A Study of TYRA-300 in Children With Achondroplasia: BEACH301

Launched by TYRA BIOSCIENCES, INC · Feb 18, 2025

Keywords

ClinConnect Summary

This clinical trial, called BEACH301, is looking at a new treatment called TYRA-300 for children with achondroplasia, a common form of dwarfism. The main goal is to see if TYRA-300 is safe and how well it works in children aged 3 to 10 who have open growth plates, which means their bones are still growing. The study is currently recruiting participants, and it is open to all genders.

To qualify for the trial, children need to be between 3 and 10 years old and have a confirmed diagnosis of achondroplasia. Parents or guardians must give consent, and children may need to provide their own written agreement if required. Participants will need to be able to stand, walk, and take medicine by mouth. Throughout the study, children will receive the new treatment and will be monitored for safety and effectiveness. It's important to note that certain health conditions or previous surgeries may prevent a child from participating, so a careful screening will take place. This trial offers a chance to explore a potential new treatment that may help children with achondroplasia grow in a healthier way.

Gender

ALL

Eligibility criteria

• 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.