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.
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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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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