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

Last updated: March 26, 2025
Sponsor: Tyra Biosciences, Inc
Overall Status: Active - Recruiting

Phase

2

Condition

Severe Short Stature

Treatment

TYRA-300 0.375 mg/kg

TYRA-300 0.50 mg/kg

TYRA-300 0.25 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 0.375 mg/kg
Phase: 2
Study Start date:
March 01, 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

  • Rare Disease Research

    Kissimmee, Florida 34746
    United States

    Site Not Available

  • Rare Disease Research

    Atlanta, Georgia 30329
    United States

    Active - Recruiting

  • Rare Disease Research

    Hillsborough, North Carolina 27278
    United States

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.