E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Myasthenia Gravis |
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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.1.3 | 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 long-term safety and tolerability of efgartigimod PH20 SC in participants with Generalized Myasthenia Gravis (gMG) |
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E.2.2 | Secondary objectives of the trial |
• To evaluate the impact of efgartigimod PH20 SC on disease severity • To evaluate the effect of efgartigimod PH20 SC on Pharmacodynamic (PD) •To evaluate the PK of efgartigimod PH20 SC • To evaluate the immunogenicity of efgartigimod PH20 SC
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Participants are eligible to be included in the study only if all of the following criteria apply: 1. Must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol 2. Previously participated in antecedent studies ARGX-113-2001 or ARGX-113-1705 and are eligible for rollover 3. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and: a. Male participants: − Male participants are not allowed to donate sperm from signing the ICF until the last end of the study. b. Female participants: − Women of Child bearing potential (WOCBP) must have a negative urine pregnancy test at baseline before IMP can be administered.
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E.4 | Principal exclusion criteria |
Participants are excluded from the study if any of the following criteria apply: 1. The participant was discontinued early from studies ARGX-113-2001 or ARGX-113-1705, unless the reason for discontinuation from study ARGX-113-1705 was to rollover into study ARGX-113-2002 a. Participants who, in the investigator’s judgment, are not benefiting from efgartigimod IV in study ARGX-113-1705 Part B are not eligible for rollover into ARGX-113-2002. 2. Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of IMP 3. Have any of the following medical conditions: a. Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at screening b. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk c. History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence for ≥3 years before the first administration of IMP. Participants with the following cancers can be included at any time: − adequately treated basal cell or squamous cell skin cancer − carcinoma in situ of the cervix − carcinoma in situ of the breast − incidental histological findings of prostate cancer (TNM classification of malignant tumors stage T1a or T1b) d. Clinical evidence of other significant serious diseases, or the participant has had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the participant at undue risk
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E.5 End points |
E.5.1 | Primary end point(s) |
• Incidence of Serious Adverse Events (SAEs) , and AEs of special interest (AESIs) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The primary end point is timeframe 'up to 2 years'. |
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E.5.2 | Secondary end point(s) |
• Percentage reduction in levels of total immunoglobulin G (IgG) from baseline and cycle baseline over time by cycle • Percentage reduction of anti-acetylcholine receptor antibodies (AChRAb) from baseline and cycle baseline over time by cycle in AChR-Ab seropositive participants • Efgartigimod serum concentrations • Incidence and prevalence of anti-drug antibodies (ADAs) to efgartigimod over time |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
All secondary end points are timeframe 'up to 2 years'. |
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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 | Yes |
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 |
Tolerability and Immunogenicity |
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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 | 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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 38 |
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 |
Bosnia and Herzegovina |
Canada |
Georgia |
Japan |
Russian Federation |
Serbia |
United States |
Belgium |
Czechia |
Germany |
Hungary |
Italy |
Poland |
United Kingdom |
Netherlands |
Spain |
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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 | 2 |
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 | 2 |