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Sirolimus for Inclusion Body Myositis
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
Summary
This trial will test if a drug already used in organ transplantation, called Sirolimus (Rapamycin (R)), can help to slow or stabilize disease progression in IBM patients, helping them to maintain function and independence.
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.Check my eligibility
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.See study design
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
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in IBM Functional Rating Scale (IBM-FRS) from Baseline to Week 84
Secondary outcome measures
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
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
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.
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.
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Who is running the clinical trial?
University of Kansas Medical CenterLead Sponsor
472 Previous Clinical Trials
171,299 Total Patients Enrolled
The Perron InstituteUNKNOWN
Mazen DimachkiePrincipal InvestigatorUniversity of Kansas Medical Center
1 Previous Clinical Trials
9 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have taken Sirolimus or Everolimus in the last 6 months.You do not have HIV, Hepatitis C, or certain markers for Hepatitis B.Your condition has gotten worse in the past year, as confirmed by a neuromuscular specialist through various tests and examinations.You cannot walk at least 200 meters during a six-minute walk test.You are allergic to Sirolimus or Everolimus.You have a history, examination, or tests that suggest you have moderate or severe breathing problems, and your forced vital capacity (FVC) is less than half of what it should be. You are allowed to use a machine to help you breathe while you sleep if you have sleep-related breathing issues.Your blood tests show very high cholesterol or triglycerides, or low levels of hemoglobin, platelets, neutrophils, or lymphocytes.You are a man or woman who is 45 years old or older.You have severe kidney disease with protein in your urine.You can walk at least 200 meters in 6 minutes, even if you need to use walking aids like frames.You have chronic liver disease or very high levels of liver enzymes, unless it's due to a muscle-related issue. If your liver enzymes are temporarily high due to something like a viral infection, you can be screened again later.You have had any type of cancer, except for skin cancers that were only in one area, in the past 5 years.You have an autoimmune disease that is not under control and requires ongoing treatment. This includes conditions like Graves' disease.You are taking medications that weaken or change your immune system.You are taking certain medications that may interact with sirolimus. Also, you are not taking any other experimental drugs besides the study medication.You have been diagnosed with IBM using specific criteria from 2011.You can walk at least 200 meters within 6 minutes, and you can use walking aids like frames if needed.You are 45 years old or older.Your condition has gotten worse in the past year, according to a specialist and certain tests.
Research Study Groups:
This trial has the following groups:- Group 1: Sirolimus
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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