A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Phase 2
92
about 5.3 years
3–10
10 sites in CA, CO, DE +5
What this study is about
This trial is testing TYRA-300, a medication, to see if it's safe and effective for children with achondroplasia. The goal is to find the best dose of TYRA-300 that can be used in this population.
Simplified from trial records by PatientMatch.
What you may be asked to do
- 1.Take TYRA-300 0.125 mg/kg
- 2.Take TYRA-300 0.25 mg/kg
- 3.Take TYRA-300 0.375 mg/kg
- +1 more
Participation Burden
What's physically and logistically required of participants.
Requires travel to a study site
How treatment is administered
Everyone gets the investigational treatment.
Extracted study details
Pulled from the trial record to show what is being tested and what the study is measuring.
Primary: Change from baseline in annualized growth velocity (Cohort 1), Incidence of treatment-related adverse events as assessed by CTCAE v5.0
Secondary: Change from baseline in annualized growth velocity (Cohort 1), Change from baseline in annualized growth velocity (Cohort 2), Change from baseline in arm span proportionality (arm span/height ratio), Change from baseline in elbow extension, Change from baseline in femur length (cm), Change from baseline in height z-score (Cohort 1), Change from baseline in height z-score (Cohort 2), Change from baseline in sitting height (cm)