Menu
Recruiting NCT06842355

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

Phase II Interventional Achondroplasia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: TYRA-300 0.125 mg/kg, TYRA-300 0.25 mg/kg, TYRA-300 0.375 mg/kg, TYRA-300 0.50 mg/kg.
Who it may be relevant to
Registry conditions: Achondroplasia. Basic parameters: 3 years — 10 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia, Canada, France, Netherlands +3
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

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.

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.

Interventions

  • 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.
  • Drug TYRA-300 0.25 mg/kg
    Subsequent dose level escalations will occur based on criteria outlined in the protocol.
  • Drug TYRA-300 0.375 mg/kg
    Subsequent dose level escalations will occur based on criteria outlined in the protocol.
  • Drug TYRA-300 0.50 mg/kg
    Subsequent dose level escalations will occur based on criteria outlined in the protocol.

Primary outcome measures

  • Incidence of treatment-related adverse events as assessed by CTCAE v5.0 [Time frame: Up to 12 months]
  • Change from baseline in annualized growth velocity (Cohort 1) [Time frame: 12 months]
Secondary outcome measures (12)
  • Change from baseline in annualized growth velocity (Cohort 1) [Time frame: 6 months]
  • Change from baseline in height z-score (Cohort 1) [Time frame: 6 and 12 months]
  • Pharmacokinetics: maximum plasma concentration (Cmax) [Time frame: 15 days]
  • Pharmacokinetics: time to reach maximum plasma concentration (Tmax) [Time frame: 15 days]
  • Pharmacokinetics: area under the plasma concentration-time curve (AUC) [Time frame: 15 days]
  • Pharmacokinetics: half-life of TYRA-300 (t1/2) [Time frame: 15 days]
  • Pharmacokinetics: apparent total clearance (CL/F) [Time frame: 15 days]
  • Pharmacokinetics: apparent volume of distribution (Vd/F) [Time frame: 15 days]
  • Change from baseline in annualized growth velocity (Cohort 2) [Time frame: 6 and 12 months]
  • Change from baseline in height z-score (Cohort 2) [Time frame: 6 and 12 months]
  • Change from baseline in standing height (cm) [Time frame: 6 and 12 months]
  • Change from baseline in sitting height (cm) [Time frame: 6 and 12 months]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 12 centers
  • Lundquist Institute for Biomedical Innovation — Torrance
  • Children's Hospital Colorado — Aurora
  • Nemours Alfred I duPont Hospital for Children — Wilmington
  • Johns Hopkins University School of Medicine — Baltimore
  • Uncommon Cures — Chevy Chase
  • University of Missouri — Columbia
  • Washington University — St Louis
  • Duke University Hospital — Durham
  • … and 4 more centers
Canada · 3 centers
  • Women's and Children's Health Research Institute, University of Alberta — Edmonton
  • Children's Hospital of Eastern Ontario — Ottawa
  • The Hospital for Sick Children — Toronto
Australia · 2 centers
  • The Children's Hospital at Westmead — Westmead
  • Royal Children's Hospital — Parkville
Spain · 2 centers
  • Unidad de Cirugía Artroscópica (MIKS Hospital) — Vitoria-Gasteiz
  • Hospital Univeristario La Paz — Madrid
United Kingdom · 2 centers
  • Great Ormond Street Hospital — London
  • Sheffield Children's Hospital — Sheffield
France · 1 center
  • Imagine Institute — Paris
Netherlands · 1 center
  • UMC Utrecht — Utrecht
Sweden · 1 center
  • Astrid Lindgren Children's Hospital (Karolinska University Hospital) — Stockholm

Identifiers

NCT: NCT06842355 · TYR300-201

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗