NCT00479934

Efficacy and Safety of Imatinib in Scleroderma

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 Involvement.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 28 (actual)
• Sponsor: University Hospital, Bordeaux · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Scleroderma, Localized
• Scleroderma, Systemic

Interventions

Type	Name	Description
DRUG	imatinib mesylate	6 month treatment with 400mg/day (per os)

Timeline

• Start date: 2007-12-01
• Primary completion: 2010-06-01
• Completion: 2010-12-01
• First posted: 2007-05-30
• Last updated: 2011-03-04

Locations

12 sites across 1 country: France