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

RecruitingPhase 2Ages 3 Years - 10 YearsAllTyra Biosciences, Inc
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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.
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What it tests

  • TYRA-300 0.125 mg/kg
  • TYRA-300 0.25 mg/kg
  • TYRA-300 0.375 mg/kg
  • TYRA-300 0.50 mg/kg
  • TYRA-300 0.625 mg/kg
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Where

25 sites
Lundquist Institute for Biomedical Innovation
Torrance, United States
Children's Hospital Colorado
Aurora, United States
Nemours Alfred I duPont Hospital for Children
Wilmington, United States
Johns Hopkins University School of Medicine
Baltimore, United States
Uncommon Cures
Chevy Chase, United States
University of Missouri
Columbia, United States
Washington University
St Louis, United States
Duke University Hospital
Durham, United States

โ€ฆand 17 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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