A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Trial Parameters
Brief 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.
Eligibility Criteria
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: aged 3 to 10 years old (inclusive) and are naive to prior growth accelerating therapy. * Cohort 2 only: aged 3 to 10 years old (inclusive) and have received prior growth accelerating therapy. Exclusion Criteria: * Presence or history of any concurrent disease or condition that would interfere with study participation, safety evaluations, or any uncontrolled or untreated condition that could impact pe