Inflammatory Bowel Disease Crohn Disease, Ulcerative Colitis MedDRA version: 6.1 Level: PT Classification code 10021972
Conditions
Interventions
Trade Name: Thalidomide Pharmion
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Thalidomide
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Pharmaceuti
Sponsors
ISTITUTO PER L INFANZIA BURLO GAROFOLO
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Children, adolescents and young adults aged 2 to 20 with chronic refractory moderate to severe inflammatory bowel disease, referred by the seven pediatric centres participating in the study. Definition of patient with refractory diseasePatients with active disease despite steroid therapy at a dose 2 mg/kg/die for 8 weeks of prednisone maximum 60mg/day or equivalent 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. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: a Patients with ileostomy or colostomy. b Disease requiring immediate surgical intervention. c Severe ulcerative colitis or toxic megacolon. d Contraindications to using thalidomide on-going pregnancy, neuropathy e 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. f Patients being treated with other drugs as part of an experimental study. g Patients treated with infliximab in the previous eight weeks.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy and tolerability of thalidomide vs. placebo in children, adolescents, and young adults with untreatable chronic inflammatory bowel disease.;Secondary Objective: pharmacogenetic study of thalidomide effect;Primary end point(s): Clinical remission for Crohn s disease PCDAI 10 o CDAI 150; for ulcerative colitis UCAI 4. Relapse for Crohn s disease PCDAI 12 o CDAI 180; for ulcerative colitis UCAI 6. Endoscopic response reduction of endoscopic activity scores CDEIS, UCEAI . Side effects Neuropathy/Others resulting in thalidomide suspension/Others. | — |
Countries
Italy
Outcome results
None listed