Actively Recruiting
Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL
Led by QED Therapeutics, a BridgeBio company · Updated on 2025-10-10
150
Participants Needed
25
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a long-term, multicenter, observational study involving children aged 2.5 to less than 17 years who have hypochondroplasia (HCH). The study aims to evaluate the growth patterns, medical complications related to HCH, quality of life, functional abilities, and cognitive functions of the participants. The data collected will help better understand the natural history of children with this condition. No medications or treatments will be given as part of this study. Participants will not receive any study medication, as this is a non-interventional observational study. Instead, researchers will monitor various health aspects over time, including changes in height and limb ratios, cognitive abilities, and any medical or surgical events related to HCH. The study will last up to three years, during which progress and health status will be observed and recorded. During the study, children and their caregivers will attend regular visits for assessments. These include measurements of height velocity, limb proportions, cognitive function evaluations, and quality of life questionnaires. Medical history and any HCH-related events will also be documented. The primary outcome is the annual growth rate in height over up to three years, with additional secondary measures tracking physical and cognitive changes. The study involves continuous monitoring to gather comprehensive information about each participant's health and development over the study period.
CONDITIONS
Brief Title
Prospective Clinical Assessment Study in Children With Hypochondroplasia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent.
- Aged 2.5 to less than 17 years at study entry.
- Diagnosis of hypochondroplasia confirmed clinically by disproportionate short stature and molecular testing.
- Participants are ambulatory and able to stand without assistance.
- Participants and their parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and procedures.
You will not qualify if you...
- Have achondroplasia or any short stature condition other than hypochondroplasia.
- Females who have had their menarche.
- Annualized height growth velocity less than or equal to 1.5 cm/year over at least 6 months before screening.
- Clinically significant disease or condition interfering with growth evaluation or study participation.
- Significant abnormal laboratory test results at screening.
- Current corneal or retinal disorders.
- Use of investigational or approved products/devices for hypochondroplasia or short stature for 30 days or more or dosing within 6 months before screening.
- Regular long-term oral corticosteroid treatment (except low-dose inhaled steroids for asthma).
- Previous limb-lengthening or guided growth surgery with plates in place or removed within 6 months before screening.
- Fracture of long bones or spine within 12 months before screening.
- History or current evidence of extensive ectopic tissue calcification.
- History of malignancy.
- Conditions or circumstances posing high risk for poor compliance or interfering with participation.
- Current participation in any other ongoing clinical study with another sponsor.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 3 years
Participants are observed over time to evaluate growth, medical complications related to hypochondroplasia, quality of life, functional abilities, and cognitive functions.
Regular visits during the study period
Trial Site Locations
Total: 25 locations
1
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
Actively Recruiting
2
Childrens Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
4
Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21287
Actively Recruiting
5
University of Missouri
Columbia, Missouri, United States, 65201
Actively Recruiting
6
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
7
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
8
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, United States, 53705
Actively Recruiting
9
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Actively Recruiting
10
London Health Sciences Centre - Children's Hospital of Western Ontario
London, Ontario, Canada, N6C 2R5
Actively Recruiting
11
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
12
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada, H3T 1C5
Actively Recruiting
13
Hôpital Femme Mère Enfant HCL
Bron, France, 69677
Actively Recruiting
14
Hôpital Necker-Enfants Malades
Paris, France, 75015
Actively Recruiting
15
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse, France, 31059
Actively Recruiting
16
Haukeland University Hospital
Bergen, Norway, 5021
Actively Recruiting
17
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo, Norway, 0372
Actively Recruiting
18
Hospital Pediátrico de Coimbra
Coimbra, Portugal, 3000-602
Actively Recruiting
19
KK Women's and Children's Hospital
Singapore, Singapore, 229899
Actively Recruiting
20
Hospital Vithas San Jose
Vitoria-Gasteiz, Spain, 01008
Actively Recruiting
21
Astrid Lindgren Children's Hospital
Solna, Sweden, 17164
Actively Recruiting
22
The Portland Hospital for Women and Children
London, England, United Kingdom, W1W 5AH
Actively Recruiting
23
Manchester University
Manchester, England, United Kingdom, M13 9WL
Actively Recruiting
24
Sheffield Children's NHS Foundation Trust
Sheffield, England, United Kingdom, S10 2TH
Actively Recruiting
25
Glasgow Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, Scotland, United Kingdom, G51 4TF
Actively Recruiting
Research Team
Q
QED Therapeutics, Inc
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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