E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Myasthenia Gravis (gMG) |
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E.1.1.1 | Medical condition in easily understood language |
Generalized Myasthenia Gravis (gMG) |
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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 | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028417 |
E.1.2 | Term | Myasthenia gravis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 evaluate the efficacy of batoclimab 680 mg SC QW or 340 mg SC QW compared to PBO as Induction Therapy in AChRAb+ participants as assessed by change in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the efficacy of batoclimab 680 mg SC QW or 340 mg SC QW compared to PBO as Induction Therapy in AChRAb+ participants as assessed by change in Quantitative Myasthenia Gravis (QMG) score.
To evaluate the efficacy of batoclimab 340 mg SC QW or 340 mg SC once every 2 weeks (Q2W) compared to PBO as Maintenance Therapy in AChRAb+ participants who responded to Induction Therapy with batoclimab as assessed by changes in the MG-ADL score.
To evaluate the efficacy of batoclimab 680 mg SC QW or 340 mg SC QW compared to PBO as Induction Therapy in AChRAb+ participants for achieving improvement (decrease in score) in QMG score.
Please refer to protocol section 2.4 for more secondary objectives. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Are ≥ 18 years of age at the Screening Visit. 2. Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification Class II, III, or IVa at the Screening Visit. 3. Have a QMG score ≥ 11 at the Screening and Baseline Visits. 4. Have a MG-ADL score of ≥ 5 at the Screening and Baseline Visits. 5. Additional inclusion criteria are defined in the protocol.
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E.4 | Principal exclusion criteria |
1. Have experienced myasthenic crisis within 3 months of the Screening Visit. 2. Have had a thymectomy performed < 6 months prior to the Screening Visit or have a planned thymectomy during the study period. 3. Have any active or untreated malignant thymoma. 4. Have received any agent or therapy (exclusive of those identified within inclusion criteria) with immunosuppressive properties (e.g., stem cell therapy, chemotherapies, etc.) within the past year. 5. Have used anti-FcRN treatment within 3 months prior to the Screening Visit or have a documented history of non-response to prior anti-FcRN treatment. 6. Additional exclusion criteria are defined in the protocol. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from Period 1 baseline in MG-ADL score to Week 12 for AChRAb+ participants (Timeframe: Period 1 baseline to Week 12)
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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) |
Change from Period 1 baseline in QMG score to Week 12 for AChRAb+ participants (Timeframe: Period 1 baseline to Week 12)
Change from Period 2 baseline in MG-ADL score to Week 24 for AChRAb+ randomized withdrawal participants (Timeframe: Week 12 to Week 24)
Proportion of AChRAb+ participants with ≥ 3-point improvement (decrease in score) in QMG from Period 1 baseline to Week 12 (Timeframe: Period 1 baseline to Week 12)
Proportion of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12 (Timeframe: Period 1 baseline to Week 12)
Change from Period 1 baseline in MG-ADL score to Week 12 for AChRAb- participants (Timeframe: Period 1 baseline to Week 12) |
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Yes |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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 | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Long-term Extension (LTE) |
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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.8.2.4 | Number of treatment arms in the trial | 9 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 23 |
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 | No |
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 |
Argentina |
Australia |
Brazil |
Canada |
Japan |
Korea, Republic of |
Mexico |
United States |
France |
Poland |
Romania |
Spain |
Czechia |
Germany |
Italy |
Georgia |
Hungary |
United Kingdom |
Serbia |
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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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The end of study is defined as the date of the last visit of the last participant in the study or last scheduled procedure shown in Section 1.3 of the protocol for the last participant in the study. |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 10 |
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 | 10 |