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
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Astrid Lindgren Children's Hospital (Karolinska University Hospital)
RECRUITINGStockholm, Sweden
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital of Eastern Ontario
RECRUITINGOttawa, Ontario, K1H 8L1, Canada
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Children's Medical Center, Dallas
RECRUITINGDallas, Texas, 75235, United States
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Duke University Hospital
RECRUITINGDurham, North Carolina, 27710, United States
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Great Ormond Street Hospital
RECRUITINGLondon, Greater London, WC1N 3JH, United Kingdom
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Hospital Univeristario La Paz
RECRUITINGMadrid, Madrid, 28046, Spain
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Imagine Institute
RECRUITINGParis, 75015, France
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Johns Hopkins University School of Medicine
RECRUITINGBaltimore, Maryland, 21205, United States
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Lundquist Institute for Biomedical Innovation
RECRUITINGTorrance, California, 90502, United States
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Nemours Alfred I duPont Hospital for Children
RECRUITINGWilmington, Delaware, 19803, United States
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Royal Children's Hospital
RECRUITINGParkville, Victoria, 3052, Australia
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Sheffield Children's Hospital
RECRUITINGSheffield, South Yorkshire, S10 2TH, United Kingdom
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The Children's Hospital at Westmead
RECRUITINGWestmead, New South Wales, 2145, Australia
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The Hospital for Sick Children
RECRUITINGToronto, Ontario, M5G1X8, Canada
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UMC Utrecht
RECRUITINGUtrecht, 3584 CX, Netherlands
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Uncommon Cures
RECRUITINGChevy Chase, Maryland, 20815, United States
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Unidad de Cirugía Artroscópica (MIKS Hospital)
RECRUITINGVitoria-Gasteiz, Alava, 1008, Spain
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University of Missouri
RECRUITINGColumbia, Missouri, 65201, United States
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University of Texas Health Science Center Medical School at Houston
RECRUITINGHouston, Texas, 77030, United States
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University of Wisconsin-Madison
RECRUITINGMadison, Wisconsin, 53715, United States
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
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Washington University
RECRUITINGSt Louis, Missouri, 63130, United States
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Women's and Children's Health Research Institute, University of Alberta
RECRUITINGEdmonton, Alberta, T6G 2R3, Canada
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a weekly shot help children with achondroplasia grow taller?
- Can growth hormone boost height in children with dwarfism?
- Weekly shot may boost growth in kids with dwarfism
- Experimental achondroplasia drug trial halted early
- No treatment, just tracking: PROPEL study watches how kids with achondroplasia grow
- New drug aims to boost height in kids with dwarfism