E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Myasthenia Gravis in patients who have generalized Muscle Weakness |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
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.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028423 |
E.1.2 | Term | Myasthenia gravis-like syndrome |
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 | 20.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10071942 |
E.1.2 | Term | Myasthenia gravis and related conditions |
E.1.2 | System Organ Class | 100000004859 |
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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 |
Efficacy of ARGX-113 as assessed by the percentage of “Myasthenia Gravis Activities of Daily Living (MG-ADL) responders” in the acetylcholine receptor (AChR)- antibody (Ab) seropositive population. |
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E.2.2 | Secondary objectives of the trial |
1. Efficacy of ARGX-113 as assessed by the percentage of “QMG responders” in the AChR-Ab seropositive population. 2. Efficacy of ARGX-113 as assessed by the percentage of “MG-ADL responders” in the overall population (AChR-Ab seropositive and AChR-Ab seronegative patients). 3. Efficacy of ARGX-113 as assessed by the percentage of time that patients show a “clinically meaningful improvement” in total MG-ADL score during the trial in the AChR-Ab seropositive population. 4. Duration of response 5. Onset of efficacy of ARGX-113 as assessed by the percentage of “early MG-ADL responders” in the AChR-Ab seropositive population. 6. Safety and tolerability of ARGX-113 in the overall population and in subgroups. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures. 2. Male or female patients aged ≥ 18 years. 3. Diagnosis of MG with generalized muscle weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa and IVb.
Other more specific inclusion criteria are further defined in the protocol. |
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E.4 | Principal exclusion criteria |
1.Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. 2. Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing. 3. MGFA Class I and V patients 4. Patients with worsening muscle weakness secondary to concurrent infections or medications. 5.Patients with known seropositivity or who test positive for an active viral infection at Screening with: o Hepatitis B Virus (HBV) (except patients who are seropositive because of HBV vaccination) o Hepatitis C Virus (HCV) o Human Immunodeficiency Virus (HIV)
Other more specific exclusion criteria are further defined in the protocol. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy of ARGX-113 as assessed by the percentage of “Myasthenia Gravis Activities of Daily Living (MG-ADL) responders” (acetylcholine receptor (AChR)- antibody (Ab) seropositive population) |
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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) |
1. Efficacy of ARGX-113 as assessed by the percentage of “QMG responders” in the AChR-Ab seropositive population. 2. Efficacy of ARGX-113 as assessed by the percentage of “MG-ADL responders” in the overall population (AChR-Ab seropositive and AChR-Ab seronegative patients). 3. Efficacy of ARGX-113 as assessed by the percentage of time that patients show a “clinically meaningful improvement” in total MG-ADL score during the trial in the AChR-Ab seropositive population. 4. Duration of response 5. Onset of efficacy of ARGX-113 as assessed by the percentage of “early MG-ADL responders” in the AChR-Ab seropositive population.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Sec EP 1: Week 8 Sec EP 2: Week 8 Sec EP 3: Duration of the trial Sec EP 4: Duration of the trial Sec EP 5: Week 8 |
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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 | Yes |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
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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 | 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 | Yes |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 22 |
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 |
Belgium |
Canada |
Czech Republic |
Denmark |
France |
Georgia |
Germany |
Hungary |
Italy |
Japan |
Netherlands |
Poland |
Russian Federation |
Serbia |
Sweden |
United Kingdom |
United States |
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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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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 | 3 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 10 |
E.8.9.2 | In all countries concerned by the trial days | 0 |