Clinical Studies & Trials
Clinical trials are an integral part of drug development and are required before a medicine can be available by prescription.
Active Trials
The Interactive Drug Development Chart highlights investigational drugs for FOP that are currently in clinical studies. Clinical trials test whether a drug is both safe and effective in humans and are typically broken into three phases of development:
Phase 1: Studies in humans that assess safety and toxicity of a treatment in a small group of healthy volunteers or patients with the disease of interest.
Phase 2: Studies that further test safety and begin to test the effectiveness of the drug, often at a variety of doses.
Phase 3: Studies that assess safety and effectiveness of the drug in a larger group of patients. The drug is typically compared to a standard treatment(s) or to no treatment (e.g. natural history study or a placebo).
In rare diseases, however, some drugs may receive regulatory approval without completing all three phases. To learn more about drug development and clinical trials, please visit the IFOPA’s clinical trial page.
The IFOPA lists sponsors that adhere to international regulatory and clinical standards and conduct ethical and transparent research. The IFOPA attempts to update this chart as soon as changes are available. Please be advised there may be delays in updating this chart.
| Phase 1 | Phase 2 | Phase 3 | Rollover | Approved | |
| Palovarotene (Ipsen) |
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| Garetosmab (Regeneron) |
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| Zilurgisertib (Incyte) |
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| Andecaliximab (āshibio) |
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| Fidrisertib (Ipsen) |
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| Saracatinib (STOPFOP Investigators) |
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| Rapamycin (Kyoto University) |
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| BCX9250 (BioCryst Pharmaceuticals) |
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| DS-6016a (Daiichi Sankyo) |
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| KER-047 (Keros Therapeutics) |
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Learn more about other FOP research that you can participate in Other Research.
Trial Locations Map
Zoom in to see a list of clinical trial sites in your region of the world. Refresh the page to reset the map. Click on the blue dot for information about the trial(s) at that location.
Why Clinical Trials Aren’t Available Everywhere
We understand how frustrating and disappointing it can be to hear that a clinical trial isn’t available in your country. Here’s why that sometimes happens:
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Rules and Approvals Are Different Everywhere
Every country has its own laws and requirements for approving clinical trials. It can take a lot of time, paperwork, and money to get permission to run a trial in each country.
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Costs and Resources
Running a clinical trial is expensive. Sometimes researchers or companies can only afford to run the trial in a few places where they already have medical teams and hospitals set up for research.
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Finding the Right Partners
Clinical trials need doctors, hospitals, and researchers who are trained and ready to run the study. In some places, it’s harder to find the right partners or to train new ones in time.
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Fewer Patients or Specialists in Some Areas
If a disease is very rare, there might not be enough patients in a certain country to include that country in the study, or there may not be enough specialists familiar with the condition.
Even when a trial isn’t available nearby, we’re committed to keeping you informed and exploring other ways to support you and your family.
Please check back frequently and watch IFOPA social media and eNewsletters for announcements about new clinical trial locations.
The IFOPA does not endorse nor recommend specific clinical trials. Please speak with your doctor if you are interested in participating in a clinical trial.
