What side effects are associated with this type of intervention?

Sirolimus (Rapamycin) is commonly associated with side effects such as mouth ulcers, increased risk of infections, elevated cholesterol and triglyceride levels, and potential kidney dysfunction. Other immunosuppressive treatments may also cause gastrointestinal disturbances, liver toxicity, and hematologic abnormalities like anemia or leukopenia. Patients should be closely monitored for these adverse effects during treatment.

What other types of research exist?

Promising novel alternatives to Sirolimus for Inclusion Body Myositis patients include Tacrolimus, which has shown efficacy in other muscle-related diseases by modulating T cells, and Mycophenolate Mofetil, which has been used successfully in refractory myositis cases. Additionally, Janus kinase (JAK) inhibitors like Tofacitinib have shown potential in treating refractory dermatomyositis, suggesting a possible benefit for IBM.

← Back to Search

Sirolimus for Inclusion Body Myositis

Q: What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Inclusion Body Myositis (IBM). However, Sirolimus (Rapamycin) is being studied for its potential benefits in IBM due to its ability to block T effector cells, preserve T regulatory cells, and induce autophagy. This mechanism of action is promising for managing IBM, but further clinical trials are needed to confirm its efficacy and safety for this condition.

Phase 3

Recruiting

Led By Mazen Dimachkie

Research Sponsored by University of Kansas Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 84

Awards & highlights

Pivotal Trial

Summary

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.

Who is the study for?

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.

What is being tested?

The 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.

What are the potential side effects?

Sirolimus may cause side effects like increased risk of infections due to immune system suppression, potential kidney or liver issues, high cholesterol levels, low blood cell counts which could lead to fatigue or bleeding problems, and possible allergic reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 84

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 84

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 84 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in IBM Functional Rating Scale (IBM-FRS) from Baseline to Week 84

Secondary study objectives

Change in 6 Minute Walk Test (6MWT) from Baseline to Week 84

Change in Manual Muscle Testing (MMT) from Baseline to Week 84

Change in Modified Timed Up and Go (mTUG) from Baseline to Week 84

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: SirolimusActive Control1 Intervention

2mg capsules once daily

Group II: PlaceboPlacebo Group1 Intervention

2mg capsules once daily

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Sirolimus (Rapamycin) is a promising treatment for Inclusion Body Myositis (IBM) due to its ability to block T effector cells, preserve T regulatory cells, and induce autophagy. Blocking T effector cells reduces inflammation and muscle damage, while preserving T regulatory cells helps maintain immune system balance. Inducing autophagy aids in the degradation of damaged proteins and organelles, addressing the protein aggregation and muscle degeneration seen in IBM. These mechanisms collectively help slow disease progression and maintain patient function and independence.

Rapamycin Treatment of Tendon Stem/Progenitor Cells Reduces Cellular Senescence by Upregulating Autophagy.Defective autophagy is associated with neuronal injury in a mouse model of multiple sclerosis.Rapamycin induces autophagy in the melanoma cell line M14 via regulation of the expression levels of Bcl-2 and Bax.