New pill could help kids with dwarfism grow – but early days
NCT ID NCT06842355
First seen Jun 24, 2026 · Last updated Jul 01, 2026 · Updated 3 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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Astrid Lindgren Children's Hospital (Karolinska University Hospital)
RECRUITINGStockholm, Sweden
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Eastern Ontario
RECRUITINGOttawa, Ontario, K1H 8L1, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Medical Center, Dallas
RECRUITINGDallas, Texas, 75235, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Johns Hopkins University School of Medicine
RECRUITINGBaltimore, Maryland, 21205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Lundquist Institute for Biomedical Innovation
RECRUITINGTorrance, California, 90502, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Nemours Alfred I duPont Hospital for Children
RECRUITINGWilmington, Delaware, 19803, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Children's Hospital
RECRUITINGParkville, Victoria, 3052, Australia
Contact Email: •••••@•••••
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The Children's Hospital at Westmead
RECRUITINGWestmead, New South Wales, 2145, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Hospital for Sick Children
RECRUITINGToronto, Ontario, M5G1X8, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Uncommon Cures
RECRUITINGChevy Chase, Maryland, 20815, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Unidad de Cirugía Artroscópica (MIKS Hospital)
RECRUITINGVitoria-Gasteiz, Alava, 1008, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Missouri
RECRUITINGColumbia, Missouri, 65201, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Texas Health Science Center Medical School at Houston
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Wisconsin-Madison
RECRUITINGMadison, Wisconsin, 53715, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Washington University
RECRUITINGSt Louis, Missouri, 63130, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Women's and Children's Health Research Institute, University of Alberta
RECRUITINGEdmonton, Alberta, T6G 2R3, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••