Phase 2 Study of TYRA-300 in Children Ages 3 to 10 With Achondroplasia and Open Growth Plates

What this Trial Is About

Researchers are evaluating the safety, tolerability, and identifying potentially effective doses of TYRA-300 in children aged 3 to 10 years with achondroplasia who have open growth plates. This Phase 2, multicenter, open-label, dose-escalation study includes three groups the Sentinel Safety Cohort, Cohort 1, and Cohort 2, to examine different dosing levels of TYRA-300. TYRA-300 is a selective fibroblast growth factor receptor-3 FGFR3 tyrosine kinase inhibitor aimed at treating this condition. Participants will receive TYRA-300 in sprinkle capsule or mini-tablet form. The total dose is calculated based on the childs weight and adjusted every 3 months. The study involves dose escalation through four dose levels 0.125 mgkg, 0.25 mgkg, 0.375 mgkg, and 0.50 mgkg. Each participant will be assigned to one of these dose groups, and dosing adjustments will be made as per the study protocol. During the study, children will be closely monitored with assessments including measurement of growth velocity, height, limb lengths, and arm span proportionality over 6 to 12 months. Safety is evaluated by tracking treatment-related adverse events. Pharmacokinetic evaluations will also be conducted within the first 15 days. Participants will have regular visits for weight-based dose adjustments and safety monitoring throughout the study duration.

A Study of TYRA-300 in Children With Achondroplasia: BEACH301