Table 4.
Study drug | Study | Phase | Study population | Study design | Cohort size | Drug dosage and frequency of doses | Primary endpoint | Results |
---|---|---|---|---|---|---|---|---|
STNM01 | Phase 1 clinical study of siRNA targeting carbohydrate sulfotransferase 15 in Crohn's disease patients with active mucosal lesions (Suzuki et al., 2017) | Phase 1 | CD patients with active endoscopic disease (SES-CD: from 1 to 11) | Double-blind, placebo-controlled study | 18 CD patients | The patients were randomized to four different treatment arms: STNM01 at a dosage of 2.5 nM, STNM01 at a dosage of 25 nM, STNM01 at a dosage of 250 nM or placebo arm. The drug was administered endoscopically with a single dose submucosal injection | To evaluate the safety of STNM01 | The study evidenced the safety of the drug and reduced the segmental SES-CD score |
SES-CD, Simple Endoscopic Score for Crohn's disease; siRNA, silenceRNA; CD, Crohn's disease.