A Study of TYRA-300 in Children With Achondroplasia: BEACH301 (NCT06842355) | Clinical Trial Compass
RecruitingPhase 2
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
United States92 participantsStarted 2025-03-04
Plain-language 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.
Who can participate
Age range3 Years – 10 Years
SexALL
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Inclusion Criteria:
* 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 Criteria:
* 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.
What they're measuring
1
Incidence of treatment-related adverse events as assessed by CTCAE v5.0
Timeframe: Up to 12 months
2
Change from baseline in annualized growth velocity (Cohort 1)