Prospective Clinical Assessment Study in Children With Hypochondroplasia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Hypochondroplasia. Basic parameters: 30 months — 16 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Canada, France, Norway +5
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL
Overview
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.
Primary outcome measures
- Annualized height velocity [Time frame: up to 3 years]
Secondary outcome measures (5)
- Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio [Time frame: up to 3 years]
- Cognitive functions [Time frame: up to 3 years]
- HCH-related medical events reported as medical history or NT-AEs [Time frame: up to 3 years]
- HCH-related surgical procedures [Time frame: up to 3 years]
- Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form. [Time frame: up to 3 years]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
United States · 8 centers
- UCSF Benioff Children's Hospital — Oakland
- Childrens Hospital Colorado — Aurora
- Children's National Hospital — Washington D.C.
- Johns Hopkins School of Medicine — Baltimore
- University of Missouri — Columbia
- Cincinnati Children's Hospital Medical Center — Cincinnati
- Vanderbilt University Medical Center — Nashville
- University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic — Madison
United Kingdom · 4 centers
- The Portland Hospital for Women and Children — London
- Manchester University — Manchester
- Sheffield Children's NHS Foundation Trust — Sheffield
- Glasgow Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow
Canada · 3 centers
- London Health Sciences Centre - Children's Hospital of Western Ontario — London
- Children's Hospital of Eastern Ontario Research Institute — Ottawa
- Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine — Montreal
France · 3 centers
- Hôpital Femme Mère Enfant HCL — Bron
- Hôpital Necker-Enfants Malades — Paris
- Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants — Toulouse
Norway · 2 centers
- Haukeland University Hospital — Bergen
- Paediatric Clinical Research Unit at Oslo University Hospital — Oslo
Australia · 1 center
- Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne — Parkville
Portugal · 1 center
- Hospital Pediátrico de Coimbra — Coimbra
Singapore · 1 center
- KK Women's and Children's Hospital — Singapore
Spain · 1 center
- Unidad de Cirugía Artroscopica, Hopsital MIKS — Vitoria-Gasteiz
Sweden · 1 center
- Astrid Lindgren Children's Hospital — Solna
Identifiers
NCT: NCT06410976 · QBGJ398-004