Evaluating TYRA-300 for children with achondroplasia

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

This study is testing a new medication called TYRA-300 to see if it is safe and helps children aged 3 to 10 with achondroplasia grow better.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 92 (estimated)
• Ages: 3 Years to 10 Years
• Sex: All
• Sponsor: Tyra Biosciences, Inc (Industry-sponsored)
• Locations: 15 sites (Torrance, California and 14 other locations)
• Trial ID: NCT06842355 on ClinicalTrials.gov

What this trial studies

This Phase 2, multicenter, open-label study aims to assess the safety and tolerability of TYRA-300, a selective FGFR-3 tyrosine kinase inhibitor, in children aged 3 to 10 years with achondroplasia and open growth plates. The study will involve a dose-escalation approach across three cohorts: a Sentinel Safety Cohort, Cohort 1 for treatment-naive participants, and Cohort 2 for those who have previously received growth accelerating therapy. Participants will receive varying doses of TYRA-300 to identify potentially effective dosages.

Who should consider this trial

Good fit: Ideal candidates are children aged 3 to 10 years with a confirmed diagnosis of achondroplasia and open growth plates.

Not a fit: Patients with concurrent diseases that could interfere with study participation or those who have closed growth plates may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option to enhance growth and improve quality of life for children with achondroplasia.

How similar studies have performed: While this approach is novel, similar studies targeting FGFR-3 pathways have shown promise in other contexts.

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.

Where this trial is running

Torrance, California and 14 other locations

• Lundquist Institute for Biomedical Innovation — Torrance, California, United States (Recruiting)
• Children's Hospital Colorado — Aurora, Colorado, United States (Recruiting)
• Nemours Alfred I duPont Hospital for Children — Wilmington, Delaware, United States (Recruiting)
• Johns Hopkins University School of Medicine — Baltimore, Maryland, United States (Recruiting)
• Uncommon Cures — Chevy Chase, Maryland, United States (Recruiting)
• University of Missouri — Columbia, Missouri, United States (Recruiting)
• Washington University — St Louis, Missouri, United States (Recruiting)
• Vanderbilt University Medical Center — Nashville, Tennessee, United States (Recruiting)
• Children's Medical Center, Dallas — Dallas, Texas, United States (Recruiting)
• University of Texas Health Science Center Medical School at Houston — Houston, Texas, United States (Recruiting)
• University of Wisconsin-Madison — Madison, Wisconsin, United States (Recruiting)
• The Children's Hospital at Westmead — Westmead, New South Wales, Australia (Recruiting)
• Royal Children's Hospital — Parkville, Victoria, Australia (Recruiting)
• The Hospital for Sick Children — Toronto, Ontario, Canada (Recruiting)
• Unidad de Cirugía Artroscópica (MIKS Hospital) — Vitoria-Gasteiz, Alava, Spain (Recruiting)

Study contacts

• Study coordinator: Sinette Heys

Conditions Achondroplasia