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The purpose of this study is to investigate the Pharmacodynamics (PD), Pharmacokinetics (PK), safety, tolerability, immunogenicity, and clinical efficacy of efgartigimod coformulated with recombinant human hyaluronidase PH20 (rHuPH20) as compared to efgartigimod IV infused in patients with generalized myasthenia gravis (gMG). The study duration is approximately 12 weeks. After screening, patients will be randomized to receive either efgartigimod infusions or efgartigimod PH20 subcutaneously (SC)
Main objective of the trial: To demonstrate that the pharmacodynamic (PD) effect of injections of 1000 mg efgartigimod PH20 SC (efgartigimod co-formulated with recombinant humanhyaluronidase PH20 for subcutaneous administration), administered once weekly for 4 administrations, is NI (noninferior) to IV infusions of efgartigimod (efgartigimod formulation for intravenous infusion) at a dose of 10 mg/kg administered once weekly for 4 administrations.
Secondary objectives: To compare the PD effect of efgartigimod PH20 SC and efgartigimod IV over time; To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC and efgartigimod IV; To evaluate the safety, tolerability, and immunogenicity of efgartigimod PH20 SC and efgartigimod IV; To evaluate the clinical efficacy of efgartigimod PH20 SC and efgartigimod IV.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| efgartigimod PH20 SC | Experimental | Patients receiving efgartigimod PH20 subcutaneous (SC) treatment |
|
| efgartigimod | Experimental | Patients receiving efgartigimod intravenous (IV) treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| efgartigimod PH20 SC | Biological | Subcutaneous injection with efgartigimod PH20 SC |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Total IgG Levels at Day 29 (mITT Analysis Set) | ANCOVA Analysis of Percent Change From Baseline in Total IgG Level at Day 29 (ie, 7 days after the fourth IV or SC administration). | From week 0 to week 4 |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set) | Total IgG level percent change from baseline over time for the overall population. | From baseline to week 10 |
| Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set) |
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Inclusion Criteria:
Bullet list of each inclusion criterium:
Must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
At least 18 years of age at the time of signing the informed consent form.
Diagnosed with generalized Myasthenia Gravis (gMG) with confirmed documentation and supported by at least 1 of the following:
Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa, or IVb
Exclusion Criteria:
Bullet list of each exclusion criterium:
Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of Investigational Medicinal Product.
Has any of the following medical conditions:
Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk.
History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time:
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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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigator site 2 - US0010032 | Carlsbad | California | 92011 | United States | ||
| Investigator Site 41 - US0010004 |
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153 patients were screened, 111 patients were randomized 1:1 to receive efgartigimod PH20 SC 1000 mg (55) or efgartigimod IV 10 mg/kg (56) once weekly for 4 administrations (4 doses on days 1, 8, 15, and 22). 110 patients started in the study (received treatment) as one participant randomized to the efgartigimod IV arm did not receive treatment due to an AE. 55 patients received study treatment in each treatment arm.
Participant's evaluation of eligibility was performed at screening and confirmed at randomization visit 1. The overall study duration per subject was approximately 12 weeks spanning the study periods - 2 weeks for screening, 3 weeks for treatment, and 7 weeks for follow-up.
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| ID | Title | Description |
|---|---|---|
| FG000 | Efgartigimod PH20 SC | Patients receiving efgartigimod PH20 subcutaneous (SC) treatment. efgartigimod PH20 SC: Subcutaneous injection with 1000 mg efgartigimod PH20 SC |
| FG001 | Efgartigimod IV |
| 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 | Jul 2, 2021 | Nov 22, 2022 |
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| efgartigimod IV | Biological | Intravenous infusion of efgartigimod |
|
Percent reduction from baseline in AChR-Ab levels over time in AChR-Ab positive patients measured in mITT Analysis Set. Descriptive statistics have been used for this secondary end point. |
| From baseline to week 10 |
| Percent Change From Baseline in IgG Subtype Levels Over Time (mITT Analysis Set) | Median (IQR) Percent Change From Baseline for the IgG Subtypes (IgG1, IgG2, IgG3, and IgG4) in the Overall Population. The highest number of patients among all weeks for the analysis is chosen for each arm. Descriptive statistics have been used for this secondary end point. | Baseline to week 10 |
| AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set) | AUEC of the percent reduction from baseline total IgG per dosing interval (days 1-8, days 8-15, days 15-22, and days 22-29), days 1-29, days 1-57 and over the entire study (days 1-71). The highest number of patients among all weeks for the analysis is chosen for each arm. Descriptive statistics have been used for this secondary end point. | From baseline to week 10 |
| Еfgartigimod IV and PH20 SC Serum Pharmacokinetic Parameter Ctrough | Evaluation of observed predose concentration (Ctrough) (after all doses for the IV and SC treatment arms). The analysis will present data from Week 1 to Week 4. Descriptive statistics have been used for this secondary end point. | From Week 1 to Week 4. |
| Efgartigimod IV Serum Pharmacokinetic Parameter Cmax | Evaluation of maximum observed concentration (Cmax) (after all doses for the IV treatment arm). The analysis will present data from Baseline to Week 3. Descriptive statistics have been used for this secondary end point. | From Baseline to Week 3 |
| Incidence of ADA Against Efgartigimod (Safety Analysis Set) | Incidence of antidrug antibodies (ADA) against Efgartigimod in the overall population. ADA analysis is performed with a validated ELISA in a 3-tiered approach (ADA screening analysis, confirmatory analysis and a titration assay). Descriptive statistics have been used for this secondary end point. | From baseline to week 10 |
| Incidence of Antibodies Against rHuPH20 in the SC Treatment Arm (Safety Analysis Set) | Incidence of antibodies against rHuPH20 in the Efgartigimod PH20 SC Arm. antibody analysis is performed with a validated ELISA in a 3-tiered approach (screening analysis, confirmatory analysis and a titration assay) Descriptive statistics have been used for this secondary end point. | From baseline to week 10 |
| Incidence and Severity of AEs and SAEs (Safety Analysis Set) | Evaluation of incidence and severity of treatment-emergent adverse events (TEAEs) and incidence of serious AEs (SAEs). Descriptive statistics have been used for this secondary end point. | From baseline to week 10 |
| MG-ADL Responders (ITT Analysis Set) | Evaluation of number and percentage of Myasthenia Gravis Activities of Daily Living (MG-ADL) responders in the overall population. The MG-ADL is an 8-item patient-reported scale that assesses MG symptoms and their effects on daily activities. It evaluates a participant's capacity to perform different activities in their daily life, including talking, chewing, swallowing, breathing, brushing their teeth, combing their hair, or getting up from a chair. The MG-ADL also assesses double vision and eyelid droop. It is a discrete quantitative variable in which the 8 items are rated by the participant on a scale of 0 to 3. The total score can range from 0 to 24, with higher total scores indicating more impairment. A participant was considered a MG-ADL responder if he/she showed a reduction of at least 2 points from baseline on the MG-ADL score for at least 4 consecutive weeks. Descriptive statistics have been used for this secondary end point. | From baseline to week 10 |
| QMG Responders (ITT Analysis Set) | Evaluation of number and percentage of Quantitative Myasthenia Gravis (QMG) responders in the overall population (ITT Analysis Set). Descriptive statistics have been used for this secondary end point. One subject in the EFG IV arm had no post-baseline QMG assessment and thus was excluded from the denominator. | From Baseline to Week 10 |
| Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set) | Evaluation of MG-ADL Total Score Change from baseline over time for the overall population (ITT Analysis Set). Descriptive statistics have been used for this secondary end point. The MG-ADL is an 8-item patient-reported scale that assesses MG symptoms and their effects on daily activities. It evaluates a participant's capacity to perform different activities in their daily life, including talking, chewing, swallowing, breathing, brushing their teeth, combing their hair, or getting up from a chair. The MG-ADL also assesses double vision and eyelid droop. It is a discrete quantitative variable in which the 8 items are rated by the participant on a scale of 0 to 3. The total score can range from 0 to 24, with higher total scores indicating more impairment. A participant was considered a MG-ADL responder if he/she showed a reduction of at least 2 points from baseline on the MG-ADL score for at least 4 consecutive weeks. | From baseline to week 10 |
| Change From Baseline in QMG Score Over Time (ITT Analysis Set) | Evaluation of QMG Total Score change from baseline over time for the overall population (ITT Analysis Set). The QMG (Quantitative Myasthenia Gravis) quantifies disease severity based on impairments of body function and structures as defined by the International Classification of Functioning, Disability and Health. The QMG consists of 13 items that assess ocular, bulbar, and limb function. Six of the 13 items are timed endurance tests measured in seconds. Each item has a possible score from 0 to 3. The total possible score is 39, where higher total scores indicate more severe impairments. It is based on qualitative testing of specific muscle groups to assess limb function. Descriptive statistics have been used for this secondary end point. A participant was considered a QMG responder if he/she showed a reduction of at least 3 points from baseline on the QMG score for at least 4 consecutive weeks. | From baseline to week 10 |
| Orange |
| California |
| 92868 |
| United States |
| Investigator site 2 - US0010108 | Boca Raton | Florida | 33428 | United States |
| Investigator site 1 - US0010110 | Port Charlotte | Florida | 33952 | United States |
| Investigator Site 27 - US0010006 | Tampa | Florida | 41076 | United States |
| Investigator Site 47 - US0010113 | Augusta | Georgia | 30912 | United States |
| Investigator Site 42 - US0010015 | Kansas City | Kansas | 66205 | United States |
| Investigator Site 11 - US0010111 | Amherst | New York | 14226 | United States |
| Investigator Site 40 - US0010003 | Chapel Hill | North Carolina | 27514 | United States |
| Investigator Site 38 - US0010077 | Durham | North Carolina | 27710 | United States |
| Investigator Site 43 - 0010019 | Cleveland | Ohio | 44195 | United States |
| Investigator site 4 - US0010008 | Cordova | Tennessee | 38018 | United States |
| Investigator Site 28 - US0010066 | Austin | Texas | 78756 | United States |
| Investigator Site 46 - US0010009 | Texas City | Texas | 78229 | United States |
| Investigator site 5 - BE0320007 | Ghent | East-Flanders | 9000 | Belgium |
| Investigator site 13 - GE9950002 | Tbilisi | 0112 | Georgia |
| Investigator site 12 - GE9950001 | Tbilisi | 0114 | Georgia |
| Investigator site 14 - GE9950003 | Tbilisi | 0114 | Georgia |
| Investigator Site 44 - GE9950004 | Tbilisi | 0160 | Georgia |
| Investigator Site 45 - GE9950016 | Tbilisi | 0160 | Georgia |
| Investigator Site 30 - DE490006 | Berlin | 10117 | Germany |
| Investigator Site 29 - DE490009 | Münster | 48149 | Germany |
| Investigator site 15 - HU0360013 | Budapest | 1082 | Hungary |
| Investigator Site 16 - HU0360020 | Debrecen | 4032 | Hungary |
| Investigator Site 17 - IT0390003 | Milan | 20133 | Italy |
| Investigator Site 39 - IT0390008 | Roma | 00189 | Italy |
| Investigator Site 31 - JP0810055 | Sapporo | Hokkaido | 063-0005 | Japan |
| Investigator Site 18 - JP0810002 | Chiba | 260-8677 | Japan |
| Investigator site 6 - JPN0810004 | Hanamaki | 025-0082 | Japan |
| Investigator Site 33 - JP0810058 | Hiroshima | 0810058 | Japan |
| Investigator Site 19 - JP0810007 | Osaka | 565-0871 | Japan |
| Investigator Site 34 - JP0810005 | Sendai | 983-8520 | Japan |
| Investigator Site 32 - JP0810059 | Tokyo | 143-8541 | Japan |
| Investigator Site 20 - JP0810009 | Tokyo | 160-0023 | Japan |
| Investigator Site 7 - NL0310001 | Leiden | 2333 | Netherlands |
| Investigator Site 21 - PL0480001 | Gdansk | 80-952 | Poland |
| Investigator Site 8 - PL0480007 | Katowice | 40-123 | Poland |
| Investigator Site 9 - PL0480024 | Krakow | 31-2002 | Poland |
| Investigator Site 22 - PL0480005 | Krakow | 31-505 | Poland |
| Investigator Site 23 - PL0480018 | Lublin | 20-093 | Poland |
| Investigator Site 24 - PL0480022 | Warsaw | 02-097 | Poland |
| Investigator Site 35 - RU0070002 | Novosibirsk | 630087 | Russia |
| Investigator Site 36 - RU0070014 | Saint Petersburg | 194354 | Russia |
| Investigator Site 37 - ES0340021 | Barcelona | 08035 | Spain |
| Investigator Site 26 - ES0340038 | Barcelona | 08041 | Spain |
| Investigator Site 25 - ES0340002 | Madrid | 28046 | Spain |
| Investigator Site 10 - ES0340039 | Valencia | 46026 | Spain |
Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment.
efgartigimod IV: Intravenous infusion of efgartigimod
| COMPLETED |
|
| NOT COMPLETED |
|
|
ITT analysis set: All randomized participants who were exposed to the IMP. mITT analysis set: All randomized participants with a value for total IgG levels at baseline and at least 1 postbaseline time point. Safety analysis set: All randomized participants who were exposed to IMP. PK analysis set: A subset of the safety analysis set with at least 1 post dose PK measurement.
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| ID | Title | Description |
|---|---|---|
| BG000 | Efgartigimod PH20 SC | Patients receiving 1000 mg efgartigimod PH20 subcutaneous (SC) treatment. efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC |
| BG001 | Efgartigimod IV | Patients receiving 10 mg/kg efgartigimod intravenous (IV) treatment. efgartigimod IV: Intravenous infusion of efgartigimod |
| BG002 | 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 | Full Range | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | 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 | Percent Change From Baseline in Total IgG Levels at Day 29 (mITT Analysis Set) | ANCOVA Analysis of Percent Change From Baseline in Total IgG Level at Day 29 (ie, 7 days after the fourth IV or SC administration). | Posted | Least Squares Mean | 95% Confidence Interval | percent | From week 0 to week 4 |
|
|
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set) | Total IgG level percent change from baseline over time for the overall population. | mITT Analysis Set: All randomized participants with a value for total IgG levels at baseline and at least 1 postbaseline timepoint. | Posted | Mean | Standard Error | percent | From baseline to week 10 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set) | Percent reduction from baseline in AChR-Ab levels over time in AChR-Ab positive patients measured in mITT Analysis Set. Descriptive statistics have been used for this secondary end point. | Posted | Mean | Standard Error | percent | From baseline to week 10 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in IgG Subtype Levels Over Time (mITT Analysis Set) | Median (IQR) Percent Change From Baseline for the IgG Subtypes (IgG1, IgG2, IgG3, and IgG4) in the Overall Population. The highest number of patients among all weeks for the analysis is chosen for each arm. Descriptive statistics have been used for this secondary end point. | mITT Analysis Set: All randomized participants with a value for total IgG levels at baseline and at least 1 postbaseline timepoint. | Posted | Median | Inter-Quartile Range | Percent change | Baseline to week 10 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set) | AUEC of the percent reduction from baseline total IgG per dosing interval (days 1-8, days 8-15, days 15-22, and days 22-29), days 1-29, days 1-57 and over the entire study (days 1-71). The highest number of patients among all weeks for the analysis is chosen for each arm. Descriptive statistics have been used for this secondary end point. | Posted | Mean | Standard Error | percent days | From baseline to week 10 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Еfgartigimod IV and PH20 SC Serum Pharmacokinetic Parameter Ctrough | Evaluation of observed predose concentration (Ctrough) (after all doses for the IV and SC treatment arms). The analysis will present data from Week 1 to Week 4. Descriptive statistics have been used for this secondary end point. | Posted | Mean | Standard Deviation | μg/mL | From Week 1 to Week 4. |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Efgartigimod IV Serum Pharmacokinetic Parameter Cmax | Evaluation of maximum observed concentration (Cmax) (after all doses for the IV treatment arm). The analysis will present data from Baseline to Week 3. Descriptive statistics have been used for this secondary end point. | Posted | Mean | Standard Deviation | μg/mL | From Baseline to Week 3 |
|
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| Secondary | Incidence of ADA Against Efgartigimod (Safety Analysis Set) | Incidence of antidrug antibodies (ADA) against Efgartigimod in the overall population. ADA analysis is performed with a validated ELISA in a 3-tiered approach (ADA screening analysis, confirmatory analysis and a titration assay). Descriptive statistics have been used for this secondary end point. | Posted | Number | Percent of patients | From baseline to week 10 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Incidence of Antibodies Against rHuPH20 in the SC Treatment Arm (Safety Analysis Set) | Incidence of antibodies against rHuPH20 in the Efgartigimod PH20 SC Arm. antibody analysis is performed with a validated ELISA in a 3-tiered approach (screening analysis, confirmatory analysis and a titration assay) Descriptive statistics have been used for this secondary end point. | Posted | Number | Percent of patients | From baseline to week 10 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Incidence and Severity of AEs and SAEs (Safety Analysis Set) | Evaluation of incidence and severity of treatment-emergent adverse events (TEAEs) and incidence of serious AEs (SAEs). Descriptive statistics have been used for this secondary end point. | Posted | Number | Percent of patients | From baseline to week 10 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | MG-ADL Responders (ITT Analysis Set) | Evaluation of number and percentage of Myasthenia Gravis Activities of Daily Living (MG-ADL) responders in the overall population. The MG-ADL is an 8-item patient-reported scale that assesses MG symptoms and their effects on daily activities. It evaluates a participant's capacity to perform different activities in their daily life, including talking, chewing, swallowing, breathing, brushing their teeth, combing their hair, or getting up from a chair. The MG-ADL also assesses double vision and eyelid droop. It is a discrete quantitative variable in which the 8 items are rated by the participant on a scale of 0 to 3. The total score can range from 0 to 24, with higher total scores indicating more impairment. A participant was considered a MG-ADL responder if he/she showed a reduction of at least 2 points from baseline on the MG-ADL score for at least 4 consecutive weeks. Descriptive statistics have been used for this secondary end point. | Posted | Number | percent | From baseline to week 10 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | QMG Responders (ITT Analysis Set) | Evaluation of number and percentage of Quantitative Myasthenia Gravis (QMG) responders in the overall population (ITT Analysis Set). Descriptive statistics have been used for this secondary end point. One subject in the EFG IV arm had no post-baseline QMG assessment and thus was excluded from the denominator. | One patient from Efgartigimod IV didn't have any post-baseline QMG assessment. | Posted | Number | percent | From Baseline to Week 10 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set) | Evaluation of MG-ADL Total Score Change from baseline over time for the overall population (ITT Analysis Set). Descriptive statistics have been used for this secondary end point. The MG-ADL is an 8-item patient-reported scale that assesses MG symptoms and their effects on daily activities. It evaluates a participant's capacity to perform different activities in their daily life, including talking, chewing, swallowing, breathing, brushing their teeth, combing their hair, or getting up from a chair. The MG-ADL also assesses double vision and eyelid droop. It is a discrete quantitative variable in which the 8 items are rated by the participant on a scale of 0 to 3. The total score can range from 0 to 24, with higher total scores indicating more impairment. A participant was considered a MG-ADL responder if he/she showed a reduction of at least 2 points from baseline on the MG-ADL score for at least 4 consecutive weeks. | Posted | Mean | Standard Error | Total score on a scale | From baseline to week 10 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in QMG Score Over Time (ITT Analysis Set) | Evaluation of QMG Total Score change from baseline over time for the overall population (ITT Analysis Set). The QMG (Quantitative Myasthenia Gravis) quantifies disease severity based on impairments of body function and structures as defined by the International Classification of Functioning, Disability and Health. The QMG consists of 13 items that assess ocular, bulbar, and limb function. Six of the 13 items are timed endurance tests measured in seconds. Each item has a possible score from 0 to 3. The total possible score is 39, where higher total scores indicate more severe impairments. It is based on qualitative testing of specific muscle groups to assess limb function. Descriptive statistics have been used for this secondary end point. A participant was considered a QMG responder if he/she showed a reduction of at least 3 points from baseline on the QMG score for at least 4 consecutive weeks. | Posted | Mean | Standard Error | Total score | From baseline to week 10 |
|
up to 12 weeks (throughout the treatment period)
AEs will be reported by the participant (or, when appropriate, by a caregiver, surrogate, legally authorized representative). The investigator are responsible for detecting, documenting, and recording events that meet the definition of an AE or SAE and remain responsible for following up AEs that are serious, considered related to the study intervention or study procedures, or that caused the participant to discontinue the study intervention.
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 | Efgartigimod PH20 SC | Patients receiving efgartigimod PH20 subcutaneous (SC) treatment efgartigimod PH20 SC: Subcutaneous injection with efgartigimod PH20 SC | 0 | 55 | 8 | 55 | 37 | 55 |
| EG001 | Efgartigimod IV | Patients receiving efgartigimod intravenous (IV) treatment efgartigimod IV: Intravenous infusion of efgartigimod | 0 | 55 | 4 | 55 | 23 | 55 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Myasthenia gravis | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Optic neuritis | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Testicular cyst | Reproductive system and breast disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Contusion | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 24.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Myasthenia gravis | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Injection site rash | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
|
None reported.
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Regulatory Manager | argenx BV | +32 9 310 34 00 | regulatory@argenx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 16, 2022 | Nov 22, 2022 | SAP_001.pdf |
Not provided
| 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 |
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Cmax Baseline |
| |||||
| Cmax week 1 |
| |||||
| Cmax week 2 |
| |||||
| Cmax week 3 |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|