A Phase 3 interventional study of Thalidomide and Infliximab in Crohn Disease, sponsored by IRCCS Burlo Garofolo. Terminated at 6 sites in Italy. Open to participants aged 6 Years to 17 Years. Per ClinicalTrials.gov, last updated 2020-09-04.
Sponsored by IRCCS Burlo Garofolo · Phase 3, Interventional, and Treatment
Crohn's disease (CD) is a life-long inflammatory bowel disease disease with an unknown pathogenesis. The ultimate goal of therapy is to modify the natural history of CD thus reducing complications. Thalidomide is a small molecule with immunomodulatory and anti-angiogenetic properties. It is currently approved for the treatment of erythema nodosum leprosum, an immunological complication of leprosy and multiple myeloma. It has also been used in several other inflammatory diseases of the skin and of the mucosal membranes, such as Behcet disease, oropharyngeal ulcers in AIDS, cutaneous lupus, and graft versus host disease. Many case series and one pediatric randomized controlled trial proved the efficacy of thalidomide in the treatment of children with CD refractory to standard treatments. In these patients, clinical remission was achieved in about 50% of the cases and was maintained for a mean time superior of 3 years. Mucosal healing after 52 weeks of treatment was observed in 40% of the patients in clinical remission. Moreover, thalidomide was found to have a steroid-sparing effect and to decrease the need for surgical interventions. The clinical and endoscopic efficacy of thalidomide was also observed in children with failure to respond or intolerance to anti-TNF biological drugs.
The aim of this multicentric prospective randomized controlled is to evaluate the efficacy and safety of thalidomide vs infliximab in changing the natural history of CD in patients with poor prognostic outcome. Moreover, the study will evaluate the immunological and genetical mechanisms of CD, the mechanisms of action thalidomide in CD and will the pharmacokinetics, metabolomics and pharmacogenomics of thalidomide, and their impact on thalidomide safety and effectiveness.
1,880 studies on the registry are indexed under Crohn Disease; 462 are open to participants now.
This study's enrollment of 9 is below the median of 66 across 1,188 interventional studies indexed under Crohn Disease.
Browse Crohn Disease studies →IRCCS Burlo Garofolo is the lead sponsor of 87 studies on the registry; 32 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Presence of at least one of the following risk factors for poor prognosis:
Exclusion Criteria:
Thalidomide is a immunomodulatory and antiangiogenetic drug with anti tumor necrosis factor (TNF) alpha properties
Drug: Thalidomide
Infliximab is a chimeric monoclonal antibody against TNF alpha
Drug: Infliximab
Thalidomide is a immunomodulatory and antiangiogenetic drug with anti TNF alpha properties
Infliximab is a chimeric monoclonal antibody against TNF alpha
Efficacy in inducing mucosal healing
Proportion of patients that achieve mucosal healing, defined by a Simplified Endoscopic Activity Index for CD (SES-CD) ≤ 2.
Time frame: 52 weeks
Efficacy in inducing clinical response
Clinical response will be evaluated with the weighted Pediatric Crohn's Disease Activity Index (wPCDAI), defined by a reduction of wPCDAI \> 50% from the basal values.
Time frame: 12 weeks
Efficacy in inducing clinical response
Clinical response will be evaluated with the weighted Pediatric Crohn's Disease Activity Index (wPCDAI), defined by a reduction of wPCDAI \> 50% from the basal values.
Time frame: 52 weeks
Efficacy in inducing clinical remission
Clinical remission will be evaluated with the weighted Pediatric Crohn's Disease Activity Index (wPCDAI), defined by a wPCDAI \<12.5.
Time frame: 12 weeks
Efficacy in inducing clinical remission
Clinical remission will be evaluated with the weighted Pediatric Crohn's Disease Activity Index (wPCDAI), defined by a wPCDAI \<12.5.
Time frame: 52 weeks
Efficacy in reducing the need to change therapy
Evaluation of the proportion of patients that need a therapeutic change
Time frame: 12 weeks
Efficacy in reducing the need to change therapy
Evaluation of the proportion of patients that need a therapeutic change
Time frame: 52 weeks
Efficacy in reducing hospitalizations
Evaluation of the proportion of patients that need hospitalization.
Time frame: 52 weeks
Efficacy in reducing the need for surgery
Evaluation of the proportion of patients that need surgery
Time frame: 52 weeks
Efficacy in reducing erythrocyte sedimentation rate
Evaluation of the trend of erythrocyte sedimentation rate (ESR)
Time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks)
Efficacy in reducing C-reactive protein
Evaluation of the trend of C-reactive protein (CRP)
Time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks)
Efficacy in reducing faecal calprotectin
Evaluation of the trend of faecal calprotectin
Time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks)
Efficacy in modifying body mass index
Evaluation of the trend of body mass index, defined as weight (kg)/height (m)\^2
Time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks)
Efficacy in modifying height-for-age z score
Evaluation of the trend of height-for-age z score
Time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks)
Efficacy in modifying weight-for-age z score
Evaluation of the trend of weight-for-age z score
Time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks)
Evaluation of the Treatment-Emergent Adverse Events
Number and type
Time frame: Between enrolment and 52 weeks
Direct and indirect costs
Comparison of direct and indirect costs (i.e. drugs, medical supplies and equipment, laboratory and diagnostic tests, hospitalizations, visits, transportation to and from healthcare facilities, missing work and school days...) between the two groups
Time frame: 52 weeks
Plan to share: No
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This study is terminated, as verified in Sep 2020. You cannot join it, but the record below documents what was studied.
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IRCCS Burlo Garofolo