E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Multifocal motor neuropathy |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10065579 |
E.1.2 | Term | Multifocal motor neuropathy |
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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 determine the safety and tolerability of eculizumab in patients with Multifocal Motor Neuropathy (MMN) receiving cycles of intravenous human immunoglobulin (IVIg). |
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E.2.2 | Secondary objectives of the trial |
To explore the effectiveness of eculizumab in terms of reduction in IVIg requirements and improvements in clinical examination, symptom rating and functionality in IVIg- responsive MMN patients. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Fulfilling the diagnostic clinical and electrophysiological entry requirements in Appendix A, as adapted from the European Federation of Neurological Societies (EFNS) and the Peripheral Nerve Society (PNS)*. 2. Able to complete self-evaluation functional rating scale 3. Documented historical responsiveness to IVIg in the opinion of the Investigator 4. Agreement to vaccination against meningococcal infection. (Tetravalent, conjugated vaccination recommended) 5. Is willing and able to give written informed consent.
*Conduction Block (CB) to have been documented at some point in the patient’s history.
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E.4 | Principal exclusion criteria |
1. Below the age of 18 years old. 2. Pregnancy, planned pregnancy or lactation* 3. Inability to comply with study related procedures or appointments. 4. Unresolved Neisseria meningitidis infection. 5. Any condition that in the opinion of the Investigator could increase the patient’s risk by participating in the study or confound the outcome of the study. 6. Hypersensitivity to eculizumab, murine proteins or to any of the excipients 7. Known or suspected hereditary complement deficiencies.
*A serum pregnancy test will be preformed at the first study visit. Trial participants of child-bearing age will be asked to use adequate contraception during the trial and for 5 months after study cessation. Participants will be instructed to inform the PI immediately if they suspect they may be pregnant
The contraindications and special warning regarding meningococcal infection from the Summary of Product Characteristics of eculizumab in its licensed indication, Paroxysmal Nocturnal Haemoglobinuria, are reflected in the inclusion and exclusion criteria of this study.
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E.5 End points |
E.5.1 | Primary end point(s) |
-Frequency of adverse events and serious adverse events -Amount of IVIg treatment required, during Run-in compared to Treatment period. -Change in scores/rating scales/measurements between baseline and during 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 | 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
| 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 | 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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Last visit of last subject |
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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 |