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This is a phase 2, randomized, double-blinded, placebo-controlled, parallel-group, multicenter trial to evaluate the safety and efficacy of 2 dose regimens of ARGX-117 versus placebo, in participants with MMN previously stabilized with IVIg (intravenous immunoglobulin).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ARGX-117 | Experimental | Intravenous administration of ARGX-117 |
|
| Placebo | Placebo Comparator | Intravenous administration of placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ARGX-117 | Biological | Intravenous administration of ARGX-117 |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With AEs and SAEs | AE : Adverse Events, SAE: Serious Adverse Events | Up to 80 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Time to the First Retreatment With IVIg | The time to first retreatment with intravenous immunoglobulin (IVIg) is defined as the time from the last IVIg administration before randomization until the first IVIg retreatment during the 16-week treatment period | Up to 16 weeks |
| Time-to-relapse |
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Inclusion Criteria:
Exclusion Criteria:
Any coexisting condition which may interfere with the outcome assessments
Clinical signs or symptoms suggestive for neuropathies other than MMN such as motor neuron disease or other inflammatory neuropathies
Severe psychiatric disorder, history of suicide attempt, or current suicidal ideation that in the opinion of the investigator could create undue risk to the participant or could affect adherence with the trial protocol.
Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection during the screening and/or IVIg monitoring period (IVMP).
Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of MMN or put the participant at undue risk (eg, SLE).
History of malignancy unless resolved by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following carcinomas will be eligible:
Clinical evidence of other significant serious diseases, have had a recent major surgery (including a splenectomy at any time), or who have any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk
Prior/concomitant therapy
Positive serum test at screening for an active viral infection with any of the following conditions:
Current or history of (ie, within 12 months of screening) alcohol, drug, or medication abuse
Known hypersensitivity reaction to 1 of the components of the IMP or any of its excipients
Female participants with a positive serum or urine pregnancy test, lactating females, and those who intend to become pregnant during the trial or within 15 months after last dose of the IMP
ALT or AST ≥2 × upper limit of normal and total bilirubin ≥1.5 × upper limit of normal of the central laboratory reference range
An estimated glomerular filtration rate of ≤60 mL/min/1.73m2
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| HonorHealth Research Institute-Neuroscience Research | Scottsdate | Arizona | 85251 | United States | ||
| California Pacific Medical Center-Forbes Norris MDA/ALS Research Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | ARGX-117 Cohort 1 | Participants receiving a single dose of 30 mg/kg followed by 4 once- weekly doses of 10 mg/kg and a maintenance regimen of a single dose of 10 mg/kg every 2 weeks until the end of the DBTP (through intravenous infusions) |
| FG001 | Placebo Cohort 1 |
| 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 | Mar 2, 2023 | May 30, 2025 |
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| Placebo |
| Other |
Intravenous administration of placebo |
|
Time-to-relapse is defined as the time from randomization until a participant met the threshold for clinically meaningful deterioration |
| Up to 16 weeks |
| iAUC of the Change From Baseline in mMRC-10 Sum Score | The Modified Medical Research Council (mMRC)-10 sum score assesses muscle strength of 10 muscles groups, both sides (left and right). A score between 0 (paralysis) and 5 (normal strength) is assigned for each muscle group. A higher value indicates better muscle strength. The total score, ranging from 0 to 100, is based on the sum of both the left and right side of the body. The Incremental Area Under Curve (iAUC) is the area under the curve of the change from baseline in the Modified Medical Research Council (mMRC)-10 score. A positive AUC indicates a favorable outcome while a negative AUC indicates an unfavorable outcome. | Up to 16 weeks |
| Change From Baseline in the Average Score of the 2 Most Important Muscle Groups as Assessed by the mMRC-14 Sum Score | The Modified Medical Research Council (mMRC)-14 assesses muscle strength of 14 muscles groups, both sides (left and right). A score between 0 and 5 (normal strength) is assigned. This endpoint is the change from baseline in the average score of the 2 most important muscle groups affected by the disease. It ranges between 0 and 5. A change of more than 0 represents an improvement in strength, and a change less than 0 represents worsening. | At week 16 |
| Change From Baseline in the mMRC-14 Sum Score | The Modified Medical Research Council (mMRC)-14 scores range from 0 to 140 with a higher score representing better muscle strength. A change of more than 0 represents an improvement in strength, and a change less than 0 represents worsening. | At week 16 |
| Proportion of Participants Showing a Deterioration of at Least 2 Points as Assessed by the mMRC-10 Sum Score | The Modified Medical Research Council (mMRC)-10 scores evaluates motor strength/weakness from 10 predetermined muscle groups. A higher proportion of participants showing a deterioration represents a worsening of the outcome. | Up to 16 weeks |
| iAUC of the Change From Baseline in GS Daily Average | Measurement of grip strength (GS) has been done using the Martin vigorimeter in kPa. The incremental Area Under Curve (iAUC) is the area under the curve of the change from baseline of GS daily average. The 3 daily measurements of GS from the left hand and the 3 daily measurements of GS from the right hand have been recorded and the daily average for the left hand and right hand has been calculated, respectively. | Up to 16 weeks |
| Percent Change From Baseline in GS 3-day Moving Average | Measurement of grip strength (GS) has been done using the Martin vigorimeter in kPa. The 3 daily measurements of GS from the left hand and the 3 daily measurements of GS from the right hand have been recorded and the daily average for the left hand and right hand has been calculated, respectively. A 3-day moving average has been generated based on the average over the last 3 days of the obtained daily averages for each hand. | At week 16 |
| Change From Baseline in the MMN-RODS Centile Score | The Rasch-built Overall Disability Scale for MMN (MMN-RODS) is a disease-specific PRO instrument constructed to capture activity limitations in patients with MMN. Raw sum scores of the 25-item MMN-RODS (range, 0-50) were converted to a centile metric score ranging from 0 to 100. Lower scores indicated a greater degree of disability. | At week 16 |
| Percent Change From Baseline in the Average Time for Upper Extremity (Arm and Hand) Function | The 9-Hole Peg Test (9-HPT) results are based on the time to complete the assessment with a shorter time representing better muscle strength. A change of less than 0 represents an improvement in strength, and a change more than 0 represents worsening. | At week 16 |
| Proportion of Participants by Level of Severity on Each Dimension of the EQ-5D-5L Scale | The EuroQol 5-Dimension 5-Level (EQ-5D-5L) scale includes five dimensions: mobility, self-care, usual activities, pain or discomfort, and anxiety or depression. Each dimension is ranked with a level 1-5 with level 1 being no problems and level 5 representing extreme problems. | At week 16 |
| Change From Baseline in Quality of Life Using EQ-5D-5L Visual Analog Scale | The EQ-5D-5L visual analog scale is from 0-100 with 0 representing the worst health. A change of more than 0 represents an improvement in health, and a change of less than 0 represents worsening. | At week 16 |
| Change From Baseline in the CAP-PRI | The Chronic Acquired Polyneuropathy Patient-reported Index (CAP-PRI) assesses disease-specific quality of life. This instrument includes the assessment of 15 items yielding a total score ranging from 0 to 30. A change of less than 0 represents an improvement in health, and a change more than 0 represents worsening. | At week 16 |
| Proportion of Participants by Level of Improvement Using the PGI-C Scale | Patient Global Impression of Change (PGI-C) scale ranks a patients condition from 1-7 with 1 representing the most improvement and 7 representing the most decline in their condition. | Up to 16 weeks |
| Change From Baseline in the 9-item FSS Average Total Score | 9-item Fatigue Severity Scale (FSS) average score is the sum of the 9 items divided by the number of items. It ranges from 0 to 7 a higher score representing more severe fatigue. A change of less than 0 indicates an improvement. | Up to 16 weeks |
| Percent of Total Hours for Work-related and Household Chore Activities Lost, as Part of the HRPQ | The Health-Related Productivity Questionnaire (HRPQ) provides data related to missed hours at work or educational activities and reduced effectiveness during any attempted work. | Up to 16 weeks |
| Change From Baseline in Effectiveness, Side Effects, Convenience, and Overall Satisfaction Scores as Assessed by the TSQM-14 | Each Treatment Satisfaction Questionnaire for Medication-14 items (TSQM-14) domain score ranges from 0-100 with higher scores representing greater satisfaction with the treatment. A change greater than 0 indicates an improvement in satisfaction. | Up to 16 weeks |
| Maximum Empasiprubart Serum Concentrations (Cmax) | Up to 16 weeks |
| Percent Change From Baseline in Free C2, Total C2, and Functional Complement Activity (CH50) | At week 16 |
| Incidence of Antidrug Antibodies (ADA) Against Empasiprubart | Up to 16 weeks |
| San Francisco |
| California |
| 94109 |
| United States |
| George Washington Medical Faculty Associates | Washington D.C. | District of Columbia | 20037 | United States |
| HonorHealth Research Institute-Neuroscience Research | Maitland | Florida | 32751 | United States |
| University of South Florida Carol and Frank Morsani Center for Advanced Healthcare | Tampa | Florida | 33612 | United States |
| NorthShore University HealthSystem | Glenview | Illinois | 60026 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| University of Minnesota Delware Clinic Research Unit | Minneapolis | Minnesota | 55414 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27514 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Perelman Center for Advanced Medicine-University of Penssylvania | Philadelphia | Pennsylvania | 19104 | United States |
| Austin Neuromuscular Center | Austin | Texas | 78756 | United States |
| West Virginia University Medicine | Morgantown | West Virginia | 26506 | United States |
| Medizinische Universitat Wien Universitatsklienik fur Neurologie | Vienna | 1090 | Austria |
| AZ Sint-Lucas | Ghent | 9000 | Belgium |
| Genge Partners Montreal | Québec | H4A 3TA | Canada |
| Toronto General Hospital | Toronto | M5G 2C4 | Canada |
| CHU de Bordeaux-Hopital Pellegrin | Bordeaux | 33076 | France |
| CHRU de Lille-Hopital Roger Salengro | Lille | 59037 | France |
| CHU de Nice-Hopital Pasteur 2 | Nice | 06001 | France |
| Hopital Pitie Salpetriere | Paris | 75651 | France |
| Katholisches Klinikum Bochum | Bochum | 44791 | Germany |
| Universitatsklinikum Essen | Essen | 45147 | Germany |
| Universitatsmedzin Gottingen, Klinik fur Neurologie | Göttingen | 37075 | Germany |
| Medizinische Hochschule Hannover Klinik Fur Neurologie | Hanover | 30625 | Germany |
| Universitatsklinikum Munster | Münster | 48419 | Germany |
| IRCCS Ospedale San Raffaele | Milan | 20132 | Italy |
| Azienda Ospedaliero Univeritaria Pisana-UOS Neurologia | Pisa | 56126 | Italy |
| Azienda Ospedaliera Sant'Andrea-UOS Malattie Neuromuscolari | Rome | 00189 | Italy |
| Instituto Clinico Humanitas (IRCCS) | Rozzano | 20089 | Italy |
| Amsterdam UMC location AMC, Dep of Neurology | Amsterdam | 1105 AZ | Netherlands |
| University Medical Centre Utrecht | Utrecht | 3584 CX | Netherlands |
| Michalscy I Partnerzy Lekarze Spolka Partnerska | Krakow | 31-426 | Poland |
| Uniwersyteckie centrum kliniczne Warszawskiego | Warsaw | 02-097 | Poland |
| Hospital Universitario Vall d'Herbon | Barcelona | 08035 | Spain |
| Hospital de la Santa Creu I Santa Pau -Sevicio Neurologia | Barcelona | 08041 | Spain |
| Hospital Universitari I Politecnic La Fe de Valencia-Servicio Neurologia | Valencia | 46026 | Spain |
| Queen Elisabeth University Hospital | Glasgow | G51 4TF | United Kingdom |
| University College London Hospital | London | ZC1N 3BG | United Kingdom |
| Oxford University Hospitals NHS Trust-Jonh Radcliffe Hospital | Oxford | OX3 9DU | United Kingdom |
Participants received placebo via intravenous (IV) infusion |
| FG002 | ARGX-117 Cohort 2 | Participants receiving a single dose of 15 mg/kg followed by 4 once- weekly doses of 5 mg/kg and a maintenance regimen of a single dose of 5 mg/kg every 4 weeks until the end of the DBTP (through intravenous infusions) |
| FG003 | Placebo Cohort 2 | Participants received placebo via IV infusion |
| COMPLETED |
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| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | ARGX-117 Cohort 1 | Participants receiving a single dose of 30 mg/kg followed by 4 once- weekly doses of 10 mg/kg and a maintenance regimen of a single dose of 10 mg/kg every 2 weeks until the end of the DBTP (through intravenous infusions) |
| BG001 | Placebo Cohort 1 | Participants received placebo via intravenous (IV) infusion |
| BG002 | ARGX-117 Cohort 2 | Participants receiving a single dose of 15 mg/kg followed by 4 once- weekly doses of 5 mg/kg and a maintenance regimen of a single dose of 5 mg/kg every 4 weeks until the end of the DBTP (through intravenous infusions) |
| BG003 | Placebo Cohort 2 | Participants received placebo via IV infusion |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||
| Age, Continuous | Median | Inter-Quartile Range | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| 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 AEs and SAEs | AE : Adverse Events, SAE: Serious Adverse Events | Posted | Count of Participants | Participants | Up to 80 weeks |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Time to the First Retreatment With IVIg | The time to first retreatment with intravenous immunoglobulin (IVIg) is defined as the time from the last IVIg administration before randomization until the first IVIg retreatment during the 16-week treatment period | Posted | Median | 95% Confidence Interval | days | Up to 16 weeks |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Time-to-relapse | Time-to-relapse is defined as the time from randomization until a participant met the threshold for clinically meaningful deterioration | Posted | Median | 95% Confidence Interval | days | Up to 16 weeks |
| |||||||||||||||||||||||||||||||||||||
| Secondary | iAUC of the Change From Baseline in mMRC-10 Sum Score | The Modified Medical Research Council (mMRC)-10 sum score assesses muscle strength of 10 muscles groups, both sides (left and right). A score between 0 (paralysis) and 5 (normal strength) is assigned for each muscle group. A higher value indicates better muscle strength. The total score, ranging from 0 to 100, is based on the sum of both the left and right side of the body. The Incremental Area Under Curve (iAUC) is the area under the curve of the change from baseline in the Modified Medical Research Council (mMRC)-10 score. A positive AUC indicates a favorable outcome while a negative AUC indicates an unfavorable outcome. | Posted | Median | Inter-Quartile Range | mMRC score*weeks | Up to 16 weeks |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the Average Score of the 2 Most Important Muscle Groups as Assessed by the mMRC-14 Sum Score | The Modified Medical Research Council (mMRC)-14 assesses muscle strength of 14 muscles groups, both sides (left and right). A score between 0 and 5 (normal strength) is assigned. This endpoint is the change from baseline in the average score of the 2 most important muscle groups affected by the disease. It ranges between 0 and 5. A change of more than 0 represents an improvement in strength, and a change less than 0 represents worsening. | Posted | Median | Inter-Quartile Range | score on a scale | At week 16 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the mMRC-14 Sum Score | The Modified Medical Research Council (mMRC)-14 scores range from 0 to 140 with a higher score representing better muscle strength. A change of more than 0 represents an improvement in strength, and a change less than 0 represents worsening. | Posted | Median | Inter-Quartile Range | score on a scale | At week 16 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Proportion of Participants Showing a Deterioration of at Least 2 Points as Assessed by the mMRC-10 Sum Score | The Modified Medical Research Council (mMRC)-10 scores evaluates motor strength/weakness from 10 predetermined muscle groups. A higher proportion of participants showing a deterioration represents a worsening of the outcome. | Posted | Count of Participants | Participants | Up to 16 weeks |
| ||||||||||||||||||||||||||||||||||||||
| Secondary | iAUC of the Change From Baseline in GS Daily Average | Measurement of grip strength (GS) has been done using the Martin vigorimeter in kPa. The incremental Area Under Curve (iAUC) is the area under the curve of the change from baseline of GS daily average. The 3 daily measurements of GS from the left hand and the 3 daily measurements of GS from the right hand have been recorded and the daily average for the left hand and right hand has been calculated, respectively. | Safety analysis set - Only participants with data for these timepoints are included. | Posted | Median | Inter-Quartile Range | kPa*weeks | Up to 16 weeks |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in GS 3-day Moving Average | Measurement of grip strength (GS) has been done using the Martin vigorimeter in kPa. The 3 daily measurements of GS from the left hand and the 3 daily measurements of GS from the right hand have been recorded and the daily average for the left hand and right hand has been calculated, respectively. A 3-day moving average has been generated based on the average over the last 3 days of the obtained daily averages for each hand. | Posted | Median | Inter-Quartile Range | Percent change | At week 16 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the MMN-RODS Centile Score | The Rasch-built Overall Disability Scale for MMN (MMN-RODS) is a disease-specific PRO instrument constructed to capture activity limitations in patients with MMN. Raw sum scores of the 25-item MMN-RODS (range, 0-50) were converted to a centile metric score ranging from 0 to 100. Lower scores indicated a greater degree of disability. | Posted | Median | Inter-Quartile Range | score on a scale | At week 16 |
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| Secondary | Percent Change From Baseline in the Average Time for Upper Extremity (Arm and Hand) Function | The 9-Hole Peg Test (9-HPT) results are based on the time to complete the assessment with a shorter time representing better muscle strength. A change of less than 0 represents an improvement in strength, and a change more than 0 represents worsening. | Posted | Median | Inter-Quartile Range | Percent change | At week 16 |
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| Secondary | Proportion of Participants by Level of Severity on Each Dimension of the EQ-5D-5L Scale | The EuroQol 5-Dimension 5-Level (EQ-5D-5L) scale includes five dimensions: mobility, self-care, usual activities, pain or discomfort, and anxiety or depression. Each dimension is ranked with a level 1-5 with level 1 being no problems and level 5 representing extreme problems. | Posted | Count of Participants | Participants | At week 16 |
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| Secondary | Change From Baseline in Quality of Life Using EQ-5D-5L Visual Analog Scale | The EQ-5D-5L visual analog scale is from 0-100 with 0 representing the worst health. A change of more than 0 represents an improvement in health, and a change of less than 0 represents worsening. | Posted | Median | Inter-Quartile Range | score on a scale | At week 16 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in the CAP-PRI | The Chronic Acquired Polyneuropathy Patient-reported Index (CAP-PRI) assesses disease-specific quality of life. This instrument includes the assessment of 15 items yielding a total score ranging from 0 to 30. A change of less than 0 represents an improvement in health, and a change more than 0 represents worsening. | Posted | Median | Inter-Quartile Range | score on a scale | At week 16 |
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| Secondary | Proportion of Participants by Level of Improvement Using the PGI-C Scale | Patient Global Impression of Change (PGI-C) scale ranks a patients condition from 1-7 with 1 representing the most improvement and 7 representing the most decline in their condition. | Posted | Count of Participants | Participants | Up to 16 weeks |
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| Secondary | Change From Baseline in the 9-item FSS Average Total Score | 9-item Fatigue Severity Scale (FSS) average score is the sum of the 9 items divided by the number of items. It ranges from 0 to 7 a higher score representing more severe fatigue. A change of less than 0 indicates an improvement. | Posted | Median | Inter-Quartile Range | score on a scale | Up to 16 weeks |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Percent of Total Hours for Work-related and Household Chore Activities Lost, as Part of the HRPQ | The Health-Related Productivity Questionnaire (HRPQ) provides data related to missed hours at work or educational activities and reduced effectiveness during any attempted work. | Safety analysis set - only participants with HRPQ data are shown. This is limited to employed/partially employed participants. | Posted | Median | Inter-Quartile Range | Percent | Up to 16 weeks |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Effectiveness, Side Effects, Convenience, and Overall Satisfaction Scores as Assessed by the TSQM-14 | Each Treatment Satisfaction Questionnaire for Medication-14 items (TSQM-14) domain score ranges from 0-100 with higher scores representing greater satisfaction with the treatment. A change greater than 0 indicates an improvement in satisfaction. | Posted | Median | Inter-Quartile Range | score on a scale | Up to 16 weeks |
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| Secondary | Maximum Empasiprubart Serum Concentrations (Cmax) | The number analyzed in some rows differs due to missing participant data. At certain timepoints 0 analyzed participants are reported since no ARGX-117 was administered at those timepoints, as per dosing regimens. | Posted | Mean | Standard Deviation | ug/mL | Up to 16 weeks |
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| Secondary | Percent Change From Baseline in Free C2, Total C2, and Functional Complement Activity (CH50) | PD set - All participants for whom at least 1 postbaseline value for pharmacodynamic parameters was available. Only participants with available data at week 16 are reported. Due to the small sample size (9 participants) in each placebo group, it was prespecified to pool the 2 placebo cohorts for the PD assessments. | Posted | Median | Inter-Quartile Range | Percent change | At week 16 |
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| Secondary | Incidence of Antidrug Antibodies (ADA) Against Empasiprubart | Only ADA-evaluable participants were analyzed for this outcome measure. ADA-evaluable participants were classified as treatment-boosted ADA, treatment-induced ADA, treatment-unaffected ADA, or ADA negative. Due to the small sample size (9 participants) in each placebo group, it was prespecified to pool the 2 placebo cohorts for the immunogenicity assessment. | Posted | Count of Participants | Participants | Up to 16 weeks |
|
Up to 80 weeks
Adverse events were assessed at each participant visit. Laboratory abnormalities were reported as AEs.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | ARGX-117 Cohort 1 | Participants receiving a single dose of 30 mg/kg followed by 4 once- weekly doses of 10 mg/kg and a maintenance regimen of a single dose of 10 mg/kg every 2 weeks until the end of the DBTP (through intravenous infusions) | 0 | 18 | 2 | 18 | 14 | 18 |
| EG001 | Placebo Cohort 1 | Participants received placebo via intravenous (IV) infusion | 0 | 9 | 0 | 9 | 5 | 9 |
| EG002 | ARGX-117 Cohort 2 | Participants receiving a single dose of 15 mg/kg followed by 4 once- weekly doses of 5 mg/kg and a maintenance regimen of a single dose of 5 mg/kg every 4 weeks until the end of the DBTP (through intravenous infusions) | 0 | 18 | 0 | 18 | 14 | 18 |
| EG003 | Placebo Cohort 2 | Participants received placebo via IV infusion | 0 | 9 | 0 | 9 | 6 | 9 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute coronary syndrome | Cardiac disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Anal fungal infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Sinusitis bacterial | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Infusion site rash | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Vaccination site pain | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Catheter site haematoma | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Chills | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Feeling cold | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Amnesia | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Fine motor skill dysfunction | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Spinal pain | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Procedural headache | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Limb crushing injury | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Traumatic haematoma | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
| |
| Blood urine present | Investigations | MedDRA (24.1) | Systematic Assessment |
| |
| Protein urine | Investigations | MedDRA (24.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (24.1) | Systematic Assessment |
| |
| Antinuclear antibody increased | Investigations | MedDRA (24.1) | Systematic Assessment |
| |
| Bilirubin urine | Investigations | MedDRA (24.1) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (24.1) | Systematic Assessment |
| |
| Serum ferritin decreased | Investigations | MedDRA (24.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Pigmentation disorder | Skin and subcutaneous tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Abdominal rigidity | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (24.1) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Regulatory Manager | argenx BV | +32 93103400 | regulatory@argenx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 12, 2024 | May 30, 2025 | SAP_001.pdf |
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Not Reported |
|
| Other |
|
| Not Hispanic or Latino |
|
| Not Reported |
|
| Unknown |
|
| Number of Participants with a Treatment-related AE |
|
| Number of Participants with a SAE |
|
| Number of Participants with a Treatment-related SAE |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| OG003 | Placebo Cohort 2 | Participants received placebo via IV infusion |
|
|
| OG003 | Placebo Cohort 2 | Participants received placebo via IV infusion |
|
|
|
|
Participants received placebo via IV infusion
|
|
| OG003 | Placebo Cohort 2 | Participants received placebo via IV infusion |
|
|
| Placebo Cohort 2 |
Participants received placebo via IV infusion |
|
|
Participants received placebo via IV infusion |
|
|
Participants received placebo via IV infusion
|
|
Participants received placebo via IV infusion
|
|
|
|
Participants received placebo via IV infusion
|
|
|
|
|
|
| Placebo Cohort 2 |
Participants received placebo via IV infusion |
|
|
Participants received placebo via IV infusion |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Title | Measurements |
|---|---|
|
| 2 - Slight Problem |
|
| 3 - Moderate Problem |
|
| 4 - Severe Problem |
|
| 5 - Unable to |
|
| 2 - Slight Problem |
|
| 3 - Moderate Problem |
|
| 4 - Severe Problem |
|
| 5 - Unable to |
|
| 2 - Slight Problem |
|
| 3 - Moderate Problem |
|
| 4 - Severe Problem |
|
| 5 - Unable to |
|
| 2 - Slight Problem |
|
| 3 - Moderate Problem |
|
| 4 - Severe Problem |
|
| 5 - Unable to |
|
|