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Fibrodysplasia ossificans progressiva – Trials in Disease

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Ongoing Clinical Trials for Fibrodysplasia Ossificans Progressiva

There are currently 5 ongoing clinical trials investigating treatments for Fibrodysplasia Ossificans Progressiva (FOP), a rare genetic disorder where soft tissues gradually turn into bone. These trials are testing different medications aimed at preventing abnormal bone formation and are being conducted across multiple countries in Europe. (Also known as: FOP, Stone Man Syndrome)

Clinical trial locations

Study on the Effectiveness of INCB000928 for Patients with Fibrodysplasia Ossificans Progressiva

This trial is testing INCB000928, a medication taken as a film-coated tablet. The drug is an ALK2 inhibitor designed to block a specific protein that may play a role in abnormal bone growth.

Main inclusion criteria: Participants must be at least 12 years old and have a confirmed clinical diagnosis of FOP, including congenital malformation of the great toes, episodes of soft-tissue swelling, or progressive bone formation in muscles. They must have reported disease activity within one year before joining. Participants need to be able to swallow tablets or receive them through a feeding tube, undergo a low-dose whole-body CT scan without breathing assistance, and agree to contraception measures.

Main exclusion criteria: People with other serious health conditions that could interfere with the study, those currently taking medications that might affect results, recent participants in other clinical trials, pregnant or breastfeeding women, individuals unable to follow study procedures, those with allergies to the medication, people with a history of drug or alcohol abuse, individuals with certain mental health conditions, those who had recent surgery or are planning surgery, and people with active infections requiring antibiotics are excluded.

Study focus: The trial evaluates how effective and safe INCB000928 is in preventing new bone growth. Participants will be randomly assigned to receive either the medication or a placebo in a double-blind manner. The study uses low-dose whole-body CT scans to assess the total volume of new bone growth at Week 24 and monitors the number of new flare-ups. Regular check-ups will track participants’ health and any changes in their condition throughout the study period.

Study on the Safety of Intradermal mRNA COVID-19 Vaccine Bretovameran for Patients with Fibrodysplasia Ossificans Progressiva

This unique trial studies a different approach by testing the safety of a COVID-19 vaccine specifically in people with FOP. The vaccine contains the active substance bretovameran and is administered as an intradermal injection just under the skin, rather than into the muscle.

Main inclusion criteria: Participants must be at least 18 years old and have a clinical diagnosis of FOP confirmed by a genetic mutation in the ACVR1 gene. They must be willing and able to attend all scheduled visits, take vaccination tests, and follow other study procedures. Participants need to provide personal signed consent.

Main exclusion criteria: The trial excludes individuals who do not have FOP, those outside the specified age range, though both male and female patients are eligible.

Study focus: The trial evaluates the safety of a fractional dose (one-fifth of the regular dose) of the COVID-19 mRNA vaccine in people with FOP. Participants will be monitored for side effects such as flare-ups, fever, fatigue, headache, chills, vomiting, diarrhea, muscle pain, and joint pain. The study also measures the body’s immune response by checking antibody levels on specific days. Local reactions at the injection site and any reactions in nearby lymph nodes are tracked, as is the use of medications like corticosteroids, antipyretics, and painkillers.

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

This trial tests fidrisertib (IPN60130), a treatment taken as a hard capsule by mouth. The medication is a kinase inhibitor designed to block signals that lead to unwanted bone formation.

Main inclusion criteria: Participants must be at least 5 years old, with children under 15 able to join only after safety is confirmed in older participants. Both males and females can participate, and they must follow birth control requirements during the study. For minors, a parent or legal guardian must provide consent. Participants must have a clinical diagnosis of FOP with a specific genetic mutation and show disease progression in the past year, such as a flare-up with swelling or pain, new bone growth, joint stiffness, or an increase in joint scores. They must be able to perform lung function tests, have a heart ultrasound, attend all study visits, and weigh at least 10 kg.

Main exclusion criteria: The trial excludes individuals with other serious health conditions that might interfere, those taking medications that could affect results, people who had recent surgery or are planning surgery, pregnant or breastfeeding individuals, recent participants in other trials, those with allergies to the study medication, people unable to follow procedures, individuals with drug or alcohol abuse history, those with certain mental health conditions, and participants with family members involved in the study.

Study focus: The study compares fidrisertib to a placebo to evaluate its effectiveness and safety in reducing new bone formation. The trial uses low-dose whole-body CT scans to measure changes in bone formation and monitors participants for up to 60 days. Regular check-ups assess health, disease progress, adverse events, cardiac health, vital signs, and other health parameters. The study is expected to conclude by August 31, 2029.

Study on the Safety and Effectiveness of Garetosmab for Adults with Fibrodysplasia Ossificans Progressiva

This trial tests Garetosmab, a monoclonal antibody designed to target specific substances in the body. It is given through an intravenous infusion directly into a vein.

Main inclusion criteria: Participants must have a clinical diagnosis of FOP confirmed by a Type I activin A receptor (ACVR1) genetic mutation. They must have experienced FOP activity within the last year, including pain, swelling, stiffness, or other flare-up symptoms, worsening joint function, or larger bone growths. Participants must be willing and able to undergo CT imaging.

Main exclusion criteria: Pregnant or breastfeeding women are excluded, as are individuals with severe allergic reactions to similar medications, those with other serious health conditions, recent participants in other trials (within 30 days), people unable to comply with procedures, individuals with drug or alcohol abuse history within the past year, those taking certain medications that might affect results, and people with unstable mental health conditions.

Study focus: The trial evaluates the safety and effectiveness of Garetosmab compared to a placebo over about 56 weeks. It uses low-dose CT scans to check for new bone formation. Throughout the study, participants undergo baseline evaluations, regular monitoring for adverse events and routine health checks, periodic CT scans, and interim evaluations at weeks 28, 56, and 84 that assess joint function, disease severity, flare-ups, and both clinician and patient-reported outcomes.

Study on the Safety and Effects of Saracatinib for Patients with Fibrodysplasia Ossificans Progressiva (FOP)

This trial tests Saracatinib (AZD0530), a tyrosine kinase inhibitor taken as a film-coated tablet. It works by inhibiting Src kinases, which play a role in signaling pathways leading to abnormal bone formation.

Main inclusion criteria: Participants must be aged 18-65 and have a clinical diagnosis of FOP, including congenital malformation of the great toes and a history of bone forming in soft tissues. They must have a confirmed classic-like FOP phenotype with the ACVR1R206H/+ genetic sequence or variant. Female participants of childbearing potential must use highly effective contraception along with condoms or barrier methods with spermicide from joining until 4 weeks after the last dose. Male participants must avoid sexual intercourse that could lead to pregnancy and use highly effective contraception, use condoms if their partner is pregnant, and refrain from donating sperm during the study and for 4 weeks after. Participants must be able to understand the study, sign informed consent, attend and comply with visits, follow rules, and undergo PET and CT imaging.

Main exclusion criteria: People not diagnosed with FOP, those younger than 3 years old, and vulnerable populations who cannot give consent or are in dependent relationships are excluded.

Study focus: The 18-month trial evaluates Saracatinib’s safety and effects on abnormal bone formation. Participants are randomly assigned to receive either Saracatinib or placebo during a 26-week randomized controlled trial phase, where safety, effectiveness, and new bone lesions are assessed using PET and CT scans. After this, participants enter a 6-month open-label extension where all receive the medication. Long-term follow-up occurs at weeks 52, 104, and 156 to assess changes in bone lesions and functional mobility, including joint involvement, mouth opening, and lung capacity.

Summary

The five ongoing clinical trials for Fibrodysplasia Ossificans Progressiva reflect the medical community’s efforts to find effective treatments for this rare and challenging condition. The trials test four different medications that work through various mechanisms to prevent abnormal bone formation: INCB000928 (an ALK2 inhibitor), fidrisertib (a kinase inhibitor), Garetosmab (a monoclonal antibody), and Saracatinib (a tyrosine kinase inhibitor). Additionally, one trial examines the safety of COVID-19 vaccination specifically for people with FOP.

The Netherlands stands out as the most active country, hosting all five trials, followed by France, Germany, Italy, and Spain with three trials each. This concentration of trials in these countries may reflect the presence of specialized FOP treatment centers and research expertise in these regions.

Most trials focus exclusively on preventing heterotopic ossification (abnormal bone growth), using low-dose CT scans to monitor new bone formation. The studies range from relatively short monitoring periods of up to 60 days to long-term follow-up extending to several years. While most trials target adult patients, the fidrisertib study notably includes children as young as 5 years old, recognizing that FOP often begins in childhood.

All trials emphasize safety monitoring alongside effectiveness, acknowledging that treatments for this rare condition must be carefully evaluated. Participants can expect regular medical assessments, imaging tests, and close monitoring for any side effects or disease progression during their participation.

Ongoing Clinical Trials on Fibrodysplasia ossificans progressiva

  • Study on the Safety of Intradermal mRNA COVID-19 Vaccine Bretovameran for Patients with Fibrodysplasia Ossificans Progressiva

    Recruiting

    3 1 1 1
    Investigated diseases:
    The Netherlands

  • Study on the Effectiveness of INCB000928 for Patients with Fibrodysplasia Ossificans Progressiva

    Recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy The Netherlands Portugal Spain

  • Study on the Safety and Effects of Saracatinib for Patients with Fibrodysplasia Ossificans Progressiva (FOP)

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Germany The Netherlands

  • Study on the Safety and Effectiveness of Garetosmab for Adults with Fibrodysplasia Ossificans Progressiva

    Not recruiting

    3 1
    Investigated diseases:
    Investigated drugs:
    Finland France Italy The Netherlands Poland Spain

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