Prospective Clinical Assessment Study in Children With Hypochondroplasia
- Conditions
- Hypochondroplasia
- Registration Number
- NCT06410976
- Lead Sponsor
- QED Therapeutics, Inc.
- Brief 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 150
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.
Have ACH or short stature condition other than HCH.
In females, having had their menarche. Annualized height growth velocity โค1.5 cm/year over a period โฅ6 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
Have been treated with growth hormone, IGF 1, or anabolic steroids the previous 6 months or long-term treatment (>3 months) at any time.
Current evidence of corneal or retinal disorders.
Have used any other investigational product or investigational medical device for the treatment of HCH or short stature.
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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Annualized height velocity up to 2 years
- Secondary Outcome Measures
Name Time Method Cognitive functions up to 2 years Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio up to 2 years HCH-related medical events reported as medical history or NT-AEs up to 2 years HCH-related surgical procedures up to 2 years Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form. up to 2 years Scale scores 0-100. Higher score=better Health-Related Quality of Llife
Related Research Topics
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Trial Locations
- Locations (25)
Hรดpital Necker-Enfants Malades
๐ซ๐ทParis, France
Hรดpital Femme Mรจre Enfant HCL
๐ซ๐ทBron, France
Sheffield Children's NHS Foundation Trust
๐ฌ๐งSheffield, United Kingdom
UCSF Benioff Children's Hospital
๐บ๐ธOakland, California, United States
Childrens Hospital Colorado
๐บ๐ธAurora, Colorado, United States
Children's National Hospital
๐บ๐ธWashington DC, District of Columbia, United States
Johns Hopkins School of Medicine
๐บ๐ธBaltimore, Maryland, United States
University of Missouri
๐บ๐ธColumbia, Missouri, United States
Vanderbilt University Medical Center
๐บ๐ธNashville, Tennessee, United States
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
๐บ๐ธMadison, Wisconsin, United States
Murdoch Children's Research Institute
๐ฆ๐บParkville, Victoria, Australia
London Health Sciences Centre - Children's Hospital of Western Ontario
๐จ๐ฆLondon, Ontario, Canada
Children's Hospital of Eastern Ontario Research Institute
๐จ๐ฆOttawa, Ontario, Canada
Universitรฉ de Montrรฉal - Centre Hospitalier Universitaire Sainte-Justine
๐จ๐ฆMontrรฉal, Quebec, Canada
Centre Hospitalier Universitaire (CHU) de Toulouse - Hรดpital des Enfants
๐ซ๐ทToulouse, France
University Hospital Freiburg - Centre for Paediatric and Adolescent Medicine
๐ฉ๐ชFreiburg, Baden-Wurtemberg, Germany
Otto Von Guericke Universitat
๐ฉ๐ชMagdeburg, Sachsen-Anhalt, Germany
Haukeland University Hospital
๐ณ๐ดBergen, Norway
Paediatric Clinical Research Unit at Oslo University Hospital
๐ณ๐ดOslo, Norway
Hospital Pediaฬtrico de Coimbra
๐ต๐นCoimbra, Portugal
KK Women's and Children's Hospital
๐ธ๐ฌSingapore, Singapore
Hospital Vithas San Jose
๐ช๐ธVitoria-gasteiz, Spain
Astrid Lindgren Children's Hospital
๐ธ๐ชSolna, Sweden
The Portland Hospital for Women and Children
๐ฌ๐งLondon, United Kingdom
Manchester University
๐ฌ๐งManchester, United Kingdom