A Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Patients with Generalized Myasthenia Gravis
- Conditions
- Generalized Myasthenia GravisMedDRA version: 20.0Level: LLTClassification code 10028415Term: MyastheniaSystem Organ Class: 100000004859Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2019-001564-30-NO
- Lead Sponsor
- Ra Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 174
To be eligible for this study, subjects must meet ALL the following inclusion criteria:
1.Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
2.Positive serology for acetylcholine receptor (AChR) binding autoantibodies
3.MG-ADL Score of =6 at Screening and Baseline
4.QMG Score of =12 at Screening and Baseline (off acetylcholinesterase inhibitor therapy for at least 10 hours)
5.No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week treatment period
6.No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week treatment period
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 110
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 46
Subjects who meet ANY of the following exclusion criteria must be excluded from the study:
1.Thymectomy within 12 months prior to baseline or scheduled to occur during the 12-week study
2.History of meningococcal disease
3.Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: •To confirm the efficacy of zilucoplan in subjects with gMG <br>•To confirm the safety and tolerability of zilucoplan in subjects with gMG <br>;Secondary Objective: Not Applicable;Primary end point(s): •Change from Baseline (CFB) in Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score;Timepoint(s) of evaluation of this end point: week 12
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. CFB to Week 12 in the Quantitative Myasthenia Gravis (QMG) Score<br>2. CFB to Week 12 in the Myasthenia Gravis Composite (MGC) Score<br>3. CFB to Week 12 in the Myasthenia Gravis – Quality of Life revised (MG-QOL15r) Score<br>4. Time to first receipt of rescue therapy over the 12-week Treatment Period<br>5. Achieving Minimal Symptom Expression (MSE), defined as an MG-ADL of 0 or 1 at Week 12 without rescue therapy<br>6. Achieving a = 3-point reduction in MG-ADL Score at Week 12 without rescue therapy<br>7. Achieving a =5-point reduction in QMG Score without rescue therapy at Week 12<br>8. Incidence of treatment-emergent AEs;Timepoint(s) of evaluation of this end point: Endpoints 1-7: week 12<br>Endpoint 8: Throughout the study