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Study on the Effects of Fidrisertib for Treating Fibrodysplasia Ossificans Progressiva in Children and Adults

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare condition called Fibrodysplasia Ossificans Progressiva (FOP). FOP is a disorder where soft tissues in the body, like muscles and tendons, gradually turn into bone, leading to restricted movement. The study is testing a new treatment called fidrisertib, also known by its code name IPN60130. This treatment is being compared to a placebo to see if it can help reduce the formation of new bone in people with FOP.

The purpose of the study is to evaluate how effective and safe fidrisertib is for both children and adults with FOP. Participants in the study will take the medication in the form of a hard capsule by mouth. The study will last for a period of up to 60 days, during which participants will be monitored for any changes in their condition and any side effects they might experience. The study will use imaging techniques like computed tomography (CT) to measure changes in bone formation.

Throughout the study, participants will have regular check-ups to assess their health and the progress of their condition. The study aims to provide valuable information on whether fidrisertib can effectively slow down or stop the progression of FOP, offering hope for better management of this challenging condition. Participants will be closely monitored to ensure their safety and well-being during the trial.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, participants are required to provide written consent. For minors, consent from a parent or legal guardian is necessary, along with age-appropriate assent from the participant.

    Participants must be at least 5 years old, with specific conditions for those under 15 years of age.

  2. Step 2

    Initial assessment

    Participants undergo a clinical diagnosis to confirm fibrodysplasia ossificans progressiva (FOP) with specific genetic mutations.

    A screening visit is conducted to assess disease progression over the past year, which may include symptoms like swelling, pain, or new bone growth.

  3. Step 3

    Medication administration

    Participants receive either the study drug, fidrisertib (IPN60130), or a placebo. The medication is administered orally in the form of hard capsules.

    The study evaluates two dosage regimens of fidrisertib for its effectiveness and safety in treating FOP.

  4. Step 4

    Monitoring and follow-up

    Participants are monitored for changes in new bone growth using low-dose whole body computed tomography (WBCT), excluding the head.

    Regular assessments include checking for adverse events, cardiac health, vital signs, and other health parameters.

  5. Step 5

    Completion of study

    The study is expected to conclude by August 31, 2029.

    Participants will have follow-up visits to assess the long-term effects of the treatment and any new developments in their condition.

Who can join the trial?

13 criteria

  • Participants must be at least 5 years old. Younger children under 15 can join only after safety is confirmed in older participants.
  • Both male and female participants can join. They must follow local rules for birth control during the study.
  • Male participants who can have children must agree to avoid heterosexual sex or use two effective birth control methods during and for 90 days after treatment.
  • Female participants who can have children must have a negative pregnancy test before starting the study. They must agree to avoid heterosexual sex or use two effective birth control methods during and for 1 month after treatment.
  • Participants must be able to give written consent. For minors, a parent or legal guardian must also give consent.
  • Participants must be at least 15 years old to join a specific imaging part of the study.
  • Participants must have a clinical diagnosis of **Fibrodysplasia Ossificans Progressiva (FOP)**, with a specific genetic mutation or other variants linked to the disease.
  • Participants must show disease progression in the past year, such as a flare-up with symptoms like swelling or pain, new bone growth, joint stiffness, or an increase in a specific joint score.
  • Participants who were in a previous study with another treatment for FOP can join after a waiting period, which depends on the previous treatment.
  • Participants must be able to perform lung function tests as required by the study.
  • Participants must be able to have a heart ultrasound to check heart structure and function.
  • Participants must be able to attend all study visits and procedures, including traveling to the study site if needed.
  • Participants must weigh at least 10 kg.

Who cannot join the trial?

10 criteria

  • Participants who have any other serious health condition that might interfere with the study.
  • Individuals who are currently taking medications that could affect the study results.
  • People who have had a recent surgery or are planning to have surgery during the study period.
  • Participants who are pregnant or breastfeeding.
  • Individuals who have participated in another clinical trial recently.
  • People who have allergies to the study medication or its ingredients.
  • Participants who are unable to follow the study procedures or attend study visits.
  • Individuals with a history of drug or alcohol abuse.
  • People who have a mental health condition that might affect their ability to participate.
  • Participants who have a family member involved in the study.
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Investigated drugs

Fidrisertib (IPN60130) is an oral medication being studied for its potential to treat fibrodysplasia ossificans progressiva (FOP). This condition causes abnormal bone growth in muscles and other tissues. The trial aims to see if fidrisertib can reduce the formation of new bone outside the normal skeleton, which is known as heterotopic ossification. The study also evaluates the safety of fidrisertib in both children and adults with FOP.

What is already known about the treatment

Fidrisertib – This medication is administered orally and is currently being studied in clinical trials for its effectiveness and safety in treating fibrodysplasia ossificans progressiva (FOP), a rare condition. The main therapeutic indication for fidrisertib is to inhibit the formation of new bone in soft tissues, known as heterotopic ossification, in both children and adults with FOP. At the molecular level, fidrisertib works by targeting specific pathways that contribute to abnormal bone growth. It is classified pharmacologically as a kinase inhibitor, which helps to block the signals that lead to unwanted bone formation.

Investigated diseases

Fibrodysplasia Ossificans Progressiva – This is a rare genetic disorder characterized by the abnormal development of bone in areas where bone should not form, such as muscles, tendons, and other connective tissues. Over time, these extra bones can restrict movement and lead to significant physical limitations. The condition often begins in early childhood, with episodes of painful swelling and inflammation that precede the formation of new bone. These episodes, known as flare-ups, can be triggered by trauma, viral illnesses, or even without any apparent cause. As the disease progresses, the formation of new bone can lead to joint immobility and fusion of bones, severely affecting the individual's ability to move. The progression of the disease varies among individuals, with some experiencing more rapid ossification than others.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-511469-13-00Protocol codeD-CA-60130-452Estimated enrolment119 patientsSponsorClementia Pharmaceuticals Inc. An Ipsen Company

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