Actively Recruiting
A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301
Led by Tyra Biosciences, Inc · Updated on 2026-06-05
92
Participants Needed
17
Research Sites
21 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and potentially effective doses of TYRA-300, a fibroblast growth factor receptor 3 (FGFR3) selective tyrosine kinase inhibitor, in children aged 3 to 10 years with achondroplasia who have open growth plates. This Phase 2, multicenter, open-label dose-escalation study includes three groups of children: the Sentinel Safety Cohort, Cohort 1, and Cohort 2. The study aims to understand how TYRA-300 affects growth velocity and related measurements over time. Participants receive TYRA-300 as sprinkle capsules or mini-tablets, with doses calculated based on each child's weight. Weight adjustments occur every three months to ensure appropriate dosing. The study features multiple dosing levels from 0.125 mg/kg up to 0.50 mg/kg, with dose escalations based on specific protocol criteria. The study spans up to 12 months for primary outcomes, with additional assessments at 6 and 12 months for secondary growth and pharmacokinetic measures. Throughout the study, children will undergo regular evaluations including growth measurements such as height, arm length, tibial and femur length, and arm span proportionality. Safety is monitored by tracking treatment-related adverse events according to established criteria. Pharmacokinetic assessments will measure how the drug is absorbed and processed. The total participation duration includes up to one year of treatment and observation to assess changes in growth and safety outcomes under medical supervision.
CONDITIONS
Brief Title
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 3 to 10 years old (inclusive) at the time of consent.
- Informed consent provided by parent(s) or legal guardian(s).
- Participants 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 naive to prior growth accelerating therapy.
- Cohort 2 only: aged 3 to 10 years old (inclusive) and have received prior growth accelerating therapy.
You will not qualify if you...
- Presence or history of any disease or condition that interferes with study participation or safety evaluations.
- Uncontrolled or untreated conditions that could affect pediatric growth.
- Diagnosis of endocrine condition affecting calcium/phosphate balance.
- Prior limb lengthening surgery or planned limb lengthening during the study.
- Taking medications that strongly inhibit or induce cytochrome P450 3A4.
- History or current corneal or retinal disorder/keratopathy.
- Presence of guided growth hardware or 8 plates.
- Planned or anticipated orthopedic surgeries during the study.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 months
Participants receive TYRA-300 oral medication with dose levels adjusted based on weight every 3 months to determine safety, tolerability, and effective dose.
Regular visits for dosing and assessments throughout treatment
Trial Site Locations
Total: 17 locations
1
Lundquist Institute for Biomedical Innovation
Torrance, California, United States, 90502
Actively Recruiting
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Nemours Alfred I duPont Hospital for Children
Wilmington, Delaware, United States, 19803
Actively Recruiting
4
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
Actively Recruiting
5
Uncommon Cures
Chevy Chase, Maryland, United States, 20815
Actively Recruiting
6
University of Missouri
Columbia, Missouri, United States, 65201
Actively Recruiting
7
Washington University
St Louis, Missouri, United States, 63130
Actively Recruiting
8
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
9
Children's Medical Center, Dallas
Dallas, Texas, United States, 75235
Actively Recruiting
10
University of Texas Health Science Center Medical School at Houston
Houston, Texas, United States, 77030
Actively Recruiting
11
University of Wisconsin-Madison
Madison, Wisconsin, United States, 53715
Actively Recruiting
12
The Children's Hospital at Westmead
Westmead, New South Wales, Australia, 2145
Actively Recruiting
13
Royal Children's Hospital
Parkville, Victoria, Australia, 3052
Actively Recruiting
14
Women's and Children's Health Research Institute, University of Alberta
Edmonton, Alberta, Canada, T6G 2R3
Actively Recruiting
15
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
16
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G1X8
Actively Recruiting
17
Unidad de Cirugía Artroscópica (MIKS Hospital)
Vitoria-Gasteiz, Alava, Spain, 1008
Actively Recruiting
Research Team
S
Sinette Heys
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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