Fibrodysplasia Ossificans Progressiva (FOP) Clinical Trials

Fibrodysplasia Ossificans Progressiva is caused by a gain-of-function mutation in the ACVR1 gene encoding Activin A Type I Receptor (ALK2), a bone morphogenetic protein (BMP) Type I receptor. Over 99% of FOP cases involve the same mutation (c.617G>A, p.R206H). This mutation causes constitutive ALK2 signaling and inappropriate BMP pathway activation in response to cellular stress.

The most common symptoms of Fibrodysplasia Ossificans Progressiva include Swelling and inflammation of soft tissues (flare-ups), Progressive stiffness and immobility at joints, Formation of ectopic bone bridges between muscles, Severely limited range of motion, Pain during flare-ups triggered by trauma or other factors, among others. Extremely rare, approximately 1 per 2 million people globally; extremely small patient population. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Fibrodysplasia Ossificans Progressiva from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Research in Fibrodysplasia Ossificans Progressiva is advancing through ALK2 inhibitors targeting the specific ACVR1 mutation that causes the disease, along with approaches to block BMP signaling pathways that drive abnormal bone formation. Clinical trials measure treatment success through imaging tracking ossification progression, assessments of functional mobility, and importantly, reductions in flare frequency and severity that cause pain and rapid progression.

Yes. As of September 2026, Trial Friend lists 3 FDA-approved drugs used to treat Fibrodysplasia Ossificans Progressiva: Sohonos (palovarotene), first approved in 2023; Atebrioz (zilurgisertib), first approved in 2026; and Pasatru (garetosmab-grts), first approved in 2026.

Each has its own page with what the label covers, side effects and how it is given, and the Treatments section of this page adds any further approved drugs found in FDA labeling data.

Fibrodysplasia Ossificans Progressiva is caused by variants in the ACVR1 gene. Most cases are de novo ACVR1 variants; only a small number are inherited from an affected parent.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it. Extremely rare, approximately 1 per 2 million people globally; extremely small patient population. Genetic testing confirms the diagnosis.

Yes. Genetic testing for Fibrodysplasia Ossificans Progressiva looks at the ACVR1 gene. Most cases are de novo ACVR1 variants; only a small number are inherited from an affected parent.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it.

A confirmed variant establishes the diagnosis and is often required to enroll in gene-specific trials. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Fibrodysplasia Ossificans Progressiva clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Fibrodysplasia Ossificans Progressiva clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.