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NCT06842355PHASE2Recruiting

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

Tyra Biosciences, Inc

Start Date

3/4/2025

Completion Date

6/1/2030

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.

Detailed Description

This is a Phase 2, multicenter, open-label, dose-escalation study to determine the safety, tolerability, and identify potentially effective dose(s) of TYRA-300, a fibroblast growth factor receptor (FGFR)-3 selective tyrosine kinase inhibitor, in children 3 to 10 years of age with achondroplasia with open growth plates that will examine three cohorts of children: the Sentinel Safety Cohort, Cohort 1, and Cohort 2.

Eligibility Criteria

Age Range: 3 years to 10 years

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.

Interventions

DRUG

TYRA-300 0.125 mg/kg

DRUG

TYRA-300 0.25 mg/kg

DRUG

TYRA-300 0.375 mg/kg

DRUG

TYRA-300 0.50 mg/kg

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Conditions

Achondroplasia

Locations

Lundquist Institute for Biomedical Innovation

Torrance, California 90502

United States

Children's Hospital Colorado

Aurora, Colorado 80045

United States

Nemours Alfred I duPont Hospital for Children

Wilmington, Delaware 19803

United States

Johns Hopkins University School of Medicine

Baltimore, Maryland 21205

United States

Uncommon Cures

Chevy Chase, Maryland 20815

United States

University of Missouri

Columbia, Missouri 65201

United States

Washington University

St Louis, Missouri 63130

United States

Duke University Hospital

Durham, North Carolina 27710

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232

United States

Children's Medical Center, Dallas

Dallas, Texas 75235

United States

University of Texas Health Science Center Medical School at Houston

Houston, Texas 77030

United States

University of Wisconsin-Madison

Madison, Wisconsin 53715

United States

The Children's Hospital at Westmead

Westmead, New South Wales 2145

Australia

Royal Children's Hospital

Parkville, Victoria 3052

Australia

Women's and Children's Health Research Institute, University of Alberta

Edmonton, Alberta T6G 2R3

Canada

Children's Hospital of Eastern Ontario

Ottawa, Ontario K1H 8L1

Canada

The Hospital for Sick Children

Toronto, Ontario M5G1X8

Canada

Imagine Institute

Paris, 75015

France

UMC Utrecht

Utrecht, 3584 CX

Netherlands

Unidad de Cirugía Artroscópica (MIKS Hospital)

Vitoria-Gasteiz, Alava 1008

Spain

Hospital Univeristario La Paz

Madrid, Madrid 28046

Spain

Astrid Lindgren Children's Hospital (Karolinska University Hospital)

Stockholm,

Sweden

Great Ormond Street Hospital

London, Greater London WC1N 3JH

United Kingdom

Sheffield Children's Hospital

Sheffield, South Yorkshire S10 2TH

United Kingdom