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Thalidomide in Pediatric Inflammatory Bowel Diseases. (TALIBDP)

This study has been completed.
Sponsor:
Collaborators:
Ospedale Meyer
Pediatric Gastroenterology Unit, IRCCS Gaslini, Genoa.
Vittore Buzzi Children's Hospital
University of Pisa
Pediatric Gastroenterology Unit, University of Messina.
Università degli Studi di Brescia
University of Trieste
Information provided by (Responsible Party):
Alessandro Ventura, IRCCS Burlo Garofolo
ClinicalTrials.gov Identifier:
NCT00720538
First received: July 18, 2008
Last updated: October 25, 2012
Last verified: October 2012
  Purpose

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.


Condition Intervention Phase
Inflammatory Bowel Diseases
Crohn's Disease
Ulcerative Colitis
Drug: Thalidomide
Drug: placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized Controlled Double-blind Vs. Placebo Multicentre Study on the Safety and Effectiveness of Thalidomide in the Treatment of Refractory Crohn's Disease and Ulcerative Colitis.

Resource links provided by NLM:


Further study details as provided by IRCCS Burlo Garofolo:

Primary Outcome Measures:
  • Clinical Remission [ Time Frame: 8°, 12°, 26, 52° weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Clinical response [ Time Frame: 4°, 8° weeks ] [ Designated as safety issue: No ]
  • Steroid dose reduction [ Time Frame: 8°, 12°, 26, 52° weeks ] [ Designated as safety issue: No ]
  • Endoscopic remission [ Time Frame: 12°, 26, 52° weeks ] [ Designated as safety issue: No ]
  • Adverse effects [ Time Frame: 4°, 8°, 12°, 26, 52° weeks ] [ Designated as safety issue: Yes ]
  • Nutritinal indicators [ Time Frame: 8°, 52° ] [ Designated as safety issue: No ]
    Weight-on-age z-scores (WAZ) 8°, 52° Body Mass Index (BMI) z-scores 8°, 52° Height-on-age z-scores (HAZ) 52°


Enrollment: 84
Study Start Date: August 2008
Study Completion Date: June 2012
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Thalidomide
Drug: Thalidomide
Thalidomide 1.5-2.5 mg/kg/die once a day, at evening.
Other Names:
  • Thalidomide PHARMION
  • Thalidomid CELGENE
Placebo Comparator: 2
Placebo
Drug: placebo
placebo capsules

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   2 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00720538

Locations
Italy
Department of Pediatrics, Ospedale Meyer.
Florence., Florence, Italy
Anatomy and Hystology, Spedali Civili di Brescia, University of Brescia.
Brescia, Italy
Pediatric Gastroenterology Unit, IRCCS Gaslini.
Genoa, Italy
Pediatric Gastroenterology Unit, University of Messina.
Messina, Italy
Department of Pediatrics, Ospedali Buzzi.
Milan., Italy
Gastroenterology and Hepatology, University of Pisa.
Pisa, Italy
Unit of Biochemistry and Pharmacology, University of Triest.
Triest, Italy
Sponsors and Collaborators
IRCCS Burlo Garofolo
Ospedale Meyer
Pediatric Gastroenterology Unit, IRCCS Gaslini, Genoa.
Vittore Buzzi Children's Hospital
University of Pisa
Pediatric Gastroenterology Unit, University of Messina.
Università degli Studi di Brescia
University of Trieste
Investigators
Study Chair: Alessandro Ventura, MD IRCCS Burlo Garofolo
Study Director: Marzia Lazzerini, MD IRCCS Burlo Garofolo
  More Information

Publications:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alessandro Ventura, MD, IRCCS Burlo Garofolo
ClinicalTrials.gov Identifier: NCT00720538     History of Changes
Other Study ID Numbers: TAL−005414−20
Study First Received: July 18, 2008
Last Updated: October 25, 2012
Health Authority: Italy: Ministry of Health
Italy: National Monitoring Centre for Clinical Trials - Ministry of Health
Italy: Agenzia Italiana del Farmaco (AIFA ) Funds for Industry-Indipendent Research (contract with sponsor signed in September 2007)

Keywords provided by IRCCS Burlo Garofolo:
Inflammatory bowel diseases.
Crohn disease.
Ulcerative, colitis.
Randomised controlled trial.
Therapy.
Thalidomide.
Children.

Additional relevant MeSH terms:
Colitis
Colitis, Ulcerative
Crohn Disease
Inflammatory Bowel Diseases
Intestinal Diseases
Ulcer
Colonic Diseases
Digestive System Diseases
Gastroenteritis
Gastrointestinal Diseases
Pathologic Processes
Thalidomide
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Anti-Bacterial Agents
Anti-Infective Agents
Antineoplastic Agents
Growth Inhibitors
Growth Substances
Immunologic Factors
Immunosuppressive Agents
Leprostatic Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 25, 2014