madison, Wisconsin, United States, 53705

London Health Sciences Centre - Children's Hospital of Western Ontario

Recruiting

London, Ontario, Canada, N6C 2R5

Children's Hospital of Eastern Ontario Research Institute

Recruiting

Ottawa, Ontario, Canada, K1H 8L1

Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine

Recruiting

Montreal, Quebec, Canada, H3T 1C5

More Information

Sponsor

QED Therapeutics, a BridgeBio company

Last update posted

Jul 16, 2026

Last verified

May, 2026

Keywords

  • skeletal dysplasia
  • endochondral ossification
  • hypochondroplasia
  • HCH
  • shortened proximal limbs
  • fibroblast growth factor receptor 3
  • FGFR3
  • endochondral bone formation
  • disproportionate short stature
  • quality of life
  • dwarfism
  • bone diseases
  • musculoskeletal diseases
  • osteochondrodysplasia
  • functional abilities
  • annualized growth velocity
  • annualized height velocity
  • growth
  • genetic diseases
  • congenital
  • AHV
  • AGV

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by QED Therapeutics, a BridgeBio company on 2026-07-16.