Rapamycin, also known as sirolimus, is an immune suppressive drug that has been approved to coat coronary stents, prevent organ transplant rejection and to treat a rare lung disease. Rapamycin has immunosuppressant and antiproliferative properties in humans and is especially useful in preventing the rejection of kidney transplants. In FOP animal models, rapamycin showed a slowing of heterotopic ossification.
About the Clinical Trial
The rapamycin trial is a national (Japan only), multi-center,
controlled trial enrolling 24 patients, ages 6-59 years of age. The first 24 weeks of the study will be
controlled, followed by 24 weeks of open label (all patients receive the rapamycin).
- AGE: ≥ 6-59
- DISEASE ACTIVITY: Prior to enrolling, patients cannot have experienced a flare-up during the last 90 days
- MUTATIONS: All mutations
- STUDY TYPE: Interventional
- RANDOMIZED STUDY: Yes
PLACEBOCONTROLLED: Yes: for the first 24 weeks, participants will be given either rapaymycin orPlacebo. At the end of that period, all participants (100%) will receive rapamycin for an additional 24 weeks.
- LENGTH OF PARTICIPATION: 48 weeks
- NUMBER OF STUDY VISITS: n/a
*For the complete list of eligibility criteria and details on this study, visit UMIN.AC.JP
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.