Phase III Trial of Sirolimus in IBM

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.

Timeline

Start

2022-07-01

Primary completion

2027-12

Completion

2027-12

Publications

• Badrising UA, Henderson R, Reddel S, Corbett A, Liang C, Reardon K, Ghaoui R, Bulsara M, Brady S, Brusch A, Chan D, Coudert JD, Fairchild T, Jain G, Kiernan MC, Leonard D, Lloyd T, Schmidt J, McDermott MP, Sanders L, Lowe C, van der Kooi AJ, Weihl C, Mohassel P, Simpson M, Carroll A, Cooper I, Beer K, Hiscock K, Walters S, Panicker A, Doverty A, Heim A, van Heur-Neuman M, Benveniste O, Dimachkie MM, Needham M. Optimism in inclusion body myositis: a double-blind randomised controlled phase III trial investigating the effect of sirolimus on disease progression in patients with IBM as measured by the IBM Functional Rating Scale. Clin Exp Rheumatol. 2025 Feb;43(2):316-325. doi: 10.55563/clinexprheumatol/zvffa0. Epub 2025 Feb 26.

Drugs

Indications