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Clinical Trials in Australia / NCT06410976
Recruiting Observational

Prospective Clinical Assessment Study in Children With Hypochondroplasia

NCT06410976 · tracked via the Priya Life Science Australia tracker
Phase
Observational
Started
2024-06-05
Last updated
2026-07-16

Condition(s) studied

Hypochondroplasia

Study summary

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

Eligibility

Sex
ALL
Min age
30 Months
Max age
16 Years
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 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.

Primary outcome measure(s)

Trial sites (25)

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

More QED Therapeutics, a BridgeBio company trials in Australia

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06410976 on ClinicalTrials.gov ↗ ← All trials in Australia