Nephrogenic Systemic Fibrosis (NSF) is a fibrosing disease strongly associated with Gadolinium Based Contrast Agents(GBCA). Hard dermal plaques appear on legs, arms and abdomen. The lesions involve the deep part of the subcutis. The skin hardens and loses flexibility. The muscles are often affected too. Involvement of the joint capsulaes leads to contractures. Some patients ends up in a wheelchair. The inner vital organs may be affected and NSF can accelerate the patients death. MedDRA version:
Conditions
Interventions
Trade Name: Glivec / Gleevec (Imatinib Mesylate)
Pharmaceutical Form: Capsule*
Sponsors
Århus University Hospital, Department of Dermatology
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age > 18 years 2. Diagnosed with NSF 3. mRodnan skinscore => 20 or 4. Rapid progression of the disease defined as a 50 % increase in mRodnan skin score in less than 7 weeks or 5. Progression of the fibrosis in the inner organs and 6. No absolute contraindications to the treatment. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Exclusion Criteria : 1. Known sensitivity to Imatinib Mesylate or to any of its components 2. Pregnant or lactating woman 3. ALAT > 3 x upper limit of normal 4. Severe congestive heart failure(NYHA Class III or IV)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To offer the NSF patients with moderate to severe disease treatment with imatinib mesylate (Glivec) as no treatment so far has shown to be acceptable effective. Primary measurement will be evaluation of the skin with the mRodnan skinscore ;Secondary Objective: To follow these patients very closely with bloodsamples, biopsies, mRodnan skinscore and DLQI in order to control if the treatment is well tolerated and is effective to the patient. Skinbiopsies to see if there is any histopathological changes. DLQI to follow any changes in quality of life;Primary end point(s): To decelerate the progression of the disease and thereby soften the skin and improve the mobility of the joint contractures. One case report by Jonathan Kay has shown beneficial effect of Imatinib mesylate in 2 patients at the Massachusetts General Hospital. We hope that our studie can bring more knowledge about the treatment of this group of patients, whom today have very few treatment options. | — |
Countries
Denmark
Outcome results
None listed