Study on Mastocytosis for Rupatadine Treatment
Study on Mastocytosis for Rupatadine Treatment
Study title:

 An exploratory, randomised, double-blind, placebo controlled crossover study to assess the
 efficacy of 20 mg Rupatadine on the treatment of mastocytosis symptoms.

 Study code:

 SMART-2010-1 Principal Investigator, Co-Investigator, sponsor, and study centre (acc. to § 40
 Abs. 4 AMG) Dr. med. F. Siebenhaar, Prof. Dr. Med. M. Maurer, Allergie-Centrum-Charité,
 Department of Dermatology and Allergy, Charité - Universitätsmedizin Berlin, Charitéplatz 1,
 D-10117 Berlin Biometry and biostatistical analyses Division of Biostatistics and Clinical
 Epidemiology, Charité - Universitätsmedizin Berlin, Charitéplatz 1, D-10117 Berlin Monitoring
 Coordination Centre for Clinical Studies (KKS), Charité - Universitätsmedizin Berlin,
 Charitéplatz 1, D-10117 Berlin Clinical phase Phase II

 Primary objective:

 Reduction of wheal and flare type skin reaction after standardised provocation testing
 assessed by volumetric and thermographic measurements.

 Secondary objectives:

 Improvement of additional related symptoms (e.g. pruritus) and subjective affliction as
 assessed by symptom score, DLQI, Itchy-QoL and VAS.

 Study design:

 An exploratory, randomised, double-blind, placebo controlled crossover study

 Type and number of patients:

 Male and female patients (n = 30) with cutaneous mastocytosis and indolent systemic
 mastocytosis with skin involvement

 Main criteria for inclusion:

 Mastocytosis patients aged 18-65 years, signed written consent, no systemic corticosteroid or
 other immunosuppressive therapy, no permanent severe diseases Test product, dose and mode of
 administration 20 mg Rupatadine or placebo before provocation testing, oral administration
 (tablets)

 Duration of treatment:

 28 days (testing will be done at the day of last treatment)

 Assessment of efficacy:

 1. Assessment of wheal and flare development by planimetric analyses of digital
 photographic, volumetric, and thermographic imaging (time lapse) before and after
 treatment with study medication,

 2. Additional assessment of symptoms,

 3. Assessment of life quality

 Main criteria of evaluation:

 Efficacy
Inclusion Criteria:

 1. Chronic stable symptomatic maculopapulous cutaneous mastocytosis or indolent systemic
 mastocytosis with skin involvement and a positive Darier's Sign

 2. Age between 18 and 65 years.

 3. Female patients must be using a highly effective method of birth control (such as
 implants, injectables, combined oral contraceptives, some IUDs, sexual abstinence,
 vasectomised partner), or they must be postmenopausal, surgically sterilised, or
 hysterectomised.

 4. Voluntarily signed written informed consent.

 Exclusion Criteria:

 1. The presence of permanent severe diseases, especially those affecting the immune
 system, except for mastocytosis

 2. History or presence of epilepsy, significant neurological disorders, cerebrovascular
 attacks or ischemia

 3. History or presence of myocardial infarction or cardiac arrhythmia which requires drug
 therapy, hyper-/hypokalemia, bradycardia < 50bpm, QTc interval > 440ms

 4. Evidence of severe renal dysfunction (creatinine > 1,5 x upper reference value)

 5. Evidence of significant hepatic disease (liver enzymes > 2 x upper reference value)

 6. History of adverse reactions to RUP, or other ingredients of the IMP

 7. Presence of active cancer which requires chemotherapy or radiation therapy

 8. Aggressive systemic mastocytosis

 9. History or presence of alcohol abuse or drug addiction

 10. Participation in any clinical trial within 4 weeks prior to enrolment

 11. Commitment to an institution in terms of § 40 Abs. 1 S. 3 Nr. 4 AMG

 12. Intake of antihistamines or leukotriene antagonists within 7 days prior to the
 beginning of the study

 13. Intake of oral corticosteroids within 14 days prior to the beginning of the study

 14. Use of depot corticosteroids or chronic systemic corticosteroids within 21 days before
 beginning of the study

 15. Pregnancy or breast-feeding