E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute and chronic Graft versus Host Disease (GVHD) |
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E.1.1.1 | Medical condition in easily understood language |
Acute and chronic Graft versus Host Disease (GVHD) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10018651 |
E.1.2 | Term | Graft versus host disease |
E.1.2 | System Organ Class | 10021428 - Immune system disorders |
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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 evaluate the effect of treatment with extracorporeal photopheresis in acute and chronic GVHD. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
The study is divided into two parts with one part regarding acute GVHD and one reagarding chronic GVHD. |
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E.3 | Principal inclusion criteria |
Patients with acute or chronic GVHD, that do not respond sufficiently to glucocorticoids. |
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E.4 | Principal exclusion criteria |
Patients with contraindications for extracorporeal photopheresis. Patients with relapse of primary hematologic disease or activity in this. |
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E.5 End points |
E.5.1 | Primary end point(s) |
For acute GVHD: -Achiement of Partial Remission (PR), Very Good Partial Remission (VGPR) or Complete Remission (CR) - Time to PR,VGPR or CR - Change in dose of immunosuppresive medication and time to half the dose of steroid. - Freedom form treatment failure at 6 months
For chronic GVHD: - Response at 3,6,9 and 12 months - Time to partiel or complete remission - Freedom from treatment failure at 6 months - REduction in steroid dose - Duration ECP treatment - Re-occurence of chronic GVHD after stopping ECP
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Acute GVHD: Evaluation weekly for 8 weeks and then in week 10, 6, 20 and 24 after initiation of treatment. Chronic GVHD: Evaluation every 3 months for the duration of treatment. |
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E.5.2 | Secondary end point(s) |
Acute GVHD: - Duration of response - Duration of immunosuppressive treatment - Infections demanding antibiotics - Incidence of cGVHD (in the acute GVHD part) - Non-relapse mortality - Overall survival - Effect on chimerism for patients with non-myeloablative conditioning - amount and distribution of T-, B- and NK-cells (acute GVHD)
Chronic GVHD: - Patient self assesment score - Occurence of infections - NIH global assesment score (2005) - Occurrence of relapse of primary hematologic disease - Chronic GVHD- free survival - Overall survival - Transplant related mortality - |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Most secondary endpoints will be assesed at the above mentioned timepoints. Non.relapse mortality ans overall survival will be assesed at the end of trial. T-,B- and NK cells + chimerism will be measured at the begining of treatment and at week 4 and 8. |
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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 | Yes |
E.6.4 | Safety | No |
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 | No |
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
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 | No |
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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The trial is expected to end in 2018. LVLS is expected in the first part of 2018 and then data will be analyzed. LVLS will be before 1/6 2018, but an exact date cannot be given since it is unknown when the last patient will develop GVHD. Patients cannot be enrolled into the study later than 1/1 2018. |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |