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Abstract
In the gut of patients with Crohn’s disease (CD), high Smad7 blocks the immune-suppressive activity of transforming growth factor (TGF)-β1, thereby contributing to amplify inflammatory signals. In vivo in mice, knockdown of Smad7 with a Smad7 antisense oligonucleotide (GED-0301) attenuates experimental colitis.
Here, we provide results of a phase 1 clinical, open-label, dose escalation study of GED-0301 in patients with active, steroid-dependent/resistant CD, aimed at assessing the safety and tolerability of the drug.
Patients were allocated to three treatment groups receiving oral GED0301 once daily for 7 days at doses of 40, 80, or 160 mg.
A total of 15 patients were enrolled. No serious adverse event was registered. GED-0301 was well tolerated and no patient dropped out during the study. Twenty-five adverse events were documented in 11 patients, the majority of whom were judged to be of mild intensity and unrelated to treatment. GED-0301 treatment reduced the percentage of inflammatory cytokine-expressing CCR9-positive T cells in the blood.
The study shows for the first time that GED0301 is safe and well tolerated in patients with active CD.