Efficacy and Safety of Miltefosine in Cutaneous Mastocytosis

INTERVENTION: Trade Name: Miltex® Pharmaceutical Form: Cutaneous liquid INN or Proposed INN: MILTEFOSINE CAS Number: 58066856 Other descriptive name: Hexadecylphosphocholine Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 60‐ Trade Name: Dermoxinale® Pharmaceutical Form: Cutaneous liquid INN or Proposed INN: CLOBETASOL PROPIONATE CAS Number: 25122467 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 0.05‐ Trade Name: Hametum® Extrakt Pharmaceutical Form: Cutaneous liquid CAS Number: 8001001169 Other descriptive name: HAMAMELIS VIRGINIANA Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 25‐ CONDITION: chronic stable symptomatic maculopapulous cutaneous mastocytosis or systemic mastocytosis with involvement of the skin and with positive Darier´s sign ; MedDRA version: 9.1 Level: LLT Classification code 10012812 Term: Diffuse cutaneous mastocytosis PRIMARY OUTCOME: Main Objective: To determine the efficacy of miltefosine compared to placebo; ; [note: placebo in this clinical trial is being defined as a liquid reference product used for skin‐care in dermatology with similar appearance as miltefosine and with no effect on cutaneous mastocytosis (negative control) (Hametum® Extrakt, see PR3).] Primary end point(s): clinical evaluation of treatment response comparing miltefosin to placebo. Evaluation of mechanically induced changes of lesions (Darier´s Sign) by the investigator using a composite score (Maximum = 9 points) evaluating wheal, erythema and itching each on a 4 point scale (0=no, 1=mild, 2=moderate, 3= severe). Secondary Objective: To determine the efficacy of miltefosine compared to Dermoxinale®; To determine the safety of miltefosine compared to placebo and Dermoxinale®; ; [note: placebo in this clinical trial is being defined as a liquid reference product used for skin‐care in dermatology with similar appearance as miltefosine and with no effect on cutaneous mastocytosis (negative control) (Hametum® Extrakt, see PR3).] INCLUSION CRITERIA: ‐) Chronic stable symptomatic maculopapulous cutaneous mastocytosis or systemic mastocytosis with involvement of the skin and with positive Darier’s Sign ‐) 3 comparable lesional areas of at least 50 cm2 excluding areas in the face and intertriginous areas ‐) Otherwise healthy according to physical examination ‐) Aged >=18 years ‐) Reliable method of contraception for women of childbearing potential (i.e. low failure rate less than 1% per year) ‐) Informed consent signed and dated 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