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

NCT ID NCT06842355

First seen Jun 24, 2026 · Last updated Jul 31, 2026 · Updated 9 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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

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

  • 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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