A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

Status: Completed
Location: See all (20) locations...
Study Type: Observational
SUMMARY

Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in future studies sponsored by BioMarin. No study drug is administered.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: No
View:

• Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, willing and able to provide written assent (as needed) after the nature of the study has been explained and prior to performance of any research-related procedure.

• Aged 0 to <= 17 years, inclusive, at study entry.

• Have ACH, documented by clinical diagnosis

• Are ambulatory and able to stand without assistance (not applicable for infants)

• Are willing and able to perform all study procedures as physically possible.

Locations
United States
California
Harbor-UCLA Medical Center
Los Angeles
Children's Hospital and Research Center Oakland
Oakland
Delaware
Nemours/Alfred I. duPont Hospital for Children
Wilmington
Georgia
Emory University
Decatur
Illinois
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Tennessee
Vanderbilt University
Nashville
Texas
Baylor College of Medicine
Houston
Wisconsin
Medical College of Wisconsin, Children's Hospital
Milwaukee
Other Locations
Australia
Murdoch Children's Research Institute
Parkville
The Children's Hospital at Westmead
Westmead
Japan
Osaka University Hospital
Osaka
Saitama Children's Medical Center
Saitama
Tokushima University Hospital
Tokushima
Spain
Hospital Sant Joan de Deu Barcelona
Barcelona
Institut Catala de Traumatologica I Medicina de l'Esport
Barcelona
Hospital Universitario Virgen de la Victoria
Málaga
Turkey
Acibadem University School of Medicine
Istanbul
United Kingdom
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
London
Sheffield Children's NHS Foundation Trust
Sheffield
Time Frame
Start Date: April 2012
Completion Date: February 2021
Participants
Target number of participants: 363
Treatments
Growth measurements
Approximately 500 patients will be enrolled. Patients from birth to <= 17 years on the date of consent will be enrolled. Approximately equal numbers of boys and girls will be enrolled.
Authors
David Weaver, Donald Basel, Gary Bellus, Howard Saal, Klane White, Daniel G Hoernschemeyer, Rosendo Ullot, Valerie Cormier-Daire, Ignacio Ginebreda, Patti Dickson, Felipe Luna, Yesemin Alanay, Selda Karaayvaz, Melita Irving, Paul Arundel
Sponsors
Leads: BioMarin Pharmaceutical

This content was sourced from clinicaltrials.gov

Similar Clinical Trials