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New pill could help kids with dwarfism grow – but early days

NCT ID NCT06842355

Disease control Sponsor: Tyra Biosciences, Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Sep 01, 2026 · Updated 13 times

Summary

This study tests a daily oral drug called TYRA-300 in 92 children aged 3–10 with achondroplasia (a common form of dwarfism). The goal is to see if it safely increases their growth rate. Researchers will try different doses to find the best one. It's a Phase 2 trial, so it's still early and not yet proven to work.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
TYRA-300 (an oral drug taken daily)
What this could lead to
If successful, this could lead to a daily pill that helps children with achondroplasia grow taller, potentially reducing some health complications.
What could go wrong
This is an early Phase 2 trial with only 92 children, so results may not apply to everyone. The drug may cause side effects or fail to improve growth significantly.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 92 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2025

Expected to finish

Jun 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 10 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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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Conditions

The condition(s) this trial relates to.

achondroplasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Achondroplasia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    25 sites in 8 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Astrid Lindgren Children's Hospital (Karolinska University Hospital)

    RECRUITING

    Stockholm, Sweden

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Eastern Ontario

    RECRUITING

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's Medical Center, Dallas

    RECRUITING

    Dallas, Texas, 75235, United States

  • Duke University Hospital

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Great Ormond Street Hospital

    RECRUITING

    London, Greater London, WC1N 3JH, United Kingdom

  • Guy's & St Thomas NHS Foundation Trust 2

    RECRUITING

    London, Greater London, SE1 7EH, United Kingdom

  • Hospital Univeristario La Paz

    RECRUITING

    Madrid, Madrid, 28046, Spain

  • Imagine Institute

    RECRUITING

    Paris, 75015, France

  • Johns Hopkins University School of Medicine

    RECRUITING

    Baltimore, Maryland, 21205, United States

  • Lundquist Institute for Biomedical Innovation

    RECRUITING

    Torrance, California, 90502, United States

  • Nemours Alfred I duPont Hospital for Children

    RECRUITING

    Wilmington, Delaware, 19803, United States

  • Royal Children's Hospital

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • Sheffield Children's Hospital

    RECRUITING

    Sheffield, South Yorkshire, S10 2TH, United Kingdom

  • The Children's Hospital at Westmead

    RECRUITING

    Westmead, New South Wales, 2145, Australia

  • The Hospital for Sick Children

    RECRUITING

    Toronto, Ontario, M5G1X8, Canada

  • UMC Utrecht

    RECRUITING

    Utrecht, 3584 CX, Netherlands

  • Uncommon Cures

    RECRUITING

    Chevy Chase, Maryland, 20815, United States

  • Unidad de Cirugía Artroscópica (MIKS Hospital)

    RECRUITING

    Vitoria-Gasteiz, Alava, 1008, Spain

  • University of Missouri

    RECRUITING

    Columbia, Missouri, 65201, United States

  • University of Texas Health Science Center Medical School at Houston

    RECRUITING

    Houston, Texas, 77030, United States

  • University of Wisconsin-Madison

    RECRUITING

    Madison, Wisconsin, 53715, United States

  • Vanderbilt University Medical Center

    RECRUITING

    Nashville, Tennessee, 37232, United States

  • Washington University

    RECRUITING

    St Louis, Missouri, 63130, United States

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

    RECRUITING

    Edmonton, Alberta, T6G 2R3, Canada

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