Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
NCT ID: NCT04035811
Last Updated: 2025-04-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
271 participants
OBSERVATIONAL
2019-08-12
2026-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
* 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.
30 Months
17 Years
ALL
No
Sponsors
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QED Therapeutics, Inc., a Bridgebio company
INDUSTRY
Responsible Party
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Principal Investigators
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QED Therapeutics, Inc. VP, Clinical Development
Role: STUDY_DIRECTOR
QED Therapeutics
Locations
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Benioff Children's Hospital Oakland
Oakland, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
University of Missouri
Columbia, Missouri, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, United States
Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
Buenos Aires, , Argentina
Murdoch Children's Research Institute
Parkville, , Australia
Stollery Children's Hospital
Edmonton, Alberta, Canada
Children's Hospital - London Health Sciences Center
London, Ontario, Canada
University of Ottawa
Ottawa, Ontario, Canada
University of Montreal
Montreal, Quebec, Canada
Hopital Femme Mere Enfant
Lyon, , France
Hopital Necker-Enfants Malades
Paris, , France
Hopital des Enfants
Toulouse, , France
Otto-von-Guericke-University Magdeburg Medical Fakulty
Magdeburg, , Germany
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlino
Milan, , Italy
Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS
Rome, , Italy
Haukeland Universitetssjukehus
Bergen, , Norway
Oslo Universitetssykehus
Oslo, , Norway
KK Women's and Children's Hospital
Singapore, , Singapore
Vithas Hospital San Jose
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario Virgen de la Victoria
Málaga, , Spain
Bristol Royal Hospital for Children
Bristol, England, United Kingdom
Birmingham Children's Hospital
Birmingham, , United Kingdom
Queen Elizabeth University Hospital
Glasgow, , United Kingdom
St. Thomas' Hospital
London, , United Kingdom
Manchester University Children's Hospital
Manchester, , United Kingdom
Sheffield Children's Hospital
Sheffield, , United Kingdom
Countries
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References
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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.
Related Links
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Related Info
Other Identifiers
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QBGJ398-001
Identifier Type: -
Identifier Source: org_study_id
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