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 https://upload.umin.ac.jp/cgi-open-bin/ctr/ctr_view.cgi?recptno=R000032495.
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.