TYRA-300 for Achondroplasia
Trial Summary
Will I have to stop taking my current medications?
The trial requires that participants do not take medications that strongly affect a specific liver enzyme (cytochrome P450 3A4). If your current medications do not fall into this category, you may not need to stop them.
How does the drug TYRA-300 differ from other treatments for achondroplasia?
TYRA-300 is unique because it is a tyrosine kinase inhibitor (a type of drug that blocks certain enzymes) targeting the FGFR3 gene mutation, which is the cause of achondroplasia. This approach is different from other treatments like vosoritide, which aims to increase growth velocity, as TYRA-300 directly addresses the genetic mutation responsible for the condition.12345
What is the purpose of this trial?
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.
Research Team
Doug Warner, MD
Principal Investigator
Tyra Biosciences
Eligibility Criteria
This trial is for children aged 3 to 10 with a confirmed diagnosis of achondroplasia, which is a bone growth disorder. They must have open growth plates, be able to walk and take oral medication independently. Consent from parents or guardians is required, and the child's willingness to assent if applicable.Inclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Dose-Escalation
Participants receive TYRA-300 in a dose-escalation format to determine safety and tolerability
Dose-Expansion
Participants continue to receive TYRA-300 to identify potentially effective dose(s)
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- TYRA-300
Find a Clinic Near You
Who Is Running the Clinical Trial?
Tyra Biosciences, Inc
Lead Sponsor