Seronegative Myasthenia Gravis - Efgartigimod IV
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorUniversity Health Network, Toronto
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Evidence of signed and dated informed consent document(s) indicating that the subject has been informed of all pertinent aspects of the trial. Subjects must be willing and able to comply with the protocol, complete study assessments, and return for follow-up visits.
Male or female subjects ≥ 18 years old.
Diagnosis of SN MG defined as: (a) clinical syndrome consistent with a diagnosis of MG, and not otherwise explained by another condition, (b) abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; and (c) negative serologic test for anti-AChR and anti- MuSK antibodies as confirmed at screening, (d) limited, if any, response to therapy with immunotherapy and/or antiacetylcholinesterase (AChE) treatment. Further testing for low affinity antibodies to rapsyn-clustered AChR by cell-based assays will be done at baseline and the results included as part of subgroup analysis. All patients will have a negative genetic test for congenital myasthenic syndromes by history or at baseline to exclude the possibility of congenital myasthenic syndrome mimicking SN MG.
MGFA Clinical Classification Class II, III, or IV at the time of screening and baseline.
Disqualifiers
Patients who discontinued early from trials of efgartigimod for pregnancy or rescue reasons or an SAE that was likely to result in a life-threatening situation or pose a serious safety risk.
Pregnant and lactating women, and those intending to become pregnant during the trial or within 3 months after the last dosing. Women of childbearing potential should have a negative urine pregnancy test at screening and baseline.
Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 3 months after the last dosing or male patients who plan to donate sperm during the trial or within 3 months after the last dosing.
Patients with known hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) seropositivity.
Trial design
Design model
Single group
Treatments tested in this trial
efgartigimod
Biological/Vaccineactive drug efgartigimod to be administer
Treatment groups
30 Participants
are divided into 1 treatment groupGroup A: efgartigimodOther 1 intervention
Trial outcomes
Primary outcomes
1
Total 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 weeksSecondary outcomes
1
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 weeks2
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 weeks3
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 weeks4
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).
Time frame
through study completion for 42 weeksSponsors and contacts
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Source ClinicalTrials.gov