A Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adults With New-onset Generalized Myasthenia Gravis (gMG)
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18+
Sponsorargenx
About this trial
The main purpose of this study is to measure how well adults with new-onset gMG (which means they've had generalized disease signs and/or symptoms for less than 1 year) respond to treatment with efgartigimod PH20 SC. The study consists of a treatment period of 51 weeks. The study duration for each participant will be approximately 58 weeks.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Is at least 18 years when signing the ICF
Has been diagnosed with gMG of MGFA class II, III, or IV
Is seropositive for AChR-Ab
Is treatment-naive for gMG or has been administered AChEI for the treatment of gMG
Disqualifiers
gMG diagnosis of MGFA class I or V
Underwent a thymectomy prior to screening, except thymectomy for treatment of nonmalignant thymoma prior to the gMG diagnosis
Prior or current use of any of any systemic corticosteroid therapy or nonsteroidal immunosuppressive therapy for the treatment of gMG
Trial design
Design model
Single group
Treatments tested in this trial
Efgartigimod PH20 SC
Biological/VaccineSubcutaneous injection of efgartigimod PH20 SC
Treatment groups
30 Participants
are divided into 1 treatment groupGroup A: Efgartigimod PH20 SCExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Proportion of participants who achieve MSE of MG at any time within the first 16 weeks of treatment with efgartigimod PH20 SC
MSE (minimal symptom expression) is defined as an MG-ADL (Myasthenia Gravis Activities of Daily Living) total score of 0 or 1. The MG-ADL scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe).
Time frame
up to 16 weeksSecondary outcomes
1
Changes from baseline in MG-ADL total scores over time
The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe).
Time frame
up to 51 weeks2
Changes from baseline in MG-QOL-15r total scores over time
The MG-QoL15r (Myasthenia Gravis Quality of Life 15-item scale revised) questionnaire is a patient-reported instrument that measures the impact of MG symptoms on Quality of Life. The score varies between 0 and 30 (with 30 the lowest quality of life).
Time frame
up to 51 weeks3
Proportion of participants who are administered systemic corticosteroid therapy as background add-on therapy throughout the study
Time frame
up to 55 weeks4
Incidence of adverse events, serious adverse events and adverse events leading to study drug discontinuation
Time frame
up to 55 weeks Source ClinicalTrials.gov