Recruiting
Phase 2

TYRA-300

Sponsor:

Tyra Biosciences, Inc

Code:

NCT06842355

Conditions

Achondroplasia

Eligibility Criteria

Sex: All

Age: 3 - 10

Healthy Volunteers: Not accepted

Interventions

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

Study Details

Brief summary:

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.

Conditions

Achondroplasia

Study ID

NCT06842355

Start date

Mar 4, 2025

Status verified date

Aug, 2026

Completion date

Jun, 2030

Anticipated

Primary completion date

Jan, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 3 - 10

Healthy Volunteers: Not accepted

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

Enrollment

92 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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.

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.

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.

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.

experimental: TYRA-300 0.625 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.

Interventions

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.

TYRA-300 0.25 mg/kg

Subsequent dose level escalations will occur based on criteria outlined in the protocol.

TYRA-300 0.375 mg/kg

Subsequent dose level escalations will occur based on criteria outlined in the protocol.

TYRA-300 0.50 mg/kg

Subsequent dose level escalations will occur based on criteria outlined in the protocol.

TYRA-300 0.625 mg/kg

Subsequent dose level escalations may occur based on criteria outlined in the protocol.

Primary outcome measure

  • 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 ]

Central Contacts and Locations

Central contacts

Suzanne Lopez

(619) 728-4805ACH@tyra.bio

Locations

Lundquist Institute for Biomedical Innovation

Recruiting

Torrance, California, United States, 90502

Contacts

Adolfo Morales Garval

(310) 222-3472adolfo.morales@lundquist.org

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Lauren Samz

720-777-1975Lauren.Samz@childrenscolorado.org

Nemours Alfred I duPont Hospital for Children

Recruiting

Wilmington, Delaware, United States, 19803

Contacts

Judith Feinson

(302) 651-5928judith.feinson@nemours.org

Johns Hopkins University School of Medicine

Recruiting

Baltimore, Maryland, United States, 21205

Contacts

Plamena Simeonova, MD

410-614-0977psimeon1@jh.edu

Uncommon Cures

Recruiting

Chevy Chase, Maryland, United States, 20815

Contacts

Tamanna Roshan Lal

240 858 4938troshanlal@uncommoncures.com

University of Missouri

Recruiting

Columbia, Missouri, United States, 65201

Contacts

Taylor Matthews

573-882-4758mathewsta@health.missouri.edu

Washington University

Recruiting

St Louis, Missouri, United States, 63130

Contacts

Michelle Ritter

(314) 362-7306michelle.ritter@wustl.edu

Duke University Hospital

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Stephanie Bryant

(919) 668-4777stephanie.l.bryant@duke.edu

Vanderbilt University Medical Center

Recruiting

Nashville, Tennessee, United States, 37232

Contacts

LeeAnna Melton

(615) 322-7601leeanna.melton@vumc.org

Children's Medical Center, Dallas

Recruiting

Dallas, Texas, United States, 75235

Contacts

Colton Youngblood

(214) 456-3163Colten.Youngblood@childrens.com

University of Texas Health Science Center Medical School at Houston

Recruiting

Houston, Texas, United States, 77030

Contacts

Heather Saavedra

(713) 500-7098heather.saavedra@uth.tmc.edu

University of Wisconsin-Madison

Recruiting

Madison, Wisconsin, United States, 53715

Contacts

Sarah M Heitmeier

(608) 263-8863sheitmeier@wisc.edu

Women's and Children's Health Research Institute, University of Alberta

Recruiting

Edmonton, Alberta, Canada, T6G 2R3

Contacts

Lee-Ann Carroll

(780) 248-5616lee-ann.carroll@albertahealthservices.ca

Children's Hospital of Eastern Ontario

Recruiting

Ottawa, Ontario, Canada, K1H 8L1

Contacts

Nasrin Khan

(613) 737-7600 ext 4119NaKhan@cheo.on.ca

The Hospital for Sick Children

Recruiting

Toronto, Ontario, Canada, M5G1X8

Contacts

Tony Tan

(416) 813 7654tony.tan@sickkids.ca

More Information

Sponsor

Tyra Biosciences, Inc

Last update posted

Aug 31, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Tyra Biosciences, Inc on 2026-08-31.