Tyra Biosciences, Inc
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.
Inclusion Criteria: * Aged 3 to 10 years old (inclusive) at the time of consent. * Informed consent provided by parent(s) or legal guardian(s). As study participants are less than 18 years old, participants are willing and able to provide written assent (where applicable and required). * Molecular diagnosis of achondroplasia (FGFR3 G380R). * Radiographically confirmed open growth plates at Screening, as determined by bone age X-ray. * Able to stand and ambulate independently. * Able to take oral medication. * Sentinel Safety Cohort only: aged 5 to 10 years old (inclusive). * Cohort 1 only: ag…
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.
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
Subsequent dose level escalations may occur based on criteria outlined in the protocol.