The purpose of this study is to evaluate the long-term safety and tolerability of efgartigimod PH20 SC 1000 mg, and the clinical efficacy, PD, pharmacokinetics (PK), immunogenicity, impact on the quality of life (QoL) of the participants, treatment satisfaction, and administration method preference, and the feasibility of self- and caregiver-supported administration of the SC injection. Treatment duration: 3-week treatment periods, repeated as needed with at least 28 days in between treatment periods Health measurements: total levels of immunoglobulin G (IgG), Acetylcholine receptor binding autoantibodies (AChR-Ab) levels, Myasthenia Gravis Activities of Daly Living (MG-ADL).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
184
Subcutaneous injection with efgartigimod PH20 SC
Investigator site 6 - US0010032
Carlsbad, California, United States
Investigator Site 47 - US0010021
Palo Alto, California, United States
Investigator Site 45 - US0010108
Boca Raton, Florida, United States
Investigator site 4 - US0010110
Port Charlotte, Florida, United States
Investigator Site 39 - US0010006
Tampa, Florida, United States
Number of AEs, SAEs and AESIs
Adverse events, Serious Adverse event and Adverse events of special interest. Adverse events in the 'Infections and infestations' SOC were defined as AESIs because efgartigimod causes a transient reduction in total IgG levels.
Time frame: Up to 3.5 years
MG-ADL Total Score Changes From Baseline
The Myasthenia Gravis Activities of Daily Living (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. The total score ranges from 0 to 24 with higher scores indicating more impairment.
Time frame: Up to week 4 of the first cycle
Percent Change in Total IgG Levels From Baseline
IgG: immunoglobulin G
Time frame: Up to week 4 of the first cycle
Percent Change in AChR-Ab From Baseline in AChR-Ab Seropositive Participants
AchR-Ab: anti-acetylcholine receptor antibodies.
Time frame: Up to week 4 of the first cycle
Efgartigimod Serum Concentrations
Time frame: Up to week 4 of the first cycle
Incidence of ADAs Against Efgartigimod Over Time
ADA: Anti-drug antibodies.
Time frame: Up to 3.5 years
Incidence of NAbs Against Efgartigimod Over Time
NAbs: neutralizing antibodies
Time frame: Up to 3.5 years
Incidence of Antibodies Against rHuPH20 Over Time
Time frame: Up to 3.5 years
Incidence of NAbs Against rHuPH20 Over Time
NAbs: neutralizing antibodies
Time frame: Up to 3.5 years
Changes in Total MG-QoL15r From Baseline
Myasthenia Gravis Quality of Life 15 item scale revised (MG-QoL 15r) scores range from 0 to 30 with a higher score representing more severe symptoms.
Time frame: Up to week 4 of the first cycle
Changes in EQ-5D-5L VAS Score From Baseline
EuroQoL 5 Dimensions 5-Level (EQ-5D-5L) visual analog scale (VAS) scores range from 0 to 100 with a higher score representing better health.
Time frame: Up to week 4 of the first cycle
Percent of Participants or Caregivers by Number of Training Visits Needed to be Competent to Start Self- or Caregiver-Supported Administration
Time frame: Up to 3.5 years
Percent of Self- or Caregiver-supported Study Drug Administration Among All Study Treatment Visits at Home
Time frame: Up to 3.5 years
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Investigator Site 41 - US0010015
Kansas City, Kansas, United States
Investigator Site 46 - US0010111
Amherst, New York, United States
Investigator Site 38 - US0010003
Chapel Hill, North Carolina, United States
Investigator Site 44 - US0010077
Durham, North Carolina, United States
Investigator Site 42 - US0010019
Cleveland, Ohio, United States
...and 37 more locations