Clinical trial: the safety and short-term efficacy of recombinant cholera toxin B subunit in the treatment of active Crohn's disease.
BACKGROUND
The cholera toxin B subunit ameliorates experimentally induced colitis in mice. In humans, cholera toxin B subunit has never been tested in the treatment of Crohn's disease (CD).


OBJECTIVE
To evaluate the safety and efficacy of treatment with recombinant cholera toxin B subunit of patients with CD.


METHODS
An open-label, multicentre, nonrandomized trial including 15 patients with mild/moderate CD. Patients received an oral solution of 5 mg recombinant cholera toxin B subunit three times weekly for 2 weeks. Reduction in CD Activity Index (CDAI) with >100 between baseline and days 15, 29, 42 and 70 defined clinical response. Patients with CDAI score < or = 150 were defined as being in remission.


RESULTS
A significant decrease in CDAI score was observed. Response rates were 40% in the full analysis set and 42% in the per protocol analysis. Two patients receiving adjuvant treatment after day 29 were excluded, after which 40% were in remission at 4 weeks and 30% at 8 weeks post-treatment. Mild side effects (arthralgia, headache and pruritus) were seen in 33% of patients.


CONCLUSIONS
Treatment with recombinant cholera toxin B subunit was safe. Approximately 40% of patients with active CD responded to treatment. Randomized studies are needed to establish the clinical efficacy of recombinant cholera toxin B subunit.
Stål
P
P
Department of Gastroenterology and Hepatology Karolinska University Hospital, S-141 86 Stockholm, Sweden. per.stal@karolinska.se
Befrits
R
R
Rönnblom
A
A
Danielsson
A
A
Suhr
O
O
Ståhlberg
D
D
Brinkberg Lapidus
A
A
Löfberg
R
R
D000293:Adolescent; D000328:Adult; D000368:Aged; D002772:Cholera Toxin / Q000008:administration & dosage* / Q000009:adverse effects; D016001:Confidence Intervals; D003424:Crohn Disease / Q000188:drug therapy*; D004305:Dose-Response Relationship, Drug; D005260:Female; D005765:Gastrointestinal Agents / Q000008:administration & dosage* / Q000009:adverse effects; D006801:Humans; D008297:Male; D008875:Middle Aged; D018570:Risk Assessment; D016896:Treatment Outcome; D055815:Young Adult
D005765:Gastrointestinal Agents; D002772:Cholera Toxin