E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Mastocytosis is characterized by an excessive increase in the number of mast cells in different tissues. Symptoms result either from organ infiltration or frequently from the release of proinflammatory mast cell mediators. Whereas in cutaneous mastocytosis the increase in mast cell numbers is limited to the skin, systemic mastocytosis is characterized by multifocal lesions in the bone marrow and/or in various extracutaneous organs. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10052577 |
E.1.2 | Term | Pure dermal mastocytosis |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10056452 |
E.1.2 | Term | Indolent systemic mastocytosis |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Reduction of wheal and flare type skin reaction after standardised provocation testing assessed by volumetric and thermographic measurements |
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E.2.2 | Secondary objectives of the trial |
Improvement of additional related symptoms (e.g. pruritus) and subjective affliction as measured by physician and patient global assessments, DLQI, Itchy-QoL and VAS |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Chronic stable symptomatic maculopapulous cutaneous mastocytosis or indolent systemic mastocytosis with skin involvement and a positive Darier's Sign - Age between 18 and 70 years - 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. - Voluntarily signed written informde consent |
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E.4 | Principal exclusion criteria |
- The presence of permanent severe diseases, especially those affecting the immune system, except for mastocytosis - History or presence of epilepsy, significant neurological disorders, cerebrovascular attacks or ischemia - History or presence of myocardial infarction or cardiac arrhythmia which requires drug therapy, hyper-/hypokalemia, bradycardia < 50bpm, QTc interval > 440ms - Evidence of severe renal dysfunction (creatinine > 1,5 x upper reference value) - Evidence of significant hepatic disease (liver enzymes > 2 x upper reference value) - History of adverse reactions to RUP, or other ingredients of the IMP - Presence of active cancer which requires chemotherapy or radiation therapy - Aggressive systemic mastocytosis - History or presence of alcohol abuse or drug addiction - Participation in any clinical trial within 4 weeks prior to enrolment - Commitment to an institution in terms of 40 Abs. 1 Nr. 4 AMG - Intake of antihistamines or leukotriene antagonists within 7 days prior to the beginning of the study - Intake of oral corticosteroids within 14 days prior to the beginning of the study - Use of depot corticosteroids or chronic systemic corticosteroids within 21 days before beginning of the study - Pregnancy or breast-feeding |
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E.5 End points |
E.5.1 | Primary end point(s) |
The main goal of the analysis is to test whether treatment with 20 mg RUP is more effective in protecting mastocytosis patients from induced symptoms as compared to placebo. The primary efficacy parameter of the study is the assessment of wheal development by planimetric analyses of digital photographic imaging (time lapse) before and after treatment with study medication. |
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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 | No |
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 | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
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.8.5 | 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 | Yes |
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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Last patient's last visit |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |