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

Last updated: June 27, 2026
Sponsor: Tyra Biosciences, Inc
Overall Status: Active - Recruiting

Phase

2

Condition

Severe Short Stature

Treatment

TYRA-300 0.50 mg/kg

TYRA-300 0.25 mg/kg

TYRA-300 0.375 mg/kg

Clinical Study ID

NCT06842355
TYR300-201
  • Ages 3-10
  • All Genders

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 Criteria

Inclusion

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 participantsare less than 18 years old, participants are willing and able to provide writtenassent (where applicable and required).

  • Molecular diagnosis of achondroplasia (FGFR3 G380R).

  • Radiographically confirmed open growth plates at Screening, as determined by boneage 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 growthaccelerating therapy.

  • Cohort 2 only: aged 3 to 10 years old (inclusive) and have received prior growthaccelerating therapy.

Exclusion

Exclusion Criteria:

  • Presence or history of any concurrent disease or condition that would interfere withstudy participation, safety evaluations, or any uncontrolled or untreated conditionthat could impact pediatric growth.

  • Diagnosis of endocrine condition that alters calcium/phosphate homeostasis.

  • Prior limb lengthening surgery or planned or expected to have limb lengtheningsurgery 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 orthopedicsurgeries.

Study Design

Total Participants: 92
Treatment Group(s): 4
Primary Treatment: TYRA-300 0.50 mg/kg
Phase: 2
Study Start date:
March 04, 2025
Estimated Completion Date:
June 30, 2030

Study 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.

Connect with a study center

  • The Children's Hospital at Westmead

    Westmead, New South Wales 2145
    Australia

    Active - Recruiting

  • Royal Children's Hospital

    Parkville, Victoria 3052
    Australia

    Active - Recruiting

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

    Edmonton, Alberta T6G 2R3
    Canada

    Active - Recruiting

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario K1H 8L1
    Canada

    Active - Recruiting

  • The Hospital for Sick Children

    Toronto, Ontario M5G1X8
    Canada

    Active - Recruiting

  • Unidad de Cirugía Artroscópica (MIKS Hospital)

    Vitoria-Gasteiz, Alava 1008
    Spain

    Active - Recruiting

  • Astrid Lindgren Children's Hospital (Karolinska University Hospital)

    Stockholm,
    Sweden

    Active - Recruiting

  • Lundquist Institute for Biomedical Innovation

    Torrance, California 90502
    United States

    Active - Recruiting

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • Nemours Alfred I duPont Hospital for Children

    Wilmington, Delaware 19803
    United States

    Active - Recruiting

  • Johns Hopkins University School of Medicine

    Baltimore, Maryland 21205
    United States

    Active - Recruiting

  • Uncommon Cures

    Chevy Chase, Maryland 20815
    United States

    Active - Recruiting

  • University of Missouri

    Columbia, Missouri 65201
    United States

    Active - Recruiting

  • Washington University

    St Louis, Missouri 63130
    United States

    Active - Recruiting

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37232
    United States

    Active - Recruiting

  • Children's Medical Center, Dallas

    Dallas, Texas 75235
    United States

    Active - Recruiting

  • University of Texas Health Science Center Medical School at Houston

    Houston, Texas 77030
    United States

    Active - Recruiting

  • University of Wisconsin-Madison

    Madison, Wisconsin 53715
    United States

    Active - Recruiting

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