Prospective Clinical Assessment Study in Children With Hypochondroplasia

Recruiting now

Conditions studied: Hypochondroplasia

In brief

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

Key facts

Study ID: NCT06410976
Run by: QED Therapeutics, a BridgeBio company
People needed: 150
Starts: 2024-06-05
Expected to finish: 2026-10-01
Last updated by the study team: 2026-07-16

Who can join

Age: 3 and older, up to 16. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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 it is running

UCSF Benioff Children's Hospital — Oakland, California, United States (enrolling)
Childrens Hospital Colorado — Aurora, Colorado, United States (enrolling)
Children's National Hospital — Washington D.C., District of Columbia, United States (enrolling)
Johns Hopkins School of Medicine — Baltimore, Maryland, United States (enrolling)
University of Missouri — Columbia, Missouri, United States (enrolling)
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
Vanderbilt University Medical Center — Nashville, Tennessee, United States (enrolling)
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic — Madison, Wisconsin, United States (enrolling)
Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne — Parkville, Victoria, Australia (enrolling)
London Health Sciences Centre - Children's Hospital of Western Ontario — London, Ontario, Canada (enrolling)
Children's Hospital of Eastern Ontario Research Institute — Ottawa, Ontario, Canada (enrolling)
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec, Canada (enrolling)
Hôpital Femme Mère Enfant HCL — Bron, France (enrolling)
Hôpital Necker-Enfants Malades — Paris, France (enrolling)
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants — Toulouse, France (enrolling)
Haukeland University Hospital — Bergen, Norway (enrolling)
Paediatric Clinical Research Unit at Oslo University Hospital — Oslo, Norway (enrolling)
Hospital Pediátrico de Coimbra — Coimbra, Portugal (enrolling)
KK Women's and Children's Hospital — Singapore, Singapore (enrolling)
Unidad de Cirugía Artroscopica, Hopsital MIKS — Vitoria-Gasteiz, Spain (enrolling)
Astrid Lindgren Children's Hospital — Solna, Sweden (enrolling)
The Portland Hospital for Women and Children — London, England, United Kingdom (enrolling)
Manchester University — Manchester, England, United Kingdom (enrolling)
Sheffield Children's NHS Foundation Trust — Sheffield, England, United Kingdom (enrolling)
Glasgow Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow, Scotland, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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