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NCT06410976Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia

QED Therapeutics, a BridgeBio company

Start Date

6/5/2024

Completion Date

10/1/2026

Summary

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

Detailed 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 Criteria

Age Range: No minimum to 16 years

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.

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Conditions

Hypochondroplasia

Locations

UCSF Benioff Children's Hospital

Oakland, California 94609

United States

Childrens Hospital Colorado

Aurora, Colorado 80045

United States

Children's National Hospital

Washington D.C., District of Columbia 20010

United States

Johns Hopkins School of Medicine

Baltimore, Maryland 21287

United States

University of Missouri

Columbia, Missouri 65201

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio 45229

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232

United States

University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

Madison, Wisconsin 53705

United States

Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne

Parkville, Victoria 3052

Australia

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

London, Ontario N6C 2R5

Canada

Children's Hospital of Eastern Ontario Research Institute

Ottawa, Ontario K1H 8L1

Canada

Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec H3T 1C5

Canada

Hôpital Femme Mère Enfant HCL

Bron, 69677

France

Hôpital Necker-Enfants Malades

Paris, 75015

France

Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants

Toulouse, 31059

France

Haukeland University Hospital

Bergen, 5021

Norway

Paediatric Clinical Research Unit at Oslo University Hospital

Oslo, 0372

Norway

Hospital Pediátrico de Coimbra

Coimbra, 3000-602

Portugal

KK Women's and Children's Hospital

Singapore, 229899

Singapore

Unidad de Cirugía Artroscopica, Hopsital MIKS

Vitoria-Gasteiz, 01010

Spain

Astrid Lindgren Children's Hospital

Solna, 17164

Sweden

The Portland Hospital for Women and Children

London, England W1W 5AH

United Kingdom

Manchester University

Manchester, England M13 9WL

United Kingdom

Sheffield Children's NHS Foundation Trust

Sheffield, England S10 2TH

United Kingdom

Glasgow Clinical Research Facility, Queen Elizabeth University Hospital

Glasgow, Scotland G51 4TF

United Kingdom