E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10067467 |
E.1.2 | Term | Nephrogenic systemic fibrosis |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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
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E.2.2 | Secondary objectives of the trial |
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 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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.
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E.4 | Principal 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)
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E.5 End points |
E.5.1 | 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. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |