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

Purpose

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.

Condition

  • Achondroplasia

Eligibility

Eligible Ages
Between 3 Years and 10 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
TYRA-300 0.125 mg/kg 		TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
  • 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.
Experimental
TYRA-300 0.25 mg/kg 		TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
  • Drug: TYRA-300 0.25 mg/kg
    Subsequent dose level escalations will occur based on criteria outlined in the protocol.
Experimental
TYRA-300 0.375 mg/kg 		TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
  • Drug: TYRA-300 0.375 mg/kg
    Subsequent dose level escalations will occur based on criteria outlined in the protocol.
Experimental
TYRA-300 0.50 mg/kg 		TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
  • Drug: TYRA-300 0.50 mg/kg
    Subsequent dose level escalations will occur based on criteria outlined in the protocol.

Recruiting Locations

Lundquist Institute for Biomedical Innovation
Torrance 5403022, California 5332921 90502
Contact:
Adolfo Morales Garval
(310) 222-3472
adolfo.morales@lundquist.org

Children's Hospital Colorado
Aurora 5412347, Colorado 5417618 80045
Contact:
Lauren Samz
720-777-1975
Lauren.Samz@childrenscolorado.org

Nemours Alfred I duPont Hospital for Children
Wilmington 4145381, Delaware 4142224 19803
Contact:
Judith Feinson
(302) 651-5928
judith.feinson@nemours.org

Uncommon Cures
Chevy Chase 4351335, Maryland 4361885 20815
Contact:
Tamanna Roshan Lal
240 858 4938
troshanlal@uncommoncures.com

University of Missouri
Columbia 4381982, Missouri 4398678 65201
Contact:
Taylor Matthews
573-882-4758
mathewsta@health.missouri.edu

Washington University
St Louis 4407066, Missouri 4398678 63130
Contact:
Michelle Ritter
(314) 362-7306
michelle.ritter@wustl.edu

Children's Medical Center, Dallas
Dallas 4684888, Texas 4736286 75235
Contact:
Colton Youngblood
(214) 456-3163
Colten.Youngblood@childrens.com

University of Wisconsin-Madison
Madison 5261457, Wisconsin 5279468 53715
Contact:
Sarah M Heitmeier
(608) 263-8863
sheitmeier@wisc.edu

More Details

NCT ID
NCT06842355
Status
Recruiting
Sponsor
Tyra Biosciences, Inc

Study Contact

Sinette Heys
(619) 728-4805
ACH@tyra.bio

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.

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ResearchMatch is funded in part by the National Institutes of Health (NIH) Clinical and Translational Science Award (CTSA) program, led by the NIH’s National Center for Advancing Translational Sciences (NCATS), grants UL1TR000445 and 1U54RR032646-01, and grant U24TR001579 from NCATS and the National Library of Medicine. The content of this website is solely the responsibility of ResearchMatch and Vanderbilt University and does not necessarily represent the official views of the NIH, NCATS, or NLM.

Vanderbilt University Medical Center