Niclosamide Enema for Active Distal Ulcerative Colitis: A Phase 1, Open-Label Study

Area of Science:

• Gastroenterology
• Pharmacology
• Inflammatory Bowel Disease Research

Background:

• Current treatments for mild-to-moderate distal ulcerative colitis (UC) are ineffective for a significant patient subset.
• Niclosamide, an established anti-helminthic drug with a strong safety profile, exhibits anti-inflammatory properties.
• Preclinical data support exploring niclosamide's potential in managing UC.

Purpose of the Study:

• To evaluate the safety and tolerability of niclosamide when formulated as an enema for patients with mild-to-moderate distal UC.
• To assess the preliminary efficacy of niclosamide enema in achieving clinical remission and improving endoscopic and histologic markers of UC.

Main Methods:

• A phase 1, open-label trial involving 17 patients with active UC who had relapsed on standard therapies.
• Participants received niclosamide enema (150 mg/60 mL) twice daily for six weeks.
• Safety was the primary endpoint, with clinical remission and endoscopic/histologic scores as secondary endpoints.

Main Results:

• Niclosamide enema was well-tolerated by all participants, with no serious adverse events reported.
• Mild adverse events were infrequent and deemed unrelated to the treatment.
• Clinical remission was observed in 59% of patients, with notable improvements in endoscopic and histologic scores.

Conclusions:

• Niclosamide enema demonstrates a favorable safety and tolerability profile for UC treatment.
• The findings suggest niclosamide enema can effectively improve clinical symptoms and disease markers in UC patients.
• Further investigation through placebo-controlled trials is warranted to confirm the efficacy of niclosamide enema.