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

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.

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