HypochondroplasiaHCHNCT06410976
Prospective Clinical Assessment Study in Children With Hypochondroplasia
An observational study of Hypochondroplasia, run by QED Therapeutics, a BridgeBio company at 25 sites in 10 countries.
Trial summary
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH). Read more about this study
- Who is it for?
- Ages 30 months to 16 years
- Conditions the study is for
- Hypochondroplasia
- How many people
- 150 participants
- Where
- 25 sitesin 10 countries
- Study dates
- 5 Jun 2024to Oct 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 months to 16 years
- All sexes
- No healthy volunteers
- Child
Study population: Children with hypochondroplasia
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 13Show fewer
- 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.
Trial locations and contacts
25 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
25 sites in 10 countries
United States8 sites, 8 recruiting
- Recruiting
Childrens Hospital Colorado
Aurora, Colorado, 80045
- Recruiting
Johns Hopkins School of Medicine
Baltimore, Maryland, 21287
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
- Recruiting
University of Missouri
Columbia, Missouri, 65201
- Recruiting
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, 53705
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
- Recruiting
UCSF Benioff Children's Hospital
Oakland, California, 94609
- Recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
United Kingdom4 sites, 4 recruiting
- Recruiting
Glasgow Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, Scotland, G51 4TF
- Recruiting
The Portland Hospital for Women and Children
London, England, W1W 5AH
- Recruiting
Manchester University
Manchester, England, M13 9WL
- Recruiting
Sheffield Children's NHS Foundation Trust
Sheffield, England, S10 2TH
Canada3 sites, 3 recruiting
- Recruiting
London Health Sciences Centre - Children's Hospital of Western Ontario
London, Ontario, N6C 2R5
- Recruiting
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
- Recruiting
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, K1H 8L1
France3 sites, 3 recruiting
- Recruiting
Hôpital Femme Mère Enfant HCL
Bron, 69677
- Recruiting
Hôpital Necker-Enfants Malades
Paris, 75015
- Recruiting
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse, 31059
Norway2 sites, 2 recruiting
- Recruiting
Haukeland University Hospital
Bergen, 5021
- Recruiting
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo, 0372
Australia1 site, 1 recruiting
- Recruiting
Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Portugal1 site, 1 recruiting
- Recruiting
Hospital Pediátrico de Coimbra
Coimbra, 3000-602
Singapore1 site, 1 recruiting
- Recruiting
KK Women's and Children's Hospital
Singapore, 229899
Spain1 site, 1 recruiting
- Recruiting
Unidad de Cirugía Artroscopica, Hopsital MIKS
Vitoria-Gasteiz, 01010
Sweden1 site, 1 recruiting
- Recruiting
Astrid Lindgren Children's Hospital
Solna, 17164
About this study
Official title: Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).
Read the 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.
- Conditions
- Hypochondroplasia
- Keywords
- skeletal dysplasia, endochondral ossification, hypochondroplasia, HCH, shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, endochondral bone formation, disproportionate short stature, quality of life, dwarfism, bone diseases, musculoskeletal diseases, osteochondrodysplasia, functional abilities, annualized growth velocity, annualized height velocity, growth, genetic diseases, congenital
- Medical subject headings
- Hypochondroplasia, Mucopolysaccharidosis IV, Dwarfism, Bone Diseases, Musculoskeletal Diseases, Osteochondrodysplasias, Genetic Diseases, Inborn
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized height velocity
Measured: up to 3 years
Secondary outcomes (5)
Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio
Measured: up to 3 years
Cognitive functions
Measured: up to 3 years
HCH-related medical events reported as medical history or NT-AEs
Measured: up to 3 years
HCH-related surgical procedures
Measured: up to 3 years
Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form.
Scale scores 0-100. Higher score=better Health-Related Quality of Life
Measured: up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2024
- Study start5 Jun 2024
- Primary completionOct 2026 (estimated)
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypochondroplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06410976
- Sponsor's protocol ID
- QBGJ398-004
- Acronym
- HCH
- Status last verified
- May 2026
- First submitted
- 8 May 2024
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL
- ClinicalTrials.gov ID
- NCT06410976
- Acronym / sponsor ID
- HCH / QBGJ398-004
- Conditions
- Hypochondroplasia
- Keywords
- skeletal dysplasia, endochondral ossification, hypochondroplasia, HCH, shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, endochondral bone formation
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- QED Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Study start
- 5 Jun 2024 (actual)
- Primary completion
- Oct 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 13 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 30 months to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children with hypochondroplasia
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Annualized height velocity
Time frame: up to 3 years
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
25 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypochondroplasia, run by QED Therapeutics, a BridgeBio company at 25 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, United Kingdom, Canada, France, Norway and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact QED Therapeutics, Inc (1-877-280-5655, medinfo@qedtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized height velocity (time frame: up to 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2026.