Phase III Trial of Sirolimus in IBM (NCT04789070) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Phase III Trial of Sirolimus in IBM
United States, Australia140 participantsStarted 2022-07-01
Plain-language summary
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 participate
Age range45 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Adults able to read and understand the Participant Information Sheet, and who freely provide written Informed Consent for the study;
✓. Males or females aged 45 years or older;
✓. Diagnosis of IBM according to the criteria proposed by the ENMC criteria 2011;
✓. Able to walk a minimum distance of 200m within 6 minutes (walking aids, including frames, may be used);
✓. 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.
Exclusion criteria
✕. Inability to complete a 6MWT with a minimum distance of 200m achieved;
✕. Inability to complete a mTUG or any other study procedure, including inability to swallow study drug, or clinical suspicion that the participant will become unable to swallow the study drug during the study period;
✕. Unwillingness or inability to comply with study interventions or study schedule;
✕. Hypersensitivity to Sirolimus, Everolimus or any compound of the oral solution;
✕. Any prior exposure to Sirolimus or Everolimus within the last 6 months;
✕. Presence of any other clinically significant disease that might interfere with patients ability to comply with study procedures, or places the patient at greater risk for SAEs;
What they're measuring
1
Change in IBM Functional Rating Scale (IBM-FRS) from Baseline to Week 84
✕. Clinical suspicion of moderate or severe respiratory insufficiency based on history, clinical examination or respiratory function tests with an FVC \< 50% of predicted; Nocturnal NIV is allowed for sleep-disordered breathing;
✕. Severe chronic kidney disease or renal insufficiency with proteinuria (e.g Estimated Glomerular Filtration Rate \< 30 ml/min and/or proteinuria as defined by spot urine protein/creatinine ratio \> 100mg/mmol;