Table 4.
Clinical trials of miRNAs with therapeutic potential in cardiovascular disease.
| Active Principle/Therapeutic Drug Name | Indication | Clinical Phase | Study No./Status | Preclinica/Clinical Study Outcomes | Corporate Sponsor | Related Cardiovascular Studies |
|---|---|---|---|---|---|---|
| miR-132-3p inhibitor (CDR132L) | Stable heart failure | Phase I | NCT04045405 (completed) | [28,170] | Cardior Pharmaceuticals | [28,31,170,171] |
| miR-122-5p inhibitor (miravirsen) | HCV | Phase I Phase I Phase I Phase IIa |
NCT00688012 (completed), NCT00979927 (completed), NCT01646489 (completed), NCT01200420 EudraCT 2010-019057-17 (completed) | [160,172,173] | Santaris Pharma | [160,172,173] |
| miR-103/107-3p inhibitor (AZD4076) | T2D with NAFLD T2D with NASH | Phase I/IIa Phase I |
NCT02826525 (halted for strategic reasons) NCT02612662 (halted for strategic reasons) | AstraZeneca | [174] | |
| miR-122-5p inhibitor (RG-101) |
HCV | Phase II PhaseII PhaseIIb Phase IIb | EudraCT 2015-004702-42 (completed), EudraCT 2015-001535-21 (completed), EudraCT 2013-002978-49 (completed), EudraCT 2016-002069-77 (completed) | [175,176] | Regulus Therapeutics | |
| miR-16-5p mimic (TargomiR) | Malignant pleural mesothelioma | Phase I | NCT02369198 (completed) | [177] | Asbestos Diseases Research Foundation | |
| miR-17-5p inhibitor (RGLS4326) | ADPKD | Phase Ib | NCT04536688 (completed) | [178] | Regulus Therapeutics | [179] |
| miR-155-5p inhibitor cobomarsen (MRG-106) | Cutaneous T-cell lymphoma | Phase I Phase II |
NCT02580552 (completed) NCT03713320 (terminated for strategic reasons |
[180] | miRagen Therapeutics (now Viridian Therapeutic) | [32,34] |
| miR-92a-3p inhibitor (MRG-110) | Wound healing | Phase I Phase I Phase I |
NCT03603431 (completed) NCT03494712 (completed) EUDRA-CT 2017-004180-12 (completed) |
[181] | miRagen Therapeutics (now Viridian Therapeutic) | [45,85,134] |
| miR-21-5p inhibitor lademirsen (RG-012) | Alport’s syndrome | Phase I Phase II |
NCT02603224 (completed) NCT02855268 (ongoing) |
Genzyme/Sanofi | ||
| miR-29-3p mimic remlarsen (MRG-201) | Keloid scar formation | Phase I Phase II |
NCT02603224 (completed) NCT03601052 (completed) |
miRagen Therapeutics (now Viridian Therapeutic) | [17,22,23,173] | |
| miR-34a-5p mimic (MRX-34) | Advanced cancer | Phase I | NCT01829971 (terminated due to serious adverse effects) | [128] | Mirna Therapeutics | [177,182] |
The table summarizes 19 clinical trials that have used microRNA-based therapeutics. Abbreviations used in this text include the following: ADPKD, autosomal dominant polycystic kidney disease; T2D, type 2 diabetes; NAFLD, nonalcoholic fatty liver disease; NASH, nonalcoholic steatohepatitis; HCV, hepatitis C virus. Refs. [17,22,23,28,32,34,85,160,170,171,172,173,174,175,176,177,178,179,180,181,182].