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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-002133-37 | EudraCT Number |
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This is a Long-Term, Single-Arm, Open-Label, Multicenter Phase 3 follow-on trial of the ARGX-113-1704 study to evaluate the safety and tolerability of ARGX-113 in patients with gMG. Patients who have completed at least 1 cycle of treatment and at least 1 year of trial ARGX-113-1705 and have started Part B are eligible to enroll in the open-label trial ARGX-113-2002 to receive efgartigimod by SC administration.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ARGX-113 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ARGX-113 | Biological | Intravenous administration of ARGX-113 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious AEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in AChR-Positive Participants | An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (electrocardiogram [ECG], radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. A serious AE (SAE) was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant. | TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population | Overall population included both AChR-Ab seropositive and AChR-Ab seronegative participants. An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (ECG, radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. An SAE was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant. |
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Inclusion Criteria:
Other more specific inclusion criteria are further defined in the protocol.
Exclusion Criteria:
Other, more specific exclusion criteria are further defined in the protocol.
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| Name | Affiliation | Role |
|---|---|---|
| Sabine Coppieters, MD | argenx | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigator Site 42 | Carlsbad | California | 92011 | United States | ||
| Investigator Site 8 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38318236 | Derived | Howard JF Jr, Bril V, Vu T, Karam C, Peric S, De Bleecker JL, Murai H, Meisel A, Beydoun SR, Pasnoor M, Guglietta A, Van Hoorick B, Steeland S, T'joen C, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT+ Study Group. Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis. Front Neurol. 2024 Jan 17;14:1284444. doi: 10.3389/fneur.2023.1284444. eCollection 2023. | |
| 34146511 |
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Participants from ARGX-113-1704 who either completed that study or required retreatment that could not be completed during a treatment cycle in that study were included in this study to receive efgartigimod. A total of 151 participants rolled over to this study and 145 of them received at least 1 dose of study treatment. Participants starting part B of this study were eligible to roll over in ARGX-113-2002 study.
This Phase III, open-label study was a follow-on study of ARGX-113-1704 (NCT03669588) and was conducted in participants with myasthenia gravis having generalized muscle weakness at 51 investigational sites in 14 countries between 01 Mar 2019 and 30 Jun 2022. This study was conducted in 2 sequential parts: Part A (1 year) and Part B (<=2 years).
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| ID | Title | Description |
|---|---|---|
| FG000 | Efgartigimod | Participants were administered efgartigimod intravenous (IV) 10 milligrams/kilograms (mg/kg) over 1 hour every 7 days for 4 administrations per treatment period (TP) for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an intertreatment period (ITP) of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 19, 2021 | Jun 21, 2023 |
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| TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years |
| Los Angeles |
| California |
| 90033 |
| United States |
| Investigator Site 33 | Orange | California | 92868 | United States |
| Investigator Site 21 | Palo Alto | California | 94304 | United States |
| Investigator Site 41 | Jacksonville | Florida | 32209 | United States |
| Investigator Site 4 | Tampa | Florida | 33612 | United States |
| Investigator Site 14 | Kansas City | Kansas | 66160 | United States |
| Investigator Site 32 | Detroit | Michigan | 48201 | United States |
| Investigator Site 2 | Chapel Hill | North Carolina | 27599 | United States |
| Investigator Site 18 | Cleveland | Ohio | 44195 | United States |
| Investigator Site 12 | Portland | Oregon | 97239 | United States |
| Investigator Site 1 | Cordova | Tennessee | 38018 | United States |
| Investigator Site 3 | San Antonio | Texas | 78229 | United States |
| Investigator Site 37 | Charlottesville | Virginia | 22908 | United States |
| Investigator Site 11 | Edegem | 2650 | Belgium |
| Investigator Site 7 | Ghent | 9000 | Belgium |
| Investigator Site 20 | Toronto | Ontario | M5G 2C4 | Canada |
| Investigator Site 25 | Montreal | Quebec | H3A 2B4 | Canada |
| Investigator Site 16 | Brno | 625 00 | Czechia |
| Investigator Site 19 | Ostrava-Poruba | 70852 | Czechia |
| Investigator Site 30 | Prague | 12800 | Czechia |
| Investigator Site 49 | Aarhus | Denmark |
| Investigator Site 17 | Copenhagen | DK-2100 | Denmark |
| Investigator Site 50 | Bordeaux | France |
| Investigator Site 51 | Marseille | France |
| Investigator Site 45 | Tbilisi | 0112 | Georgia |
| Investigator Site 31 | Tbilisi | 0114 | Georgia |
| Investigator Site 46 | Tbilisi | 0114 | Georgia |
| Investigator Site 28 | Berlin | 10117 | Germany |
| Investigator Site 35 | Budapest | 1204 | Hungary |
| Investigator Site 52 | Szeged | Hungary |
| Investigator Site 10 | Milan | MI | 20133 | Italy |
| Investigator Site 5 | Naples | 80131 | Italy |
| Investigator Site 38 | Roma | 00189 | Italy |
| Investigator Site 24 | Sapporo | Hokkaido | 060-8543 | Japan |
| Investigator Site 13 | Hanamaki-shi | Iwate | 025-0075 | Japan |
| Investigator Site 27 | Sendai | Miyagi | 983-8520 | Japan |
| Investigator Site 23 | Ōsaka-sayama | Osaka | 589-8511 | Japan |
| Investigator Site 22 | Suita | Osaka | 565-0871 | Japan |
| Investigator Site 40 | Meguro City | Tokyo | 153-8515 | Japan |
| Investigator Site 43 | Shinjuku-Ku | Tokyo | 160-0023 | Japan |
| Investigator Site 47 | Chiba | Japan |
| Investigator Site 44 | Hiroshima | 730-8518 | Japan |
| Investigator Site 48 | Minato | Japan |
| Investigator Site 36 | Leiden | 2333 ZA | Netherlands |
| Investigator Site 9 | Gdansk | 80-952 | Poland |
| Investigator Site 29 | Katowice | 40-123 | Poland |
| Investigator Site 6 | Krakow | 31-505 | Poland |
| Investigator Site 15 | Warsaw | 02-097 | Poland |
| Investigator Site 34 | Novosibirsk | 630087 | Russia |
| Investigator Site 39 | Samara | 443095 | Russia |
| Investigator Site 26 | Belgrade | 11000 | Serbia |
| Derived |
| Howard JF Jr, Bril V, Vu T, Karam C, Peric S, Margania T, Murai H, Bilinska M, Shakarishvili R, Smilowski M, Guglietta A, Ulrichts P, Vangeneugden T, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT Investigator Study Group. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9. |
| COMPLETED |
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| NOT COMPLETED |
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The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received >=1 dose or part of a dose of efgartigimod in this study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Efgartigimod | Participants were administered efgartigimod IV 10 mg/kg over 1 hour every 7 days for 4 administrations per TP for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an ITP of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants | No |
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| Race/Ethnicity, Customized | Count of Participants | Participants | No |
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| Race/Ethnicity, Customized | Count of Participants | Participants | No |
| ||||||||||||||||||||||
| Anti-acetylcholine receptor antibodies (AChR-Ab) status | Count of Participants | Participants | No |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious AEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in AChR-Positive Participants | An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (electrocardiogram [ECG], radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. A serious AE (SAE) was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant. | The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received >=1 dose or part of a dose of efgartigimod in this study. Only those participants with AChR-positive status are included in this analysis. | Posted | Count of Participants | Participants | No | TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years |
|
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population | Overall population included both AChR-Ab seropositive and AChR-Ab seronegative participants. An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (ECG, radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. An SAE was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant. | The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received >=1 dose or part of a dose of efgartigimod in this study. | Posted | Count of Participants | Participants | No | TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years |
|
TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received >=1 dose or part of a dose of efgartigimod in this study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Efgartigimod | Participants were administered efgartigimod IV 10 mg/kg over 1 hour every 7 days for 4 administrations per TP for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an ITP of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response. | 5 | 145 | 36 | 145 | 83 | 145 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
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| Cardiac failure congestive | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Defect conduction intraventricular | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
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| Retinal detachment | Eye disorders | MedDRA 24.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Death | General disorders | MedDRA 24.1 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| COVID-19 pneumonia | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Dysentery | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Pneumonia escherichia | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Pseudomonal sepsis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Septic shock | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
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| SARS-CoV-2 test positive | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Type 1 diabetes mellitus | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
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| Adenocarcinoma of colon | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
| |
| Hepatocellular carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
| |
| Lung neoplasm malignant | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
| |
| Pancreatic carcinoma metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
| |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
| |
| Squamous cell carcinoma of the vulva | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
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| Uterine leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
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| Cerebral venous sinus thrombosis | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Myasthenia gravis | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Myasthenia gravis crisis | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Stupor | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Acute kidney injury | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
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| Bladder neck obstruction | Renal and urinary disorders | MedDRA 24.1 | Systematic Assessment |
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| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Pneumonitis aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Shoulder arthroplasty | Surgical and medical procedures | MedDRA 24.1 | Systematic Assessment |
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| Spinal decompression | Surgical and medical procedures | MedDRA 24.1 | Systematic Assessment |
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| Spinal operation | Surgical and medical procedures | MedDRA 24.1 | Systematic Assessment |
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| Shock | Vascular disorders | MedDRA 24.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 24.1 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA 24.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Regulatory Manager | argenx BVBA | +32 93103400 | regulatory@argenx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 20, 2022 | Jun 21, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| C000718373 | efgartigimod alfa |
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| Black or African American |
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| White |
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| Multiple |
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| Not Hispanic or Latino |
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| Title | Measurements |
|---|---|
|
| Fatal TEAE |
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|
|