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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| INCB000928 (Incyte) |
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| IPN60130 (Ipsen) |
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| Rapamycin (Kyoto University) |
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| Saracatinib (STOPFOP Investigators) |
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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.
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.
