Thalidomide in Pediatric Inflammatory Bowel Diseases.

NCT00720538 | Completed Phase 3 DMC

• 1 other identifier: TAL-005414-20
• Study Type: interventional
• Target: 84
• Locations: 1 country
• Sites: 7

Brief 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.

Conditions

Inflammatory Bowel Diseases; Crohn's Disease; Ulcerative Colitis

Keywords

Inflammatory bowel diseases.; Crohn disease.; Ulcerative, colitis.; Randomised controlled trial.; Therapy.; Thalidomide.; Children.

Outcome Measures

Primary Outcomes (1)

• Clinical Remission

  8°, 12°, 26, 52° weeks

Secondary Outcomes (5)

• Clinical response

  4°, 8° weeks

• Steroid dose reduction

  8°, 12°, 26, 52° weeks

• Endoscopic remission

  12°, 26, 52° weeks

• Adverse effects

  4°, 8°, 12°, 26, 52° weeks

• Nutritinal indicators

  8°, 52°

Study Arms (2)

1

EXPERIMENTAL

Thalidomide

Drug: Thalidomide

2

PLACEBO COMPARATOR

Placebo

Drug: placebo

Interventions

Thalidomide DRUG

Thalidomide 1.5-2.5 mg/kg/die once a day, at evening.

Also known as: Thalidomide PHARMION, Thalidomid CELGENE

1

placebo DRUG

placebo capsules

2

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• IRCCS Burlo Garofolo: lead
• Ospedale Meyer: collaborator
• Pediatric Gastroenterology Unit, IRCCS Gaslini, Genoa.: collaborator
• Vittore Buzzi Children's Hospital: collaborator
• University of Pisa: collaborator
• University of Messina: collaborator
• Università degli Studi di Brescia: collaborator
• University of Trieste: collaborator

Study Sites (7)

Anatomy and Hystology, Spedali Civili di Brescia, University of Brescia.

Brescia, Brescia, Italy

Location

Department of Pediatrics, Ospedale Meyer.

Florence, Florence, Italy

Location

Pediatric Gastroenterology Unit, IRCCS Gaslini.

Genoa, Genoa, Italy

Location

Pediatric Gastroenterology Unit, University of Messina.

Messina, Messina, Italy

Location

Department of Pediatrics, Ospedali Buzzi.

Milan, Milan., Italy

Location

Gastroenterology and Hepatology, University of Pisa.

Pisa, Pisa, Italy

Location

Unit of Biochemistry and Pharmacology, University of Triest.

Trieste, Triest, Italy

Location

Related Publications (2)

• Lazzerini M, Martelossi S, Marchetti F, Scabar A, Bradaschia F, Ronfani L, Ventura A. Efficacy and safety of thalidomide in children and young adults with intractable inflammatory bowel disease: long-term results. Aliment Pharmacol Ther. 2007 Feb 15;25(4):419-27. doi: 10.1111/j.1365-2036.2006.03211.x.

  PMID: 17269997 BACKGROUND

• Lazzerini M, Martelossi S, Magazzu G, Pellegrino S, Lucanto MC, Barabino A, Calvi A, Arrigo S, Lionetti P, Lorusso M, Mangiantini F, Fontana M, Zuin G, Palla G, Maggiore G, Bramuzzo M, Pellegrin MC, Maschio M, Villanacci V, Manenti S, Decorti G, De Iudicibus S, Paparazzo R, Montico M, Ventura A. Effect of thalidomide on clinical remission in children and adolescents with refractory Crohn disease: a randomized clinical trial. JAMA. 2013 Nov 27;310(20):2164-73. doi: 10.1001/jama.2013.280777.

  PMID: 24281461 DERIVED

MeSH Terms

Conditions

Inflammatory Bowel Diseases; Crohn Disease; Colitis, Ulcerative

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases; Colitis; Colonic Diseases

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Alessandro Ventura, MD

  IRCCS Burlo Garofolo

  STUDY CHAIR

• Marzia Lazzerini, MD

  IRCCS Burlo Garofolo

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: July 18, 2008
• First Posted: July 22, 2008
• Study Start: August 1, 2008
• Primary Completion: September 1, 2011
• Study Completion: June 1, 2012
• Last Updated: October 26, 2012

Record last verified: 2012-10

Locations

IT (7)