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.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
92
Initial dose level of TYRA-300 per protocol, subsequent dose level escalations will occur based on criteria outlined in the protocol.
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
Lundquist Institute for Biomedical Innovation
Torrance, California, United States
RECRUITINGIncidence of treatment-related adverse events as assessed by CTCAE v5.0
Time frame: Up to 12 months
Change from baseline in annualized growth velocity (Cohort 1)
Time frame: 12 months
Change from baseline in annualized growth velocity (Cohort 1)
Time frame: 6 months
Change from baseline in height z-score (Cohort 1)
Time frame: 6 and 12 months
Pharmacokinetics: maximum plasma concentration (Cmax)
Time frame: 15 days
Pharmacokinetics: time to reach maximum plasma concentration (Tmax)
Time frame: 15 days
Pharmacokinetics: area under the plasma concentration-time curve (AUC)
Time frame: 15 days
Pharmacokinetics: half-life of TYRA-300 (t1/2)
Time frame: 15 days
Pharmacokinetics: apparent total clearance (CL/F)
Time frame: 15 days
Pharmacokinetics: apparent volume of distribution (Vd/F)
Time frame: 15 days
Change from baseline in annualized growth velocity (Cohort 2)
Time frame: 6 and 12 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
Children's Hospital Colorado
Aurora, Colorado, United States
RECRUITINGNemours Alfred I duPont Hospital for Children
Wilmington, Delaware, United States
RECRUITINGJohns Hopkins University School of Medicine
Baltimore, Maryland, United States
RECRUITINGUncommon Cures
Chevy Chase, Maryland, United States
RECRUITINGUniversity of Missouri
Columbia, Missouri, United States
RECRUITINGWashington University
St Louis, Missouri, United States
RECRUITINGVanderbilt University Medical Center
Nashville, Tennessee, United States
RECRUITINGChildren's Medical Center, Dallas
Dallas, Texas, United States
RECRUITINGUniversity of Texas Health Science Center Medical School at Houston
Houston, Texas, United States
RECRUITING...and 7 more locations
Change from baseline in height z-score (Cohort 2)
Time frame: 6 and 12 months
Change from baseline in standing height (cm)
Time frame: 6 and 12 months
Change from baseline in sitting height (cm)
Time frame: 6 and 12 months
Change from baseline in upper and lower arm length (cm)
Time frame: 6 and 12 months
Change from baseline in tibial length (cm)
Time frame: 6 and 12 months
Change from baseline in femur length (cm)
Time frame: 6 and 12 months
Change from baseline in arm span proportionality (arm span/height ratio)
Time frame: 6 and 12 months
Change from baseline in upper segment/lower segment ratio
Time frame: 6 and 12 months
Change from baseline in elbow extension
Time frame: 6 and 12 months