Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
Status: Active_not_recruiting
Location: See all (32) locations...
Study Type: Observational
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 participants. No study medication will be administered.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 17
Healthy Volunteers: f
View:
• 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
Locations
United States
California
Benioff Children's Hospital Oakland
Oakland
Colorado
Children's Hospital Colorado
Aurora
Delaware
Nemours Alfred I. Dupont Hospital for Children
Wilmington
Maryland
Johns Hopkins School of Medicine
Baltimore
Missouri
University of Missouri
Columbia
Ohio
Cincinnati Children's Hospital
Cincinnati
Tennessee
Vanderbilt University Medical Center
Nashville
Wisconsin
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison
Other Locations
Argentina
Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
Buenos Aires
Australia
Murdoch Children's Research Institute
Parkville
Canada
Stollery Children's Hospital
Edmonton
Children's Hospital - London Health Sciences Center
London
University of Montreal
Montreal
University of Ottawa
Ottawa
France
Hopital Femme Mere Enfant
Lyon
Hopital Necker-Enfants Malades
Paris
Hopital des Enfants
Toulouse
Germany
Otto-von-Guericke-University Magdeburg Medical Fakulty
Magdeburg
Italy
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlino
Milan
Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS
Rome
Norway
Haukeland Universitetssjukehus
Bergen
Oslo Universitetssykehus
Oslo
Singapore
KK Women's and Children's Hospital
Singapore
Spain
Vithas Hospital San Jose
Barcelona
Hospital Universitario La Paz
Madrid
Hospital Universitario Virgen de la Victoria
Málaga
United Kingdom
Birmingham Children's Hospital
Birmingham
Bristol Royal Hospital for Children
Bristol
Queen Elizabeth University Hospital
Glasgow
St. Thomas' Hospital
London
Manchester University Children's Hospital
Manchester
Sheffield Children's Hospital
Sheffield
Time Frame
Start Date: 2019-08-12
Completion Date: 2026-06
Participants
Target number of participants: 271
Authors
Related Therapeutic Areas
Sponsors
Leads: QED Therapeutics, a BridgeBio company