Experimental pill aims to stop extra bone growth in rare disease

NCT ID NCT05039515

Disease control Sponsor: Clementia Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

113 people

The number who actually took part.

Started

Dec 2021

Finished

Mar 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

5 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Participants must be at least 5 years of age, to be confirmed (entry for younger paediatric participants \<15 years of age will only be once safety in adult and older paediatric participants ≥15 years of age has been established) at the time of signing the informed participant/parent consent and, for participants who are minors, age-appropriate assent. * Participants must be at least 15 years of age at the time of signing the informed participant/parent consent for the main study and, for participants who are minors, age-appropriate assent * Participants must be clinically diagnosed with FOP, with the R206H ACVR1 mutation or other FOP variants associated with progressive HO. * Participants must have disease progression in the preceding year of the screening visit. * Participants who have participated in a prior clinical study using another investigational product for the treatment of FOP may be enrolled after a washout of at least 5 half-lives of the other investigational product. Participants with prior treatment such as, but not limited to, imatinib, isotretinoin, garetosmab, or palovarotene may be enrolled 30 days after discontinuation or after washout of at least 5 half-lives, whichever is longer. 1. Washout period for palovarotene is 30 days 2. Washout period for garetosmab is 4 months * Participants must be able to perform pulmonary function tests adequately and reliably. * Participants must be able to have an adequate echocardiography assessment at screening for evaluation of left ventricular structure and function as defined by the protocol. * Participants must be accessible for treatment and follow-up and be able to undergo all study procedures. Participants living at distant locations from the investigational site must be able and willing to travel to a site for the initial and all on-site follow-up visits. Participants must be able to undergo low-dose WBCT (excluding head) without sedation. * Body weight ≥10 kg. * Abstinent or using two highly effective forms of birth control. Females must also have a negative blood or urine pregnancy test prior to administration of study drug. * Participants must be capable of giving written, signed, and dated informed participant/parent consent; and for participants who are minors, age-appropriate assent and/or legal guardian consent (performed according to local regulations) Key Exclusion Criteria: * Participants with complete heart block and left bundle branch block on screening electrocardiogram. * Participants with screening echocardiography showing septal or left ventricular free wall thickness \>12 mm for adult participants or a z-score \>3 compared with population norms for children and adolescent participants or left ventricular ejection fraction (LVEF) \<50%. * Participants with severe mitral or tricuspid regurgitation on echocardiography at screening. * Participants with significant underlying lung disease requiring supplementary oxygen or forced vital capacity \<35% of predicted at screening. * Participants with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or another significant disease as judged by the investigator. * Participants with severe hepatic impairment. * Concomitant medications that are strong inhibitors (including grapefruit juice) or inducers (including St John's Wort) of cytochrome P450 (CYP) 3A4 activity; or kinase inhibitors such as imatinib. * Prior use in the past year and concomitant use of bisphosphonates for participants in the PET-CT sub study. * Concurrent participation in another interventional clinical study, or a noninterventional study with radiographic measures or invasive procedures (e.g. collection of blood or tissue samples). * Amylase or lipase \>2× the upper limit of normal (ULN) or with a history of chronic pancreatitis. * Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>5×ULN. * Participants with hematologic abnormalities: * Hgb\<10g/dL * Platelets\<75,000/mm3 * WBC\<2000/mm3

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Conditions

The condition(s) this trial relates to.

fibrodysplasia ossificans progressiva myositis ossificans

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fibrodysplasia ossificans progressiva

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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