A Study of TYRA-300 in Children With Achondroplasia: BEACH301
ConditionAchondroplasia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age3-10
SponsorTyra Biosciences, Inc
About 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.
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- TYRA-300 0.125 mg/kg
- TYRA-300 0.25 mg/kg
- TYRA-300 0.375 mg/kg
- TYRA-300 0.50 mg/kg
Treatment groups
92 Participants
are divided into 4 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov