This is to study the efficacy, safety and tolerability of efgartigimod in patients with seronegative generalized myasthenia gravis. This is an open label study. There will be 30 participants to enroll at University Health Network Toronto General Hospital. Study duration is 43 weeks from screening to end of study.
There will be screening period that includes ECG, safety blood collection, collection of demographics, vital signs, questionnaires, weight, pregnancy test (if applicable), genetic test. If eligible, participants will have run-in period from week 2 to week 5. Baseline is at week 6. Induction phase will be weekly from week 7, 8, and 9. Maintenance phase will be once every 2 weeks thereafter. Observation period will have 4 visits, End of study is at week 43.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
active drug efgartigimod to be administer
University Health Network, Division of Neurology, Toronto General Hospital
Toronto, Ontario, Canada
RECRUITINGTotal Myasthenia Gravis Impairment Index (MGII) score
Myasthenia Gravis Impairment Index (MGII) is a composite score based on a patient self-reported multidomain questionnaire as well as physician-documented clinical measures. MGII has 22 patient-reported and 6 physician-assessed items with a final composite score of 84, with higher score signifying greater disability.
Time frame: through study completion for 42 weeks
MG-ADL (Myasthenia Gravis-Activities of Daily Living)
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity.
Time frame: through study completion for 42 weeks
QMG (Quantitative Myasthenia Gravis) score
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.
Time frame: through study completion for 42 weeks
MG-QOL (Myasthenia Gravis Quality of Life)
MG-QOL revised consists of 15 items/ question. These include mobility (9 items), symptoms (3 items), and contentment and emotional well-being (3 items). Items are scored in a Likert scale from 0 to 4 and the total sum score ranges from 0 to 60, where higher scores indicate worse HRQoL.
Time frame: through study completion for 42 weeks
PASS (Patient Acceptable Symptom State) response
The PASS response is based on a dichotomous 'Yes' or 'No' response, asking whether a patient is satisfied overall with their current status and thus measures holistic satisfaction with their MG state. The PASS response of "Yes" has been shown to correlate with a favourable MGII and the simple single question (SSQ, percentage of normal).
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Time frame: through study completion for 42 weeks
Single Simple Question (SSQ)
The SSQ is a question that asks what percentage of normal that the patient feels with respect to their MG. A SSQ threshold ≥72.5% out of normal at 100% predicts an acceptable MG state.
Time frame: through study completion for 42 weeks
Myasthenia Gravis Foundation of America (MGFA)
The MGFA classes are pure ocular (class I), mild generalized (class II), moderate generalized (class III), severe generalized (class IV), and intubation/myasthenic crisis (class V). Within the generalized categories II, III, and IV, patients are subclassified as class A if their symptoms are predominantly generalized or class B if their symptoms are predominantly bulbar. The MGFA also has a system to classify patients based on postintervention outcomes and includes remission, defined as 1 year or longer without signs or symptoms and without any symptomatic (pyridostigmine) treatment, and which can be divided in complete (no pharmacologic treatment at all) or pharmacologic remission. Minimal manifestation status is defined as minimal signs or symptoms (no specific timeframe was defined) and pyridostigmine use may be accepted.
Time frame: through study completion for 42 weeks