E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients affected with MG showing clinically meaningful improvement after therapeutic plasmapheresis. |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028417 |
E.1.2 | Term | Myasthenia gravis |
|
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 |
The primary objective of this pilot study is to obtain preliminary data on the potential therapeutic activity of PIX in patient affected with MG in a short-term. Tolerability and safety will be assessed for all subjects for the entire duration of the study. |
|
E.2.2 | Secondary objectives of the trial |
Select appropriate dose schedules to be further investigated with long-term confirmative studies. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
caucasian male or female aged 18 to 55 years inclusive diagnosis of MG classes II, III or IV according to MGFA classification (MG Foundation of America) positive acetylcholine receptor antibodies (AChR Ab) LVEF ≥ 55% no conditions known to be contraindications to the use of PIX written informed consent. Positive response to plasmapheresis, i.e reduction of QMGS of at least 3,5 points (at randomization visit) |
|
E.4 | Principal exclusion criteria |
ocular MG (MGFA class I) MG crisis thymectomy in the last 3 months immunosuppressive therapy in the last 3 months other than corticosteroids subjects with history of myocardial infarction subjects with clinically evident cardiac disease (ischemic heart disease or congestive heart failure) subjects with uncontrolled hypertension contraindication to plasmapheresis subjects with active malignancy female subjects who are pregnant or lactating subjects who have received an experimental drug or have participated in a clinical trial within 3 months prior to screening employees of the investigator or study centre with direct involvement in the proposed study or other studies under the direction of that investigator or study centre. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary evaluation parameter will be the QMGS (Quantitative Myasthenia Gravis Score) |
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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 | Yes |
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 | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | Yes |
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 | Yes |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
- same IMP used at different dosage |
|
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |