Sarcoidosis with cutaneous affections
Conditions
Interventions
Trade Name: Rapamune
Pharmaceutical Form: Oral solution
INN or Proposed INN: SIROLIMUS
CAS Number: 53123-88-9
Current Sponsor code: -
Other descriptive name: Rapamune
Concentration unit: mg/ml milligr
Sponsors
Medical University of Vienna
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Patients of full age (18 – 80 years old) that fulfil the ATS/ERS/WASOG diagnostic criteria for sarcoidosis and have a progressive disease and skin involvement (more than 3x3cm). 2) Histologically proven sarcoidosis, diagnosed = 6 year prior to screening 3) If immunosuppressants are taken at the time of screening, doses of these medications have to remain stable for =1 month (no additional immunosuppressants, no major alterations of the dosages) prior to the screening visit. During the study, background medication regimen and doses should remain stable until month 8, when tapering of oral glucocorticosteroids can be started. 4) Written informed consent 5) Compliance of the patients to follow instructions and appointments of the study 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 10 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: 1) Hypersensitivity / intolerance against sirolimus or other components of the medication, patients with an allergy against peanuts or soya 2) Pregnancy, Lactation 3) Concurrent malignancy 4) Chronic or serious infections within 2 months prior screening 5) Transplantation of an organ in the medical history
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate treatment efficacy of sirolimus as improvement or stabilization of disease in patients with sarcoidosis;Secondary Objective: To assess safety and tolerability of sirolimus in patients with sarcoidosis To assess immunological signature(s) and baseline mTOR activity as biomarkers identifying patients as responders to sirolimus or enabling monitoring of response to sirolimus by flow cytometry (skin, blood), immunofluorescence (skin), genome, epigenome and transcriptome sequencing (skin, blood) on single cell level. ;Primary end point(s): Phase 1: a. absolute change in cutaneous Sarcoidosis Activity Index after two months of topic treatment and baseline Phase 2: a. absolute change in pulmonary function (FVC) after four months of systemic treatment and baseline b. absolute change in cutaneous Sarcoidosis Activity Index after four months of systemic treatment and baseline.;Timepoint(s) of evaluation of this end point: 2, 3, 5, 6, 10, 12 months after baseline visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Health-related quality of life (>20%), Fatigue scale (rise of >2 points), Corticosteroid dose reduction (>50%). Inhibition of mTOR pathway by sirolimus in peripheral blood and the skin on a single cell level Immunological signature(s) by genome, epigenome and transcriptome sequencing Baseline mTOR activity identifying patients as responders or enabling monitoring of response to sirolimus by flow cytometry and immunofluorescence;Timepoint(s) of evaluation of this end point: 2, 3, 5, 6, 10, 12 months after baseline visit | — |
Countries
Austria
Contacts
Public ContactGeorg Stary
Medical University of Vienna
Outcome results
None listed