GED-0301 is an antisense oligodeoxynucleotide with a sequence complementary to the Smad7 mRNA transcript. Smad7 is a negative regulator of transforming growth factor-β, which is increased in the intestinal mucosa of patients with active Crohn’s disease (CD). We randomly assigned 63 CD patients to 4-, 8-, or 12-week treatment groups receiving oral GED-0301 (160 mg/day). The primary objective was to determine GED-0301’s effect on endoscopic CD measures; secondary objectives included effects on clinical activity. Endoscopic improvement was observed in 37% of participants with evaluable endoscopy results at week 12. At week 12, 32% (4 weeks), 35% (8 weeks), and 48% (12 weeks) of patients receiving GED-0301 were in remission (CD activity index score <150); corresponding reductions from baseline in mean CD activity index scores were −124, −112, and −133 points. No new safety signals were observed. These findings support a GED-0301 benefit in active CD. ClinicalTrials.gov no: NCT02367183.

GED-0301是一种反义寡脱氧核苷酸，具有与Smad7互补的序列mRNA转录物。Smad7是转化生长因子-β的负调节剂，其在患有活动性克罗恩病（CD）的患者的肠粘膜中增加。我们将63名CD患者随机分配到接受口服GED-0301（160 mg /天）治疗的4周，8周或12周治疗组中。主要目的是确定GED-0301对内窥镜CD测量的影响。次要目标包括对临床活动的影响。在第12周时，有37％的内窥镜检查结果可观察到内窥镜改善，在第12周时，接受GED-0301的患者中有32％（4周），35％（8周）和48％（12周）的患者接受了内镜检查。缓解（CD活动指数评分<150）；CD活动指数均值相对于基线的相应降低是-124，-112和-133点。没有观察到新的安全信号。这些发现支持了有源CD的GED-0301优势。ClinicalTrials.gov编号：NCT02367183。