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

Description

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.

Conditions

Achondroplasia

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Achondroplasia
Intervention / Treatment

-

Contacts and Locations

Kissimmee

Rare Disease Research, Kissimmee, Florida, United States, 34746

Atlanta

Rare Disease Research, Atlanta, Georgia, United States, 30329

Hillsborough

Rare Disease Research, Hillsborough, North Carolina, United States, 27278

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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.

Ages Eligible for Study

3 Years to 10 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Tyra Biosciences, Inc,

Doug Warner, MD, STUDY_CHAIR, Tyra Biosciences

Study Record Dates

2030-06