Clinical Trial: Efficacy and Safety of Imatinib in Scleroderma

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase II Randomized Double Blind Clinical Trial of'Imatinib Mesylate STI571 (Glivec®) Versus Placebo in Patients With Severe Cutaneous Scleroderma or Systemic Sclerosis With Severe Cutaneous Invo

Brief Summary: In vitro studies have shown that imatinib 1mM inhibits strongly the growth of cutaneous fibroblasts. The hypothesis is that imatinib inhibits PDGFR which is known to be a potential target for the molecule, as recently also proposed after the discovery of autoantibodies activating the PDGF receptors. Recent data indicate that TGFb is also a potential target of imatinib. Cutaneous scleroderma is characterized by progressive cutaneous fibrosis caused by hyperactive dermal fibroblasts. Since no established treatment for skin sclerosis in scleroderma is currently available. This study will test the safety and efficacy of imatinib in the treatment of patients with scleroderma and severe cutaneous involvement.

Detailed Summary: This study will test the efficacy and tolerance of patients with a high score of induration (modified Rodnan score > 20/54) Comparison : 34 patients with severe forms of cutaneous involvement will be evaluated in a double blind RCT comparing imatinib 400mg/j and placebo in a 6 month period. Efficacy will be assessed using a cutaneous induration scale and skin biopsy, and quality of life questionnaires.
Sponsor: University Hospital, Bordeaux

Current Primary Outcome: Compare the efficacy of imatinib mesylate vs placebo based on the percent variation of modified Rodnan score (0-51) between inclusion and 6-month visits. [ Time Frame: 6 month ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Compare efficacy of imatinib mesylate vs placebo based on the percent variation of modified Rodnan score between the inclusion and the various time points of follow-up. [ Time Frame: 1, 3 and 12 month ]
  • Assess skin thickness at inclusion and at 6 months using skin biopsies [ Time Frame: 6 month ]
  • Assessment of quality of life using DLQI (Dermatology Quality of Life Index) and HAQ (Health Assessment Questionnaire). [ Time Frame: At 1, 3, 6 month and 1 year, ]
  • Assess tolerance of treatment (clinical and laboratory monitoring of side effects) [ Time Frame: All along the trial ]
  • Assess effects of treatment on non cutaneous symptoms in systemic sclerosis patients [ Time Frame: All along the trial ]


Original Secondary Outcome:

  • Compare efficacy of imatinib mesylate vs placebo based on the percent variation of modified Rodnan score between the inclusion and the various time points of follow-up.
  • Assess skin thickness at inclusion and at 6 months using skin biopsies
  • At 1, 3, 6 and 1 year, assessment of quality of life using DLQI (Dermatology Quality of Life Index) and HAQ (Health Assessment Questionnaire).
  • Assess tolerance of treatment (clinical and laboratory monitoring of side effects)
  • Assess effects of treatment on non cutaneous symptoms in systemic sclerosis patients


Information By: University Hospital, Bordeaux

Dates:
Date Received: May 29, 2007
Date Started: December 2007
Date Completion:
Last Updated: March 3, 2011
Last Verified: March 2011