Table 2.
MicroRNA-directed clinical trials.
| Study Title (NCT) | miR under investigation | Disease(s) | Status |
|---|---|---|---|
| A Multicenter Phase I Study of MRX34, MicroRNA miR-RX34 Liposomal Injection (NCT01829971) | miR-34 | Primary Liver Cancer, SCLC, Lymphoma, Melanoma, Multiple Myeloma, Renal Cell Carcinoma, NSCLC | Terminated (5 immune mediated adverse events) |
| A Phase 1, Open-Label Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of RG-012 for Injection, Including Its Effect on Renal microRNA-21, in Subjects With Alport Syndrome (NCT03373786) | miR-21 | Alport Syndrome | Completed |
| MesomiR 1: A Phase I Study of Intravenously Administered Epidermal Growth Factor Receptor -Targeted, EnGeneIC Delivery Vehicle (EDV)-Packaged, miR-16 Mimic (TargomiRs) for Patients With Malignant Pleural Mesothelioma (MPM) and Advanced Non-Small Cell Lung Cancer (NSCLC) Failing on Std Therapy (NCT02369198) | miR-16 | Mesothelioma and NSCLC | Completed |
| A Phase 1, Randomized, Double-blind, Placebo-controlled, Single and Multiple Ascending Dose-escalation Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Activity of MRG-110 Following Local Intradermal Injection After Skin Excisional Wound Creation in Normal Healthy Volunteers (NCT03603431) | miR-92a | Normal healthy volunteer | Completed |
| A Phase 2, Double-blind, Placebo-Controlled Study to Investigate the Efficacy, Safety and Tolerability of MRG-201 Following Intradermal Injection in Subjects With a History of Keloids (NCT03601052) | miR-92 | Keloids | Active, not recruiting |
| A Phase I/II, Randomized, Double-blind, Sham Control Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington Disease (NCT04120493) | miHTT | Huntington’s Disease | Recruiting |
| A Placebo-controlled, Double-blind, Randomized, Single Dose, Dose Escalating Trial in Healthy Men to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SPC3649 (Miravirsen) (NCT00688012) | miR-122 | Healthy volunteers | Completed |
| SOLAR: A Phase 2, Randomized, Open-label, Parallel-group, Active Comparator, Multi-center Study to Investigate the Efficacy and Safety of Cobomarsen (MRG-106) in Subjects With Cutaneous T-Cell Lymphoma (CTCL), Mycosis Fungoides (MF) Subtype (NCT03713320) | miR-155 | CTCL/Mycosis Fungoides | Active, not recruiting |
| A Multicenter Phase 1B Pharmacodynamics Study of MRX34, MicroRNA miR-Rx34 Liposomal Injection, in Patients With Advanced Melanoma and Biopsy Accessible Lesions (NCT02862145) | miR-34 | Melanoma | Withdrawn |