A randomized, double-blind, placebo controlled, multicenter Phase 3 trial to evaluate the efficacy, safety, tolerability, quality of life and impact on normal daily activities of ARGX-113 in patients with gMG.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
167
Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered an MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to baseline of C1 \[C1B\]) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1.
Time frame: Baseline up to Day 63 (end of TC1)
Percentage of Quantitative Myasthenia Gravis (QMG) Responders During C1; Analyzed in the AChR-Ab Seropositive Population
The QMG scale quantifies disease severity based on impairments of body functions and structures as defined by the International Classification of Disability and Health. The QMG scale consists of 13 items that measure endurance or fatigability, and accounts for fluctuations in disease state. The QMG total score range is 0-39, with higher scores indicative of greater disease severity. A patient was considered a QMG responder during C1 if there was a reduction of ≥3-points on the QMG total score (compared to C1B) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1.
Time frame: Baseline up to Day 63 (end of TC1)
Percentage of MG-ADL Responders During C1; Analyzed in the Overall Population
The percentage of MG-ADL responders during C1 in the overall population is reported for this secondary end point; percentage of MG-ADL responders during C1 in the AChR-Ab seropositive population is reported previously as a primary end point.
Time frame: Baseline up to Day 63 (end of TC1)
Percentage of Time That Patients Had a Clinically Meaningful Improvement (CMI) in MG-ADL Total Score up to and Including Day 126; Analyzed in the AChR-Ab Seropositive Population
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Investigator Site 29
Phoenix, Arizona, United States
Investigator Site 66
Carlsbad, California, United States
Investigator Site 5
Los Angeles, California, United States
Investigator Site 49
Los Angeles, California, United States
Investigator Site 18
Orange, California, United States
Investigator Site 40
Palo Alto, California, United States
Investigator Site 59
San Francisco, California, United States
Investigator Site 58
Aurora, Colorado, United States
Investigator Site 34
Jacksonville, Florida, United States
Investigator Site 4
Tampa, Florida, United States
...and 56 more locations
An MG-ADL CMI was defined as a reduction of ≥2 points on the total MG-ADL score compared to study entry baseline (SEB).
Time frame: Baseline up to Day 126
Time From Week 4 to Qualify for Retreatment; Analyzed in the AChR-Ab Seropositive Population
Time to qualify for retreatment was defined as time from the Week 4 assessment until the first visit with a \<2-point reduction compared to SEB in the MG-ADL total score and MG-ADL total score ≥5 points with \>50% of the total score attributable to nonocular symptoms.
Time frame: Week 4 up to Day 182 (end of study [EoS])
Percentage of Early MG-ADL Responders During C1; Analyzed in the AChR-Ab Seropositive Population
A patient was considered an early MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to C1B) for ≥4 consecutive weeks with the first reduction occurring no later than Week 2 (ie, after 1 or maximum 2 infusions of IMP in C1).
Time frame: Baseline up to Day 63 (end of TC1)