Zilucoplan for Severe gMG Exacerbations
ConditionGeneralized Myasthenia Gravis (gMG)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorMiriam Freimer
About this trial
This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Patient determined to have severe MG exacerbation (e.g. bulbar and/or respiratory symptoms requiring hospitalization, neck extension weakness)
MGFA class II - IVb
Male or female aged ≥18
MG-ADL ≥6 in non-ocular domains
Disqualifiers
History of meningococcal disease
Participants requiring intubation prior to study start.
Recent significant infections which could have caused exacerbation e.g. sepsis and wound infections
Pregnancy or lactating
Trial design
Design model
Single group
Treatments tested in this trial
Zilucoplan®
DrugSubcutaneous injections of zilucoplan will be administered daily. The dose is dependent on the body weight of the patient. Each patient will receive a kit with prefilled syringes.
Treatment groups
15 Participants
are divided into 1 treatment groupGroup A: ZilucoplanExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Change from baseline to Week 2 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score
The MG-ADL is an assessment that measures MG symptom severity. There are 8 items measured on a scale of 0-3 with 0 being the least severe. The total of the 8 items represents the MG-ADL score. The score can range from 0 (least severe) to 24 (most severe).
Time frame
From baseline to Week 2Secondary outcomes
1
Change from baseline to week 12 in Myasthenia Gravis - Activities of Daily Living (MG-ADL) score
The MG-ADL is an assessment that measures MG symptom severity. There are 8 items measured on a scale of 0-3 with 0 being the least severe. The total of the 8 items represents the MG-ADL score. The score can range from 0 (least severe) to 24 (most severe).
Time frame
From baseline to week 12Sponsors and contacts
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Source ClinicalTrials.gov