A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Start Date
3/4/2025
Completion Date
6/1/2030
Summary
The purpose of this study is to evaluate the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with achondroplasia with open growth plates.
Detailed Description
This is a Phase 2, multicenter, open-label, dose-escalation study to determine the safety, tolerability, and identify potentially effective dose(s) of TYRA-300, a fibroblast growth factor receptor (FGFR)-3 selective tyrosine kinase inhibitor, in children 3 to 10 years of age with achondroplasia with open growth plates that will examine three cohorts of children: the Sentinel Safety Cohort, Cohort 1, and Cohort 2.
Eligibility Criteria
Age Range: 3 years to 10 years
Interventions
TYRA-300 0.125 mg/kg
TYRA-300 0.25 mg/kg
TYRA-300 0.375 mg/kg
TYRA-300 0.50 mg/kg
Conditions
Locations
Lundquist Institute for Biomedical Innovation
Torrance, California 90502
United States
Children's Hospital Colorado
Aurora, Colorado 80045
United States
Nemours Alfred I duPont Hospital for Children
Wilmington, Delaware 19803
United States
Johns Hopkins University School of Medicine
Baltimore, Maryland 21205
United States
Uncommon Cures
Chevy Chase, Maryland 20815
United States
University of Missouri
Columbia, Missouri 65201
United States
Washington University
St Louis, Missouri 63130
United States
Duke University Hospital
Durham, North Carolina 27710
United States
Vanderbilt University Medical Center
Nashville, Tennessee 37232
United States
Children's Medical Center, Dallas
Dallas, Texas 75235
United States
University of Texas Health Science Center Medical School at Houston
Houston, Texas 77030
United States
University of Wisconsin-Madison
Madison, Wisconsin 53715
United States
The Children's Hospital at Westmead
Westmead, New South Wales 2145
Australia
Royal Children's Hospital
Parkville, Victoria 3052
Australia
Women's and Children's Health Research Institute, University of Alberta
Edmonton, Alberta T6G 2R3
Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario K1H 8L1
Canada
The Hospital for Sick Children
Toronto, Ontario M5G1X8
Canada
Imagine Institute
Paris, 75015
France
UMC Utrecht
Utrecht, 3584 CX
Netherlands
Unidad de Cirugía Artroscópica (MIKS Hospital)
Vitoria-Gasteiz, Alava 1008
Spain
Hospital Univeristario La Paz
Madrid, Madrid 28046
Spain
Astrid Lindgren Children's Hospital (Karolinska University Hospital)
Stockholm,
Sweden
Great Ormond Street Hospital
London, Greater London WC1N 3JH
United Kingdom
Sheffield Children's Hospital
Sheffield, South Yorkshire S10 2TH
United Kingdom