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Experimental pill aims to stop extra bone growth in rare disease
NCT ID NCT05039515
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tested two doses of an oral drug called fidrisertib (IPN60130) in children and adults with fibrodysplasia ossificans progressiva (FOP), a rare condition where soft tissues turn into bone. The goal was to see if the drug could reduce new abnormal bone growth compared to a placebo. The study was terminated early, so full results are not available.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
fidrisertib (IPN60130)
What this could lead to
If it works, fidrisertib could slow or prevent the buildup of extra bone in FOP, helping preserve movement and reduce disability.
What could go wrong
The trial was terminated early, so results are limited. FOP is rare and complex, and what works in a small study may not help everyone.
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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Locations
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Aichi Children's Health and Medical Center
Ōbu, Japan
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Children's Hospital Capital Institute of Pediatrics (CIP)
Beijing, China
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Groupe Hospitalier Necker Enfants Malades
Paris, 75015, France
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Hopital Lariboisiere
Paris, France
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Hospital Center Lisbon North, E.P.E- Hospital Santa Maria
Lisbon, Portugal
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Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
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Hospital Universitario Ramon y Cajal
Pozuelo de Alarcón, 28224, Spain
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Hospital Universitario Y Politecnico La Femerge
Valencia, 46026, Spain
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Instituto Nacional De Rehabilitacion
Mexico City, Mexico
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Irccs Gaslini Institute
Genoa, 16147, Italy
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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Nagoya University Hospital
Nagoya, Japan
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Norrlands Universitetssjukhus
Umeå, Sweden
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Peking Union Medical College Hospital
Beijing, China
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Royal North Shore Hospital - New South Wales
Sydney, Australia
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Seoul National University Hospital
Seoul, South Korea
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Shangai Children Medical Center
Shanghai, China
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Perelman School of Medicine - The University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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The University of Tokyo Hospital
Tokyo, Japan
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Tongi University - Tongi Hospital
Shanghai, China
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University Health Network (UHN), Toronto General Hospital (TGH)
Toronto, Canada
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University Hospitals Leuven
Leuven, Belgium
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University of Alberta, Alberta Health Services (AHS)
Edmonton, Canada
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University of California San Francisco (UCSF)
San Francisco, California, 94143, United States
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