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Clinical Trials in the UK / NCT06842355
Recruiting Phase 2

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

NCT06842355 · tracked via the Priya Life Science UK tracker
Sponsor
Tyra Biosciences, Inc
Phase
Phase 2
Started
2025-03-04
Last updated
2026-08-31

Condition(s) studied

Achondroplasia

Investigational drug(s) / intervention(s)

TYRA-300 0.125 mg/kgTYRA-300 0.25 mg/kgTYRA-300 0.375 mg/kgTYRA-300 0.50 mg/kgTYRA-300 0.625 mg/kg

TYRA-300 0.125 mg/kg: Initial dose level of TYRA-300 per protocol, subsequent dose level escalations will occur based on criteria outlined in the protocol.

TYRA-300 0.25 mg/kg: Subsequent dose level escalations will occur based on criteria outlined in the protocol.

TYRA-300 0.375 mg/kg: Subsequent dose level escalations will occur based on criteria outlined in the protocol.

TYRA-300 0.50 mg/kg: Subsequent dose level escalations will occur based on criteria outlined in the protocol.

TYRA-300 0.625 mg/kg: Subsequent dose level escalations may occur based on criteria outlined in the protocol.

Study 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

Sex
ALL
Min age
3 Years
Max age
10 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (25)

FacilityCityRegionStatus
Lundquist Institute for Biomedical Innovation Torrance California Recruiting
Children's Hospital Colorado Aurora Colorado Recruiting
Nemours Alfred I duPont Hospital for Children Wilmington Delaware Recruiting
Johns Hopkins University School of Medicine Baltimore Maryland Recruiting
Uncommon Cures Chevy Chase Maryland Recruiting
University of Missouri Columbia Missouri Recruiting
Washington University St Louis Missouri Recruiting
Duke University Hospital Durham North Carolina Recruiting
Vanderbilt University Medical Center Nashville Tennessee Recruiting
Children's Medical Center, Dallas Dallas Texas Recruiting
University of Texas Health Science Center Medical School at Houston Houston Texas Recruiting
University of Wisconsin-Madison Madison Wisconsin Recruiting
The Children's Hospital at Westmead Westmead New South Wales Recruiting
Royal Children's Hospital Parkville Victoria Recruiting
Women's and Children's Health Research Institute, University of Alberta Edmonton Alberta Recruiting
Children's Hospital of Eastern Ontario Ottawa Ontario Recruiting
The Hospital for Sick Children Toronto Ontario Recruiting
Imagine Institute Paris France Recruiting
UMC Utrecht Utrecht Netherlands Recruiting
Unidad de Cirugía Artroscópica (MIKS Hospital) Vitoria-Gasteiz Alava Recruiting
Hospital Univeristario La Paz Madrid Madrid Recruiting
Astrid Lindgren Children's Hospital (Karolinska University Hospital) Stockholm Sweden Recruiting
Guy's & St Thomas NHS Foundation Trust 2 London Greater London Recruiting
Great Ormond Street Hospital London Greater London Recruiting
Sheffield Children's Hospital Sheffield South Yorkshire Recruiting

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06842355 on ClinicalTrials.gov ↗ ← All trials in the UK