E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with cutaneous manifestations of CTCL who have progressive, persistent, or recurrent cutaneous T-cell lymphoma on or following two systemic therapies. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 6.1 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10025321 |
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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 |
To provide drug on a compassionate use basis for the treatment of cutaneous manifestations in patients with cutaneous T-cell lymphoma who have progressive, persistent, or recurrent disease on or following two systemic therapies and who would not otherwise have access to vorinostat. |
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E.2.2 | Secondary objectives of the trial |
1) To assess the safety and tolerability of treatment with vorinostat in patients with CTCL in a compassionate use setting. Hypothesis: Oral vorinostat at a dose of 400 mg once daily is well-tolerated in patients with CTCL. 2) To assess the efficacy of vorinostat in patients with CTCL in a compassionate use setting. Hypothesis: Oral vorinostat at a dose of 400 mg once daily is effective in a substantial proportion of patients with CTCL. |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. Patient has cutaneous manifestations of CTCL, with progressive, persistent, or recurrent disease on or following two systemic therapies. 2. Patients age is ≥18 years. 3. Female patients of childbearing potential must have a negative serum pregnancy test (βhCG) within 72 hours of receiving the first dose of vorinostat. 4. Female patient is either post menopausal, free from menses for >2 years, surgically sterilized or willing to use 2 adequate barrier methods of contraception to prevent pregnancy or agrees to abstain from heterosexual activity throughout the study, starting with visit 1. 5. Male, patient agrees to use an adequate method of contraception for the duration of the study. 6. Patient has ability to understand and willingness to sign the informed consent form. |
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E.4 | Principal exclusion criteria |
1. Patient is currently under treatment with any potential HDAC inhibitor (e.g., valproic acid). 2. Patient is currently receiving other systemic treatment for CTCL other than corticosteroids at a dose of no more than the equivalent of 20 mg of prednisone daily. 3. Patient is pregnant or lactating. 4. Patient has known allergy to any component of the study drug. 5. Patient is eligible for any other study of vorinostat in CTCL. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint is the response rate to oral vorinostat in patients with CTCL who have progressive, persistent or recurrent disease on or following two systemic therapies as measured by the physician assessment. |
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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 | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |