Sirolimus for Inclusion Body Myositis
Trial Summary
What is the purpose of this trial?
This trial tests Sirolimus, a drug used in organ transplants, on patients with Inclusion Body Myositis (IBM). IBM causes muscle weakness, and current treatments are often ineffective. Sirolimus may help by calming harmful immune responses and cleaning up damaged proteins in muscle cells. Sirolimus has been used in organ transplant recipients to reduce the risk of skin cancer and manage immune system activity.
Do I need to stop my current medications to join the trial?
Yes, you may need to stop certain medications. The trial excludes participants who have taken immunosuppressive or immunomodulatory medications recently, such as high-dose prednisolone, IVIG, methotrexate, mycophenolate, Sirolimus, Everolimus, calcineurin inhibitors, azathioprine, rituximab, alemtuzumab, or other biologics. Additionally, medications affecting Sirolimus metabolism, like strong CYP3A4 inhibitors or inducers, are also restricted.
What data supports the idea that the drug Sirolimus for Inclusion Body Myositis is an effective treatment?
The available research shows that Sirolimus led to rapid and sustained improvement in motor deficits for a patient with Inclusion Body Myositis. This suggests that Sirolimus might be effective in improving muscle function in people with this condition. Compared to other treatments like Alemtuzumab and Simvastatin, which did not show significant improvement or were not recommended, Sirolimus appears to have a more positive impact on muscle strength and function.12345
What safety data exists for Sirolimus in treating Inclusion Body Myositis?
The available research does not provide direct safety data for Sirolimus in treating Inclusion Body Myositis. However, a study on a murine model of experimental autoimmune myositis showed that Rapamycin (another name for Sirolimus) had better immune suppressive effects compared to methylprednisolone, with decreased inflammation and improved muscle strength. No specific safety concerns were mentioned in this study. Further clinical trials are needed to establish the safety profile of Sirolimus in human patients with Inclusion Body Myositis.12356
Is the drug Sirolimus a promising treatment for Inclusion Body Myositis?
Research Team
Mazen Dimachkie
Principal Investigator
University of Kansas Medical Center
Eligibility Criteria
Adults aged 45+ with Inclusion Body Myositis (IBM) who can walk at least 200m with or without aids and have shown disease progression in the past year. Participants must understand the study, consent to it, not have used Sirolimus recently, be HIV and hepatitis negative, not pregnant or planning pregnancy, and agree to use contraception.Inclusion Criteria
Exclusion Criteria
Treatment Details
Interventions
- Placebo (Drug)
- Sirolimus (mTOR inhibitor)
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Kansas Medical Center
Lead Sponsor
Dr. Steve Stites
University of Kansas Medical Center
Chief Executive Officer
MD from University of Kansas School of Medicine
Dr. Matthias Salathe
University of Kansas Medical Center
Chief Medical Officer
MD from University of Kansas School of Medicine
The Perron Institute
Collaborator