Table 1.
Recent clinical trials for IBM in the USA and European Union (2000–present).
| Therapeutic | Start Date | Completion Date | Publication Date | Type of Trial | End Point | Ref. |
|---|---|---|---|---|---|---|
| Pharmacological | ||||||
| ABC008 | 2021 | N/A * | N/A * | Phase I; open-label | Safety and tolerability | [26] |
| Alemtuzumab | 2004 | 2007 | 2009 | Phase II | Efficacy and safety | [6,7] |
| Anakinra | 2003 | 2008 | N/A | Phase II/III; non-randomized, open-label, non-placebo-controlled | Efficacy | [24] |
| Anakinra | N/A | N/A | 2013 | Pilot study; open-label, uncontrolled | Efficacy | [23] |
| Antithymocyte Ig+MTX versus MTX alone | N/A | N/A | 2003 | Pilot study; randomized, open-label, non-placebo-controlled | Efficacy | [12] |
| Arimoclomol | 2019 | N/A * | N/A * | Phase III (extension); open-label non-randomized | Efficacy | [19] |
| Arimoclomol | 2019 | N/A * | N/A * | Phase III; open-label, non-randomized | Efficacy and safety of early vs late start of therapy | [27] |
| Arimoclomol | 2018 | N/A * | N/A * | Phase II/III; randomized, double-blind, placebo-controlled | Efficacy | [28] |
| Arimoclomol | 2017 | 2021 | N/A | Phase II; randomized, double-blind, placebo-controlled | Efficacy | [21] |
| Arimoclomol | 2009 | 2012 | 2017 | Phase II; randomized, double-blind, placebo-controlled | Safety and tolerability | [22] |
| Arimoclomol | 2008 | 2012 | 2017 | Phase II/III; randomized, double-blind, placebo-controlled | Efficacy and safety | [18,20] |
| Baricitinib | 2020 | N/A * | N/A * | Phase IIa; randomized, controlled | Assessment of clinical response across 12- and 24-week treatment arms | [29] |
| Botulism | 2014 | 2018 | 2021 | Phase II; open-label | Alleviating dysphagia | [30] |
| BYM338 (Bimagrumab) | 2015 | 2017 | 2018 | Phase IIb/III; randomized, double-blind, placebo-controlled (extension) | Efficacy, safety and tolerability | [31] |
| BYM338 | 2015 | 2016 | 2018 | Phase IIb/III (extension); non-random, double-blind, placebo-controlled | Efficacy, safety and tolerability | [32,33] |
| BYM338 | 2014 | 2016 | 2018 | Phase II/III; open-label | Efficacy, safety and tolerability | [34,35] |
| BYM338 | 2014 | 2016 | 2017 | Phase IIb/III; randomized, double-blind, placebo-controlled | Efficacy, safety and tolerability | [36] |
| BYM338 | 2013 | 2016 | 2017 | Phase II/III; randomized, double-blind, placebo-controlled | Efficacy | [37,38] |
| BYM338 | 2011 | 2012 | 2014 | Phase II; randomized, double-blind, placebo-controlled | Efficacy, safety and tolerability | [39,40] |
| Etanercept | 2005 | 2014 | N/A | Phase I; randomized, double-blind, placebo-controlled | Efficacy | [41] |
| Etanercept | N/A | N/A | 2006 | Pilot study; non-randomized open-label, non-placebo-controlled | Efficacy | [42] |
| IFNβ1a (low-dose) | N/A | N/A | 2001 | Pilot study; randomized, double-blind, placebo-controlled | Efficacy, safety and tolerability | [43] |
| IFNβ1a (high-dose) | N/A | N/A | 2004 | Pilot study; randomized, double-blind, placebo-controlled | Efficacy, safety and tolerability | [44] |
| IVIg + prednisone | N/A | N/A | 2001 | Phase II; randomized, double-blind, placebo-controlled | Efficacy and safety | [15] |
| IVIg | 1990 | 2002 | 1997, 2001 | Phase II; double-blind, placebo-controlled | Efficacy and safety | [13,14,45] |
| Lithium | 2008 | 2009 | N/A | Pilot study; cohort | Efficacy | [46] |
| MTX | 1996 | 2000 | 2002 | Pilot study; randomized, double-blind, placebo-controlled | Efficacy | [16] |
| Natalizumab | 2013 | N/A | N/A | Phase I; open-label, non-placebo-controlled | Efficacy and safety | [47] |
| Oxandrolone | N/A | N/A | 2002 | Pilot study; randomized, double-blind, placebo-controlled | Efficacy | [48] |
| Phenylbutyrate | 2020 | N/A * | N/A * | Phase I; open-label | Efficacy, safety and tolerability | [49] |
| Pioglitazone | 2018 | 2020 | N/A | Pilot study; open-label, non-randomized, non-placebo-controlled | Efficacy | [50] |
| Rapamycin | 2015 | 2018 | N/A | Phase II/III; randomized, double-blind, placebo-controlled | Efficacy | [51] |
| Simvastatin | 2007 | N/A | N/A | Phase III; randomized, controlled | Efficacy, safety and tolerability | [52] |
| Simvastatin | 2007 | N/A | N/A | Phase III; randomized, controlled, | Efficacy, safety and tolerability | [53] |
| Sirolimus | N/A * | N/A * | N/A * | Phase III; randomized, double-blind, placebo-controlled | Efficacy | [54] |
| Cell-based | ||||||
| Adipose-derived stem cells | 2021 | N/A * | N/A * | Open-label; non-random | Efficacy and safety | [55] |
| Adipose-derived stromal vascular fraction | N/A * | N/A * | N/A * | Phase I; open-label, non-placebo-controlled | Efficacy, safety and tolerability | [56] |
| Gene-based | ||||||
| Follistatin | 2012 | 2017 | N/A | Phase I; non-randomized, open-label, non-placebo-controlled | Efficacy and safety | [57,58] |
Efficacy measurements include molecular, histological/pathological and physical parameters. Abbreviations: Ig, immunoglobulin; INFβ1a, interferon β1a; IVIg, intravenous immunoglobulin; MTX, methotrexate; N/A, not applicable (information not available); N/A *, not applicable as study has not initiated or is ongoing.