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Sirolimus for Inclusion Body Myositis

Phase 3
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
Evidence of disease progression over the previous 12 months, as determined by a neuromuscular specialist through patient history, physical examination, MMT, IBM-FRS or other metrics
Males or females aged 45 years or older
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 84
Awards & highlights
Pivotal Trial

Study 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.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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.
You can walk at least 200 meters in 6 minutes, even if you need to use walking aids like frames.
You have been diagnosed with IBM using specific criteria from 2011.


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

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

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

Find a Location

Who is running the clinical trial?

University of Kansas Medical CenterLead Sponsor
454 Previous Clinical Trials
162,112 Total Patients Enrolled
The Perron InstituteUNKNOWN
Mazen DimachkiePrincipal InvestigatorUniversity of Kansas Medical Center
1 Previous Clinical Trials
9 Total Patients Enrolled

Media Library

Placebo Clinical Trial Eligibility Overview. Trial Name: NCT04789070 — Phase 3
Inclusion Body Myositis Research Study Groups: Sirolimus, Placebo
Inclusion Body Myositis Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT04789070 — Phase 3
Placebo 2023 Treatment Timeline for Medical Study. Trial Name: NCT04789070 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people are being recruited for this clinical trial?

"That is correct. The clinicaltrials.gov site says that this study, which was first advertised on July 1st, 2022, is still looking for participants. They need to find 140 people total between 2 hospitals or clinics."

Answered by AI

Are new volunteers being accepted for this research program?

"Yes, that is accurate. The clinical trial in question is actively recruiting patients, as stated on the website clinicaltrials.gov. This specific study was originally posted on July 1st, 2022 and was edited October 12th of the same year. A total of 140 patients are being recruited from 2 sites."

Answered by AI

How is Sirolimus most often employed?

"Sirolimus has shown efficacy in treating transplant rejection, liver disease, and kidney disease."

Answered by AI

Does the FDA sanction the use of Sirolimus?

"Sirolimus has been studied in Phase 3 clinical trials, demonstrating some efficacy and supporting multiple rounds of data that suggest it is safe."

Answered by AI

Has Sirolimus been looked at under a microscope before?

"Currently, there are one hundred and thirty clinical trials involving sirolimus with thirteen of those in Phase 3. Although a few of the tests are taking place in Cincinnati, Ohio, there are 1084 locations worldwide running clinical trials for sirolimus."

Answered by AI

Who else is applying?

What state do they live in?
How old are they?
18 - 65
What site did they apply to?
Johns Hopkins University
University of Kansas Medical Center
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
How many prior treatments have patients received?

Why did patients apply to this trial?

I am a transplant patient and have been taking sirolimus a few years.
PatientReceived 1 prior treatment
~54 spots leftby Feb 2025