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 Read more

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

Locations

  • Aichi Children's Health and Medical Center

    Ōbu, Japan

  • Children's Hospital Capital Institute of Pediatrics (CIP)

    Beijing, China

  • Groupe Hospitalier Necker Enfants Malades

    Paris, 75015, France

  • Hopital Lariboisiere

    Paris, France

  • Hospital Center Lisbon North, E.P.E- Hospital Santa Maria

    Lisbon, Portugal

  • Hospital Italiano de Buenos Aires

    Buenos Aires, Argentina

  • Hospital Universitario Ramon y Cajal

    Pozuelo de Alarcón, 28224, Spain

  • Hospital Universitario Y Politecnico La Femerge

    Valencia, 46026, Spain

  • Instituto Nacional De Rehabilitacion

    Mexico City, Mexico

  • Irccs Gaslini Institute

    Genoa, 16147, Italy

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Nagoya University Hospital

    Nagoya, Japan

  • Norrlands Universitetssjukhus

    Umeå, Sweden

  • Peking Union Medical College Hospital

    Beijing, China

  • Royal North Shore Hospital - New South Wales

    Sydney, Australia

  • Seoul National University Hospital

    Seoul, South Korea

  • Shangai Children Medical Center

    Shanghai, China

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • The Perelman School of Medicine - The University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • The University of Tokyo Hospital

    Tokyo, Japan

  • Tongi University - Tongi Hospital

    Shanghai, China

  • University Health Network (UHN), Toronto General Hospital (TGH)

    Toronto, Canada

  • University Hospitals Leuven

    Leuven, Belgium

  • University of Alberta, Alberta Health Services (AHS)

    Edmonton, Canada

  • University of California San Francisco (UCSF)

    San Francisco, California, 94143, United States

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