Thalidomide in Pediatric Inflammatory Bowel Diseases.

• Status: Completed
• Trial end date: 2012-06-01
• Target enrollment: 0
• Participant gender: All

Summary

Several open-label studies reported thalidomide efficacy in inducing clinical remission and steroid tapering in refractory Inflammatory Bowel diseases (IBD), both in adults and in children.

This is a randomized placebo controlled (RCT) double blind study, to evaluate the efficacy of thalidomide in inducing clinical remission at 8 weeks in refractory IBD patients aged 2-20 years.

The primary hypotheses of the study is that thalidomide would be more effective than placebo in inducing clinical remission.

The RCT phase is followed by a open-label phase, to further evaluate efficacy and safety of thalidomide in thalidomide responders, with a total follow up of one year.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: IRCCS Burlo Garofolo
• Collaborators: Ospedale Meyer; Pediatric Gastroenterology Unit, IRCCS Gaslini, Genoa.; Università degli Studi di Brescia; University of Messina; University of Pisa; University of Trieste; Vittore Buzzi Children's Hospital
• Treatments: Thalidomide

Criteria

Inclusion Criteria:

• Children, adolescents and young adults aged 2 to 20 with chronic refractory moderate to severe inflammatory bowel disease, referred by the six pediatric gastroenterological centres participating in the study.

Definition of patient with refractory disease:

• Patients with active disease despite steroid therapy (prednisone at a dose 2 mg/kg/die, maximum 60mg/day, or equivalent) for 8 weeks and/or an immunosuppressive of proven efficacy (azathioprine o 6-mercaptopurine for 4 months; methotrexate for 3 months; Infliximab at the dose of 5 mg/kg at week 0,2,6 weeks; cyclosporine at the dose of 2mg/kg/day for 4 weeks or 1 week at 1m/kg/day EV) or patients exhibiting intolerance to these drugs which prevent them from continuing treatment.

Exclusion Criteria:

• Patients with ileostomy or colostomy.

• Disease requiring immediate surgical intervention.

• Severe ulcerative colitis or toxic megacolon.

• Contraindications to using thalidomide (on-going pregnancy, neuropathy) .

• Any of the following conditions: active infection, stool culture positive for enteric pathogens, tumors, HIV, transplanted organ, or non-controlled disease of the kidney, liver, endocrine system, heart, blood, nervous system or brain.

• Patients being treated with other drugs as part of an experimental study.

• Patients treated with infliximab in the previous eight weeks.