Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
Completed
Conditions studied: Achondroplasia
In brief
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.
Key facts
- Study ID
- NCT04035811
- Run by
- QED Therapeutics, a BridgeBio company
- People needed
- 271
- Starts
- 2019-08-12
- Expected to finish
- 2025-08-27
- Last updated by the study team
- 2026-04-03
Who can join
Age: 3 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
Where it is running
- 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
Full record on ClinicalTrials.gov
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