Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Study Overview
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Brief Summary
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The RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.

Official Title
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A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Conditions
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Generalized Myasthenia Gravis

Intervention / Treatment
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* Drug: zilucoplan (RA101495)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Completion of a qualifying zilucoplan study Exclusion Criteria: With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted)

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 0.3 mg/kg zilucoplan (RA101495)<br> | Drug: zilucoplan (RA101495)<br>* Daily subcutaneous (SC) injection<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events (TEAEs) | A treatment-emergent adverse event is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. | From Baseline (Day 1) to Safety Follow-Up Visit (up to 36 months) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score | The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 8 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe). | From Baseline (Day 1) to Week 12 | 
| Change from Baseline to Week 12 in the the Quantitative Myasthenia Gravis (QMG) Score | The QMG test is a standardized quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score. The QMG score can range from 0 (least severe) to 39 (most severe). | From Baseline (Day 1) to Week 12 | 
| Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score | The MGC is a 10-item scale that has been used to measure the clinical status of patients with Myasthenia Gravis (MG) in order to evaluate treatment response. The MGC has 4-point Likerttype Scale response options ranging from 0 to 2, 3, 4, 5, 6 or 9 according to the item (weighted response options). The total score is the sum of all items (range 0-50) where higher scores indicate more severe impairment due to the disease. | From Baseline (Day 1) to Week 12 | 
| Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score | The MG-QOL15r is a 15-item survey that was designed to assess quality of life in patients with MG. The MG-QoL has 3-point Likert Scale response options ranging from 0 to 2. The MGQoL15r score can range from 0 to 30, where higher scores indicate more severe impact of the disease on aspects of the patient's life. | From Baseline (Day 1) to Week 12 |