E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028417 |
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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 |
is to assess the efficacy of mycophenolate mofetil therapy compared to placebo in myasthenia gravis patients receiving prednisone |
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E.2.2 | Secondary objectives of the trial |
is to assess the safety and tolerability of mycophenolate mofetil therapy compared to placebo in mysthenia gravis patients receiving prednisone |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Subject of either sex, 18 to 80 years of age (inclusive) diagnosis of MG meeting all of the following criteria: history of myasthenic weakness involving more than ocular or peri-ocular muscles history of positive edrophonium chloride test OR abnormal neuromuscular transmission demonstrated by electrodiagnostic testing history of elevated AChR antibodies disease severity history: Myasthenia Gravis Foundation of America (MGFA) classification II, III, or IVa duration of MG symptoms (including ocular symptoms) ≤ 10 years requirement of immunosuppressive therapy in the judgment of the investigator prednisone dose of ≥20mg/day (or equivalent alternate day dose) for at least 4 weeks prior to randomization if subject is taking a cholinesterase inhibitor (i.e. pyridostigmine bromide), a stable regimen is required for at least 2 weeks prior to randomization |
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E.4 | Principal exclusion criteria |
pregnancy, breastfeeding, or lactation receiving regularly scheduled plasma exchange (PE) or intravenous immunoglobulin (IVIG) treatment or receiving PE or IVIG treatment within 2 weeks prior to randomization receiving MMF or other immunosuppressant therapy (except corticosteroids) within 8 weeks prior to randomization any prior clinically significant use of MMF or other immunosuppressant therapy (except corticosteroids) severe weakness of oropharyngeal and/or respiratory muscles (MGFA Class IVb or V; compromised airway protection; MG crisis or impending crisis) thymoma thymectomy within 6 months prior to randomization presence or history of: severe active gastrointestinal disease; persistent severe diarrhea; gastrointestinal hemorrhage active unhealed peptic ulcer within 3 months prior to randomization immune deficiency malignancy lymphoproliferative disease or previous total lymphoid irradiation chronic or frequent drug-resistant bacterial infections or presence of active infection requiring antimicrobial treatment frequent and/or serious viral infection systemic or invasive fungal disease within 2 years prior to randomization significant kidney or liver dysfunction pulmonary insufficiency requiring supplemental oxygen bone marrow insufficiency |
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E.5 End points |
E.5.1 | Primary end point(s) |
Treatment groups will be compared to measure the proportion of subjects reaching responder status. A subject will be considered a responder if he or she meets all the following criteria: Minimal Manifestations or Pharmacologic Remission (MGFA Postintervention Status definitions modified) from Week 32 until study termination at Week 36 AND Prednisone dose of not more than 7.5 mg/day from Week 32 until study termination at Week 36 AND Cholinesterase inhibitor dose of ≤120 mg/day from Week 33* until study termination at Week 36 *subjects have one week to reduce cholinesterase inhibitor dose after reaching 7.5mg/day prednisone
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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 | Information not present in EudraCT |
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) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
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 | 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 | Yes |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | |
E.8.9.2 | In all countries concerned by the trial years | 3 |