NCT06842355

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
92

participants targeted

Target at P50-P75 for phase_2

Timeline
50mo left

Started Mar 2025

Longer than P75 for phase_2

Geographic Reach
4 countries

15 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress22%
Mar 2025Jun 2030

First Submitted

Initial submission to the registry

January 29, 2025

Completed
26 days until next milestone

First Posted

Study publicly available on registry

February 24, 2025

Completed
8 days until next milestone

Study Start

First participant enrolled

March 4, 2025

Completed
4.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2030

Expected
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2030

Last Updated

April 24, 2026

Status Verified

April 1, 2026

Enrollment Period

4.8 years

First QC Date

January 29, 2025

Last Update Submit

April 23, 2026

Conditions

Achondroplasia

Outcome Measures

Primary Outcomes (2)

  • Incidence of treatment-related adverse events as assessed by CTCAE v5.0

    Up to 12 months

  • Change from baseline in annualized growth velocity (Cohort 1)

    12 months

Secondary Outcomes (18)

  • Change from baseline in annualized growth velocity (Cohort 1)

    6 months

  • Change from baseline in height z-score (Cohort 1)

    6 and 12 months

  • Pharmacokinetics: maximum plasma concentration (Cmax)

    15 days

  • Pharmacokinetics: time to reach maximum plasma concentration (Tmax)

    15 days

  • Pharmacokinetics: area under the plasma concentration-time curve (AUC)

    15 days

  • +13 more secondary outcomes

Study Arms (4)

TYRA-300 0.125 mg/kg

EXPERIMENTAL

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

TYRA-300 0.25 mg/kg

EXPERIMENTAL

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

TYRA-300 0.375 mg/kg

EXPERIMENTAL

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

TYRA-300 0.50 mg/kg

EXPERIMENTAL

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

Interventions

TYRA-300 0.125 mg/kgDRUG

Initial dose level of TYRA-300 per protocol, subsequent dose level escalations will occur based on criteria outlined in the protocol.

TYRA-300 0.125 mg/kg
TYRA-300 0.25 mg/kgDRUG

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

TYRA-300 0.25 mg/kg
TYRA-300 0.375 mg/kgDRUG

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

TYRA-300 0.375 mg/kg
TYRA-300 0.50 mg/kgDRUG

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

TYRA-300 0.50 mg/kg

Eligibility Criteria

Age3 Years - 10 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

Lundquist Institute for Biomedical Innovation

Torrance, California, 90502, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Nemours Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, United States

RECRUITING

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

RECRUITING

Uncommon Cures

Chevy Chase, Maryland, 20815, United States

RECRUITING

University of Missouri

Columbia, Missouri, 65201, United States

RECRUITING

Washington University

St Louis, Missouri, 63130, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

Children's Medical Center, Dallas

Dallas, Texas, 75235, United States

RECRUITING

University of Texas Health Science Center Medical School at Houston

Houston, Texas, 77030, United States

RECRUITING

University of Wisconsin-Madison

Madison, Wisconsin, 53715, United States

RECRUITING

The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

RECRUITING

Royal Children's Hospital

Parkville, Victoria, 3052, Australia

RECRUITING

The Hospital for Sick Children

Toronto, Ontario, M5G1X8, Canada

RECRUITING

Unidad de Cirugía Artroscópica (MIKS Hospital)

Vitoria-Gasteiz, Alava, 1008, Spain

RECRUITING

MeSH Terms

Conditions

Achondroplasia

Condition Hierarchy (Ancestors)

DwarfismBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesOsteochondrodysplasiasGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Doug Warner, MD

    Tyra Biosciences

    STUDY CHAIR

Central Study Contacts

Sinette Heys

CONTACT

(619) 728-4805ACH@tyra.bio

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 29, 2025

First Posted

February 24, 2025

Study Start

March 4, 2025

Primary Completion (Estimated)

January 1, 2030

Study Completion (Estimated)

June 1, 2030

Last Updated

April 24, 2026

Record last verified: 2026-04

Locations

AU(2)CA(1)US(11)ES(1)