E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Severe progressive multiple sclerosis in which there is no other documented treatment avalilable or allready tested. It is estimated that the prognosis for these patients are poor without any effective treatment |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028245 |
E.1.2 | Term | Multiple sclerosis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 |
Investigate tolerability and efficacy of intrathecally administered Mabthera in severe progressive MS where there is no available effective treatment |
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E.2.2 | Secondary objectives of the trial |
Investigate the effects on immune parameters and magnetic resonance imaging of the treatment |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
A subject will be eligible for inclusion in this study if all of the following criteria apply:
• Between the age of 18 and 65 years of age • Progressive MS since at least three years • Some kind of documented progression of neurological symptoms during the previ-ous two years. • EDSS 4,0 - 7.0 (inclusive) (basically spared arm functions) • Conventional therapy not indicated, contraindicated or failed • Judged as compliant with the protocol
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E.4 | Principal exclusion criteria |
A subject will not be eligible for inclusion in this study if any of the following criteria ap-plies:
• Eligible for any of the conventional MS therapies • RRMS • Bleeding diathesis or medication contraindicating neurosurgical procedures or lumbar puncture • Cognitive defect making informed consent unreliable • Any medidal condition contraindicating minor surgical procedures, as judged by anaesthesiologist • Severe uncontrolled heart disease • Pregnant or lactating women • Patients having contraindication for or otherwise not compliant with MRI investigations • Documented vulnerability to infections • Simultaneous treatment with other immunosuppressive drugs • Documented allergy or intolerance to Rituximab • Severe psychiatric condition
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is to document safety parameters during the study The secondary endpoints are clinical variables, ie stabilization of the disease, measured as tes for both leg, arm and cognitiv functions another secondary and exploratory endpoint is by analysing biomarkers and lymfocyte subsets in the CSF and blood to document expected biological effects of the treatment.
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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 | Yes |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
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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 | Yes |
E.7.1.3.1 | Other trial type description |
Safety and first exploratory |
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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 | 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) | 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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End of trial when last patient has been followed up clinically and radiologically for one year after last dose. |
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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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |