Table 1 Current clinical applications targeting miRNAs in human

miR-122

NCT01646489

Miravirsen Telaprevir

Hapatitis C chronic hepatitis C

To assess the safety, tolerability, and affect on blood levels of miravirsen and telaprevir when co-administered miravirsen and telaprevir in healthy subjects. <Phase 1>

NCT01872936

Miravirsen Telaprevir Ribavirin

Chronic hepatitis C (genotype1) Null responders to treatment with peg IFNα/RBV therapy.

To assess the safety, tolerability, antiviral activity, genotype resistance associated with virological failure, pharmacokinetics and pharmacodynamics of two dose regimens of miravirsen in combination with telaprevir and ribavirin in subjects with hepatitis C virus genotype 1 infection. <Phase 2>

NCT01200420

Miravirsen

Hepatitis C

1. Determining the safety and tolerability of multiple dosing of miravirsen in subjects infected with chronic hepatitis C.

Saline

2. Assessing of pharmacokinetics of miravirsen and assessment of miravirsen's effect on HCV viral titer. <Phase 2>

NCT01727934

Miravirsen

Hepatitis C (genotype1) Null responders to treatment with peg IFNα/RBV therapy.

To aseess the safety, antiviral activity, and pharmacokinetics of 9 subcutaneous injections of miravirsen monotherapy over a total of 12 weeks of treatment.<Phase 2>

NCT00688012

SPC3649

Hepatitis C

A placebo-controlled, double-blind, randomized, single dose, dose escalating trial in healthy men to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of SPC3649. <Phase 1>

miR-34

NCT01829971

MRX-34

Primary HCC metastatic liver cancer

Evaluating the safety of MRX34 in patients with primary liver cancer or those with liver metastasis from other cancers. <Phase 1>