A Study of TYRA-300 in Children With Achondroplasia: BEACH301
A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301
Tyra Biosciences, Inc
92 participants
Mar 4, 2025
INTERVENTIONAL
Conditions
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
Inclusion Criteria9
- 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 Criteria6
- 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 pediatric growth.
- Diagnosis of endocrine condition that alters calcium/phosphate homeostasis.
- Prior limb lengthening surgery or planned or expected to have limb lengthening surgery while enrolled in the study.
- Taking medications that are strong inhibitors or inducers of cytochrome P450 (Cyp) 3A4.
- History or current evidence of corneal or retinal disorder/keratopathy.
- Presence of guided growth hardware/8 plates. Planned or anticipated orthopedic surgeries.
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Interventions
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.
Locations(15)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06842355