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

June 3, 2026 updated by: Tyra Biosciences, Inc

A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

92

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Sinette Heys
  • Phone Number: (619) 728-4805
  • Email: ACH@tyra.bio

Study Locations

  • Australia
    • New South Wales
      • Westmead, New South Wales, Australia, 2145
    • Victoria
      • Parkville, Victoria, Australia, 3052
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2R3
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L1
        • Recruiting
        • Children's Hospital of Eastern Ontario
        • Contact:
      • Toronto, Ontario, Canada, M5G1X8
        • Recruiting
        • The Hospital for Sick Children
        • Contact:
  • Spain
    • Alava
      • Vitoria-Gasteiz, Alava, Spain, 1008
        • Recruiting
        • Unidad de Cirugía Artroscópica (MIKS Hospital)
        • Contact:
  • United States
    • California
      • Torrance, California, United States, 90502
        • Recruiting
        • Lundquist Institute for Biomedical Innovation
        • Contact:
    • Colorado
      • Aurora, Colorado, United States, 80045
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Recruiting
        • Nemours Alfred I duPont Hospital for Children
        • Contact:
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Recruiting
        • Johns Hopkins University School of Medicine
        • Contact:
      • Chevy Chase, Maryland, United States, 20815
    • Missouri
      • Columbia, Missouri, United States, 65201
      • St Louis, Missouri, United States, 63130
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University Medical Center
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75235
      • Houston, Texas, United States, 77030
        • Recruiting
        • University of Texas Health Science Center Medical School at Houston
        • Contact:
    • Wisconsin
      • Madison, Wisconsin, United States, 53715
        • Recruiting
        • University of Wisconsin-Madison
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TYRA-300 0.125 mg/kg
TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
Drug: 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.
Experimental: TYRA-300 0.25 mg/kg
TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
Drug: TYRA-300 0.25 mg/kg
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
Experimental: TYRA-300 0.375 mg/kg
TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
Drug: TYRA-300 0.375 mg/kg
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
Experimental: TYRA-300 0.50 mg/kg
TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
Drug: TYRA-300 0.50 mg/kg
Subsequent dose level escalations will occur based on criteria outlined in the protocol.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment-related adverse events as assessed by CTCAE v5.0
Time Frame: Up to 12 months
Up to 12 months
Change from baseline in annualized growth velocity (Cohort 1)
Time Frame: 12 months
12 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in annualized growth velocity (Cohort 1)
Time Frame: 6 months
6 months
Change from baseline in height z-score (Cohort 1)
Time Frame: 6 and 12 months
6 and 12 months
Pharmacokinetics: maximum plasma concentration (Cmax)
Time Frame: 15 days
15 days
Pharmacokinetics: time to reach maximum plasma concentration (Tmax)
Time Frame: 15 days
15 days
Pharmacokinetics: area under the plasma concentration-time curve (AUC)
Time Frame: 15 days
15 days
Pharmacokinetics: half-life of TYRA-300 (t1/2)
Time Frame: 15 days
15 days
Pharmacokinetics: apparent total clearance (CL/F)
Time Frame: 15 days
15 days
Pharmacokinetics: apparent volume of distribution (Vd/F)
Time Frame: 15 days
15 days
Change from baseline in annualized growth velocity (Cohort 2)
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in height z-score (Cohort 2)
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in standing height (cm)
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in sitting height (cm)
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in upper and lower arm length (cm)
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in tibial length (cm)
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in femur length (cm)
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in arm span proportionality (arm span/height ratio)
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in upper segment/lower segment ratio
Time Frame: 6 and 12 months
6 and 12 months
Change from baseline in elbow extension
Time Frame: 6 and 12 months
6 and 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Tyra Biosciences, Inc

Investigators

  • Study Chair: Doug Warner, MD, Tyra Biosciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 4, 2025

Primary Completion (Estimated)

January 1, 2030

Study Completion (Estimated)

June 1, 2030

Study Registration Dates

First Submitted

January 29, 2025

First Submitted That Met QC Criteria

February 18, 2025

First Posted (Actual)

February 24, 2025

Study Record Updates

Last Update Posted (Actual)

June 5, 2026

Last Update Submitted That Met QC Criteria

June 3, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TYR300-201

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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