AchondroplasiaNCT04035811
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
An observational study of Achondroplasia, run by QED Therapeutics, a BridgeBio company at 32 sites in 11 countries.
Trial summary
This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study… Read more about this study
- Who is it for?
- Ages 30 months to 17 years
- Conditions the study is for
- Achondroplasia
- How many people
- 271 participants
- Where
- 32 sitesin 11 countries
- Study dates
- 12 Aug 2019to 27 Aug 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Children with achondroplasia
Who may be able to join
- Signed informed consent by study participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the study participant (when applicable)
- Aged 2.5 to <17 years at study entry
- Diagnosis of ACH
- Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures
Who cannot join
- Have hypochondroplasia or short stature condition other than ACH (e.g. trisomy 21, pseudoachondroplasia, psychosocial short stature)
- In females, having had their menarche
- Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH
- Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening
- Current evidence of corneal or retinal disorder/keratopathy
- Current evidence of endocrine alterations of calcium/phosphorus homeostasis
Show all 11Show fewer
- Have a concurrent disease or condition that in the view of the Investigator and/or Sponsor, may impact growth or where the treatment is known to impact growth.
- Significant abnormality in screening laboratory results.
- Have been treated with growth hormone, insulin-like growth factor 1 (IGF 1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time
- Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
- Have had previous guided growth surgery or limb-lengthening surgery within 12 months prior to screening.
Trial locations and contacts
32 sites in 11 countries.
32 sites in 11 countries
United States8 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins School of Medicine
Baltimore, Maryland, 21211
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
University of Missouri
Columbia, Missouri, 65211
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, 53705
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Benioff Children's Hospital Oakland
Oakland, California, 94609
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, 19803
United Kingdom6 sites
Birmingham Children's Hospital
Birmingham
Bristol Royal Hospital for Children
Bristol, England
Queen Elizabeth University Hospital
Glasgow
St. Thomas' Hospital
London
Manchester University Children's Hospital
Manchester
Sheffield Children's Hospital
Sheffield
Canada4 sites
Stollery Children's Hospital
Edmonton, Alberta, T6G 2H7
Children's Hospital - London Health Sciences Center
London, Ontario, N6A 5W9
University of Montreal
Montreal, Quebec, H3C 3J7
University of Ottawa
Ottawa, Ontario, K1N 6N5
France3 sites
Hopital Femme Mere Enfant
Lyon
Hopital Necker-Enfants Malades
Paris
Hopital des Enfants
Toulouse
Spain3 sites
Vithas Hospital San Jose
Barcelona, 08305
Hospital Universitario La Paz
Madrid, 24086
Hospital Universitario Virgen de la Victoria
Málaga
Italy2 sites
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlino
Milan, 20122
Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS
Rome, 00168
Norway2 sites
Haukeland Universitetssjukehus
Bergen, 5009
Oslo Universitetssykehus
Oslo, 0372
Argentina1 site
Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
Buenos Aires
Germany1 site
Otto-von-Guericke-University Magdeburg Medical Fakulty
Magdeburg, 39120
About this study
Official title: Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.
- Conditions
- Achondroplasia
- Keywords
- Skeletal dysplasia, Endochondral ossification, Achondroplasia (ACH), Shortened proximal limbs, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate dwarfism, Quality of life in achondroplasia, Dwarfism, Bone diseases, Musculoskeletal diseases, Osteochondrodysplasia, Genetic diseases, Inborn, Functional abilities, Average growth velocity, Average height velocity, growth, Congenital
- Medical subject headings
- Achondroplasia, 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 (cm/year)
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2019
- Study start12 Aug 2019
- Primary completion27 Aug 2025
- Study completion27 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AchondroplasiaStudies 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
- 271 participants (actual)
Sponsor and investigators
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- QED Therapeutics, Inc. VP, Clinical Development, Study director, QED Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04035811
- Sponsor's protocol ID
- QBGJ398-001
- Status last verified
- Mar 2026
- First submitted
- 20 Jul 2019
- Last updated
- 3 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Savarirayan R, De Bergua JM, Arundel P, McDevitt H, Cormier-Daire V, Saraff V, Skae M, Delgado B, Leiva-Gea A, Santos-Simarro F, Salles JP, Nicolino M, Rossi M, Kannu P, Bober MB, Phillips J 3rd, Saal H, Harmatz P, Burren C, Gotway G, Cho T, Muslimova E, Weng R, Rogoff D, Hoover-Fong J, Irving M. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. Ther Adv Musculoskelet Dis. 2022 Mar 21;14:1759720X221084848. doi: 10.1177/1759720X221084848. eCollection 2022.
PubMed 35342457
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
- ClinicalTrials.gov ID
- NCT04035811
- Acronym / sponsor ID
- QBGJ398-001
- Conditions
- Achondroplasia
- Keywords
- Skeletal dysplasia, Endochondral ossification, Achondroplasia (ACH), Shortened proximal limbs, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate dwarfism
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 271 participants (actual)
- Lead sponsor
- QED Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Study start
- 12 Aug 2019 (actual)
- Primary completion
- 27 Aug 2025 (actual)
- Study completion
- 27 Aug 2025 (actual)
- First posted
- 29 Jul 2019 (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, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 30 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children with achondroplasia
Key inclusion criteria (4 listed)
- Signed informed consent by study participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the study participant (when applicable)
- Aged 2.5 to <17 years at study entry
- Diagnosis of ACH
- Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures
Key exclusion criteria (11 listed)
- Have hypochondroplasia or short stature condition other than ACH (e.g. trisomy 21, pseudoachondroplasia, psychosocial short stature)
- In females, having had their menarche
- Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH
- Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening
- Current evidence of corneal or retinal disorder/keratopathy
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 (cm/year)
Time frame: Up to 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2026.
- Derived from this study: PubMed 35342457Savarirayan R, De Bergua JM, Arundel P, McDevitt H, Cormier-Daire V, Saraff V, Skae M, Delgado B, Leiva-Gea A, Santos-Simarro F, Salles JP, Nicolino M, Rossi M, Kannu P, Bober MB, Phillips J 3rd, Saal H, Harmatz P, Burren C, Gotway G, Cho T, Muslimova E, Weng R, Rogoff D, Hoover-Fong J, Irving M. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. Ther Adv Musculoskelet Dis. 2022 Mar 21;14:1759720X221084848. doi: 10.1177/1759720X221084848. eCollection 2022.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Achondroplasia
Enrolling by invitationPhase 2
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
Achondroplasia
RecruitingPhase 2/3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Achondroplasia
Active, not recruitingPhase 2
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Achondroplasia
Active, not recruitingPhase 3
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
Achondroplasia
RecruitingPhase 2
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Achondroplasia
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. This study is not recruiting at the moment.
QED Therapeutics, Inc. VP, Clinical Development study director
QED Therapeutics
32 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Achondroplasia, run by QED Therapeutics, a BridgeBio company at 32 sites in 11 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 4 inclusion and 11 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: finished, as of the sponsor's last update on 3 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, United Kingdom, Canada, France, Spain and 6 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized height velocity (cm/year) (time frame: Up to 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04035811, which also lists 1 linked publication.
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 3 Apr 2026; AzTrials last refreshed its copy on 7 Nov 2026.