This study is designed to explore mechanism of action of mongersen (GED-0301) 160 mg once daily in patients with active Crohn's Disease
Subjects will be screened to provide 20 enrolled subjects who complete 12 weeks of mongersen (GED-0301) 160 mg QD treatment as open-label therapy. The study will consist of 4 periods: * Screening Period - up to 4 weeks * Induction Period - 12 weeks (Week 0 Visit through Week 12 Visit) * Maintenance Period - 88 weeks (after Week 12 Visit through Week 100 Visit) * Follow-up Period - 4 weeks (ie, no IP taken) Subjects who prematurely discontinue treatment from this study prior to Week 100 will have an Early Termination Visit and also enter the 4-week Follow-up Period. At the Screening Visit, all subjects who meet the entrance criteria will be eligible to enter the study. The number of subjects with previous exposure to Tumor Necrosis Factor-Alpha (TNF-α) blockers is targeted to be approximately 40% (ie, approximately 8 subjects).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
During Induction period patient will receive mongersen 160 mg daily for 12 weeks. This study also offers subjects the option to continue with maintenance treatment at the discretion of the Investigator, beginning after the Week 12 Visit with alternating no IP for 4 weeks, followed by mongersen (GED-0301) 160 mg QD for 4 weeks, up through the Week 52 Visit during the Maintenance Period. At Week 52, subjects who have achieved an endoscopic improvement of \>50% from baseline based on the SES-CD, as assessed by the central reader, and clinical improvement (HBI \<7), will have the option to continue receiving treatment for an additional year, up through Year 2 (ie, the Week 100 Visit).
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy
Policlinico Tor Vergata
Rome, Italy
Change from baseline of Smad7 (Mothers Against Decapentaplegic homolog 7) expression in the intestinal mucosa
Smad7 is a protein important in cell interactions and stands for "Mothers against decapentaplegic homolog 7" protein
Time frame: Baseline and Week 12
Change from baseline in messenger ribonucleic acid (mRNA) expression of inflammatory cytokines
Change from baseline in the mRNA expression of inflammatory cytokines such as, but not limited to, interleukin (IL)-10, IL-25, chemokine (C-C motif) ligand 20 (CCL20) and tumor necrosis factor alpha (TNF-α) in the intestinal mucosa at Week 12, from biopsy samples during ileocolonoscopy
Time frame: Baseline and Week 12
The proportion of subjects achieving clinical remission, defined as a Crohn's Disease Activity Index (CDAI) score < 150, at Weeks 4, 8, and 12
The CDAI is a measure of disease activity in patients with Crohn's Disease.
Time frame: Up to week 12
Change from baseline in the Simple Endoscopic Score for Crohn's Disease (SES-CD) at Week 12 and 52
The SES-CD is a measure that indicates mucosal inflammation in the end part of the ileum (or small intestine) and colon (or large intestine) in patients with Crohn's Disease
Time frame: Week 12 and week 52
Number of Adverse Events
The evaluation of safety and tolerability of GED-0301, assessed by the type, frequency and severity of adverse events, and its relationship to investigational product (IP), discontinuation due to adverse events, and clinically significant changes in electrocardiograms (ECGs), vital signs, and/or laboratory findings
Time frame: Up to Week 56
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