E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
MS patients who suffer from severe Optic Neuritis without satisfying improvement after treatment with steroids (3-5 days with 1 g daily at least 7 days prior to randomization) and with persisting visual acuity < 0.7, duration of symptoms should be ≤ 4 weeks |
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E.1.1.1 | Medical condition in easily understood language |
MS patients who suffer from severe Optic Neuritis without satisfying improvement after treatment with steroids and with persisting visual acuity < 0.7, duration of symptoms should be ≤ 4 weeks |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
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 | 10063400 |
E.1.2 | Term | Secondary progressive multiple sclerosis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10070425 |
E.1.2 | Term | Multiple sclerosis exacerbation |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 | 10063399 |
E.1.2 | Term | Relapsing-remitting multiple sclerosis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 | 10048393 |
E.1.2 | Term | Multiple sclerosis relapse |
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 |
- to gain further evidence for the efficacy of plasma exchange in steroid-unresponsive optic neuritis - to study new outcome tools for neuronal regeneration in the ON model via OCT
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E.2.2 | Secondary objectives of the trial |
- to study MRI parameters in patients with steroid-unresponsive optic neuritis treated with plasma exchange (contrast-enhancement, T2-signal, edema and atrophy will be assessed as well as DTI at baseline, week 16 and 52) - to further study biomarkers for ON heterogeneity and neurodegeneration
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
o Optic neuritis with visual acuity < 0.7 after steroid treatment (3-5x1g) o Duration of symptoms from onset < 4 weeks o Age: 18-60 years o EDSS: 1.0 – 6.5 o CIS, RR-MS or SP-MS
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E.4 | Principal exclusion criteria |
o absence of evidence of inflammatory activity which is defined as a lack of inflammatory CSF signs (pleocytosis and/or OCBs) or a present MRI without at least 2 MS-typical lesions o bilateral optic neuritis o current treatment with natalizumab o patients with neuromyelitis optica o Pregnancy o Unwillingness or inability to comply with the requirements of this protocol including the presence of any condition (physical, mental, or social) that is likely to affect the patient returning for follow-up visits on schedule. o Patients with cognitive impairments who are unable to provide written, informed consent prior to any testing under this protocol |
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E.5 End points |
E.5.1 | Primary end point(s) |
Difference of Mean RNFL thickness in both groups at week 16 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Treatment failure at week 2, Opthalmological parameters (Visual acuity, visual evoked potentials, perimetry) at week 1, 2, 4, 16, 52, MRI: Gadolineum-enhancing lesions, T2 signal in the optic nerve, optic diameter postbulbar and prechiasmatic, Diffusion-tesnor imaging of optic nerve
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | 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 | Yes |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 5 |
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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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 | |