Rapamycin

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,
placebo
Placebo (control): An inactive product that resembles the test medicine or procedure, but without a treatment value. A placebo can be a pill, a powder, or a solution that resembles the test drug, but has no physical effect.  
controlled trial enrolling 24 patients, ages 6-59 years of age. The first 24 weeks of the study will be
placebo
Placebo (control): An inactive product that resembles the test medicine or procedure, but without a treatment value. A placebo can be a pill, a powder, or a solution that resembles the test drug, but has no physical effect.  
controlled, followed by 24 weeks of open label (all patients receive the rapamycin).

Eligibility Criteria Icon
Eligibility Criteria*
  • AGE: ≥ 6-59
  • DISEASE ACTIVITY: Prior to enrolling, patients cannot have experienced a flare-up during the last 90 days
  • MUTATIONS: All mutations
Study Design Icon
Study Design*
  • STUDY TYPE:  Interventional
  • RANDOMIZED STUDY:  Yes
  • PLACEBO
    Placebo (control): An inactive product that resembles the test medicine or procedure, but without a treatment value. A placebo can be a pill, a powder, or a solution that resembles the test drug, but has no physical effect.  
    CONTROLLED:  Yes: for the first 24 weeks, participants will be given either rapaymycin or
    Placebo
    Placebo (control): An inactive product that resembles the test medicine or procedure, but without a treatment value. A placebo can be a pill, a powder, or a solution that resembles the test drug, but has no physical effect.  
    . 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
Status Icon
Status

Phase 1
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.  
, Active
Therapy Approach Icon
Therapy Approach

Immune suppression

Study Sponsor Icon
Study Sponsor

Kyoto University

*For the complete list of eligibility criteria and details on this study, visit UMIN.AC.JP

Make a Difference

Donate Connect Fundraise

Already a Member? Sign In

Sign Up for FOP Connection, Our Monthly eNewsletter