Phase III Trial of Sirolimus in IBM (NCT04789070)
A Double-Blind Randomised Controlled Trial (dbRCT) Phase III Trial Investigating the Effect of Sirolimus on Disease Progression in Patients With Inclusion Body Myositis (IBM) as Measured by the IBM Functional Rating Scale (IBM-FRS)
🩺 Plain-English Summary (8th-Grade Level)
The hypothesis is that Sirolimus, (Rapamycin (R)) which is currently used in organ transplantation and works by blocking the activity of T effector cells but preserving T regulatory cells, as well as by inducing autophagy (protein degradation), will be effective in IBM to slow or stabilize disease progression, helping to maintain patient function and independence. This phase III trial will confirm pilot data showing statistically significant clinical outcomes.
Who can join: Review the eligibility criteria below.
Location: University of Kansas Medical Center — Kansas City, Kansas
Age range: 45 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (10 Sites)
- University of Kansas Medical Center — Kansas City, Kansas
- Johns Hopkins University — Baltimore, Maryland
- Concord Repatriation Hospital — Sydney, New South Wales
- Royal Northshore Hospital — Sydney, New South Wales
- Royal Brisbane and Women's Hospital — Brisbane, Queensland
- Royal Adelaide Hospital — Adelaide, South Australia
- Austin Health — Melbourne, Victoria
- St Vincent's Hospital — Melbourne, Victoria
- Perron Institute — Perth, Washington
- Leiden University Medical Center — Leiden,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.