~50 spots leftby Dec 2026

Sirolimus for Inclusion Body Myositis

Recruiting in Palo Alto (17 mi)
+8 other locations
Dr. Mazen M Dimachkie, MD - Kansas City ...
Overseen byMazen Dimachkie
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Waitlist Available
Sponsor: University of Kansas Medical Center
Pivotal Trial (Near Approval)
Prior Safety Data
Breakthrough Therapy

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?

Yes, Sirolimus shows promise as a treatment for Inclusion Body Myositis. It has been reported to improve muscle function and reduce inflammation in both human and animal studies. This suggests it could help people with this muscle disease.13678

Research Team

Dr. Mazen M Dimachkie, MD - Kansas City ...

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

Your condition has gotten worse in the past year, as confirmed by a neuromuscular specialist through various tests and examinations.
You are a man or woman who is 45 years old or older.
Adults able to read and understand the Participant Information Sheet, and who freely provide written Informed Consent for the study
See 5 more

Exclusion Criteria

You have taken Sirolimus or Everolimus in the last 6 months.
If patient has received a live vaccine within the last 12 weeks
Has taken any investigational study drug within 30 days or five half-lives of the prior agent (whichever is longer) prior to the Baseline visit
See 17 more

Treatment Details

Interventions

  • Placebo (Drug)
  • Sirolimus (mTOR inhibitor)
Trial OverviewThe trial is testing if Sirolimus can slow down IBM's progress compared to a placebo. It's based on earlier results suggesting benefits from Sirolimus due to its effects on immune cells and protein degradation. Patients will be randomly assigned either the drug or a placebo for comparison.
Participant Groups
2Treatment groups
Active Control
Placebo Group
Group I: SirolimusActive Control1 Intervention
2mg capsules once daily
Group II: PlaceboPlacebo Group1 Intervention
2mg capsules once daily

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Kansas Medical Center

Lead Sponsor

Trials
527
Recruited
181,000+
Dr. Steve Stites profile image

Dr. Steve Stites

University of Kansas Medical Center

Chief Executive Officer

MD from University of Kansas School of Medicine

Dr. Matthias Salathe profile image

Dr. Matthias Salathe

University of Kansas Medical Center

Chief Medical Officer

MD from University of Kansas School of Medicine

The Perron Institute

Collaborator

Trials
1
Recruited
140+

Findings from Research

Long-term treatment with bimagrumab for up to 2 years in patients with sporadic inclusion body myositis (sIBM) was found to be safe and well-tolerated, with a high incidence of treatment-emergent adverse events similar to the placebo group.
Despite its safety profile, bimagrumab did not demonstrate any significant clinical benefits in improving mobility, as indicated by the progressive deterioration in the 6-minute walk distance across all treatment groups.
Efficacy and Safety of Bimagrumab in Sporadic Inclusion Body Myositis: Long-term Extension of RESILIENT.Amato, AA., Hanna, MG., Machado, PM., et al.[2022]
A single series of Alemtuzumab infusions in 13 patients with sporadic inclusion-body myositis (sIBM) significantly slowed disease progression, with only a 1.9% decline in muscle strength over 6 months compared to a 14.9% decline in the natural history, indicating a potential therapeutic benefit.
The treatment led to a notable reduction in endomysial T cells and inflammatory markers in muscle biopsies, suggesting that Alemtuzumab not only depletes peripheral lymphocytes but also reduces inflammation associated with sIBM.
Effect of Alemtuzumab (CAMPATH 1-H) in patients with inclusion-body myositis.Dalakas, MC., Rakocevic, G., Schmidt, J., et al.[2022]
In a study comparing rapamycin and methylprednisolone (MP) in a mouse model of autoimmune myositis, rapamycin significantly reduced inflammation and improved muscle strength more effectively than MP over a 14-day treatment period.
The therapeutic effects of rapamycin were associated with increased plasma levels of transforming growth factor-β (TGF-β), suggesting that its immune-suppressive effects may be mediated through the TGF-β signaling pathway.
Comparison of rapamycin and methylprednisolone for treating inflammatory muscle disease in a murine model of experimental autoimmune myositis.Kang, J., Feng, D., Yang, F., et al.[2020]

References

Sirolimus leads to rapid and sustained clinical improvement of motor deficits in a patient with inclusion body myositis. [2022]
Epidemiology, Survival, and Clinical Characteristics of Inclusion Body Myositis. [2022]
Efficacy and Safety of Bimagrumab in Sporadic Inclusion Body Myositis: Long-term Extension of RESILIENT. [2022]
Effect of Alemtuzumab (CAMPATH 1-H) in patients with inclusion-body myositis. [2022]
Pilot trial of simvastatin in the treatment of sporadic inclusion-body myositis. [2021]
Comparison of rapamycin and methylprednisolone for treating inflammatory muscle disease in a murine model of experimental autoimmune myositis. [2020]
Beneficial role of rapamycin in experimental autoimmune myositis. [2021]
[Inclusion body myositis--a rarely recognized disorder]. [2013]