Efficacy and Safety Study of Mongersen (GED-0301) for the Treatment of Subjects With Active Crohn's Disease

Summary

The purpose of study is to test the effects of an experimental medication GED-0301 (mongersen) in patients who have active Crohn's disease. The study will test GED-0301 compared to placebo for 52 weeks. The study treatment is blinded which means that patients and the study doctor will not know which treatment has been assigned. Patients in this study will be allowed treatment with stable doses of oral aminosalicylates, oral corticosteroids, immunosupressants and antibiotics for the treatment of Crohn's disease. After 12 weeks in the study until the end of the study, patients who do not have an improvement in their Crohns disease symptoms will have the option to enter a long term active treatment study. Participants who discontinued the study anytime or completed the study at Week 52 were then observed for an additional 4 weeks.

Timeline

Start

2015-12-08

Primary completion

2018-01-05

Completion

2018-01-05

Outcome

Missed primary endpoint

registry analysis (superiority test); Placebo vs GED-0301 160 mg / GED-0301 40 mg 4 Week Alt; p = 0.9141 NCT02596893 ↗

paper was attained in 22.8% of patients on GED-0301 vs 25% on placebo (P = 0.6210). PMID 31850931 ↗

Publications

• Background Gold SL, Cohen-Mekelburg S, Schneider Y, Steinlauf A. Perianal Fistulas in Patients With Crohn's Disease, Part 2: Surgical, Endoscopic, and Future Therapies. Gastroenterol Hepatol (N Y). 2018 Sep;14(9):521-528.
• Sands BE, Feagan BG, Sandborn WJ, Schreiber S, Peyrin-Biroulet L, Frederic Colombel J, Rossiter G, Usiskin K, Ather S, Zhan X, D'Haens G. Mongersen (GED-0301) for Active Crohn's Disease: Results of a Phase 3 Study. Am J Gastroenterol. 2020 May;115(5):738-745. doi: 10.14309/ajg.0000000000000493.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	MONGERSEN SODIUM	Antisense oligonucleotide	40 mg	Oral
Subject	MONGERSEN SODIUM	Antisense oligonucleotide	160 mg	Oral

Indications