E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
The cutaneous T-cell lymphoma (also known as Sézary syndrome) is a common disorder of proliferated cells with characteristics of maligne t-lymphocytes immigrating from the cutaneous blood vessels to dermis and epidermis. Efalizumab inhibits extravasation of t-lymphocytes to dermis and inhibits epidermal stimulation by their interaction with the Langerhans-cells. |
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MedDRA Classification |
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 |
- Response rate evaluated by Severity-Weighted Assessment Tool (SWAT) |
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E.2.2 | Secondary objectives of the trial |
- Response rate evaluated by Physicians Global Assessment of Clinical Condition (PGA) - Documentation of symptoms and patient recruitment - Evalution and foto documentation of response of index lesions - Duration of response - Duration until response - Progression free survival rate - Pharmacocinetics of Efalizumab - Analysis of biological marker (histology, t-cell receptor-beta PCR, PCR, flow cytometry and evaluation of cytokines IL-12 and IFN-gamma) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- written informed consent - age min. 18 years - patients with Sézary syndrome evaluated by skin biopsy - patients with at minimum one systemic therapy. systemic therapies are PUVA, photophoresis. chemotherapy e.g. methotrexat, and IFN-alpha - patients complying the following laboratory parameters: *leukocytes >= 1,5x10^9/L *hemoglobin >= 9 g/dL *thrombocytes >= 100x10^9/L *potassium >= lower limit of normal *total calcium (corrected for serum albumin) or iionised calcium >= lower limit of normal *magnesium >= lower limit of normal *AST/SGOT and ALT/SGPT <= 2,5 times upper limit of normal *serum bilirubin <= 1,5 times lower limit of normal *serum creatinin <= 1,5 times lower limit of normal or 24h clearance >= 50mL/min - World Health Organization (WHO) performance status < 2 - no participation in another clinical trial within 1 month before study enrolment and during the study |
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E.4 | Principal exclusion criteria |
- notification of sepsis, current tuberculosis or another serious infection disease - general appearance including CNS - cardiac or renal insufficiency - concurrent chemotherapy or radiotherapy including whole body irradiation and phototherapy (PUVA, UVA, UVB, UVB 311nm) - treatment with chemotherapy or investigational drug or major surgery within 4 weeks before study enrolment or patients - pregnant or nursing patients - female patients of childbearing potential or male patients and their partners without highly effective contraception (PEARL-Index < 1%, e.g., hormonal contraception, intrauterine device, condom with spermicide, etc.) during and for 6 months after discontinuation of study treatment - diagnosis of another cancer within 5 years before study enrolment except of a curatively treated Melanoma-in-situ or basal cell carcinoma or squamous epithelium carcinoma of the skin - patients with HIV - patients who are detained officially or legally to an official institute - lack of compliance - all contraindications against investigational product esp. hypersensitivity against Efalizumab or pharmaceutical additives as formation - lack of willingness to save and provide pseudonymised data within the clinical trial - specific forms of psoriasis |
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E.5 End points |
E.5.1 | Primary end point(s) | |
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 | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
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 | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
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 | No |
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 | 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 | 1 |
E.8.9.1 | In the Member State concerned days | |