Phase 2
N=45
Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Bottom Line
View on ClinicalTrials.gov: NCT03315130 ↗Enrolled (actual)
45
Serious AEs
25.9%
Results posted
Jun 2022
Primary outcome: Primary: Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score — -5.5; -6.0; -3.2 score on a scale — p==0.0941
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- zilucoplan (RA101495) (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ra Pharmaceuticals
- Primary completion
- Dec 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score |
-5.5; -6.0; -3.2 | =0.0941 |
| SECONDARY Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale |
-3.3; -3.4; -1.1 | =0.0470 sig |
| SECONDARY Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life 15r (MG-QOL15r) Survey |
-7.4; -5.9; -2.1 | =0.0170 sig |
| SECONDARY Main Portion: Change From Baseline to Week 12 in the MG Composite Scale Total Score |
-5.3; -7.4; -3.3 | =0.1866 |
| SECONDARY Main Portion: Percentage of Participants With >= 3-point Reduction in QMG Total Score at Week 12 |
66.7; 71.4; 53.3 | — |
| SECONDARY Main Portion: Percentage of Participants Who Required Rescue Therapy Over the 12-week Treatment Period |
6.7; 0; 20.0 | — |
| SECONDARY Main Portion: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
15; 12; 14; 0; 5; 3 | — |
| SECONDARY Main Portion: Change From Baseline in Sheep Red Blood Cell (sRBC) Lysis Assay at Week 12 (Pre-dose) |
-81.822; -94.914; 0.775 | <0.0001 sig |
| SECONDARY Main Portion: Change From Baseline in C5 Levels at Week 12 (Pre-dose) |
57.349; 54.302; -2.774 | <0.0001 sig |
| SECONDARY Main Portion: Plasma Concentration of RA101495 and Its Major Metabolites |
652.136; 2272.257; 1691.267; 4176.000; 1925.357; 4636.000 | — |
| SECONDARY Main Portion: Maximum Plasma Concentration (Cmax) on Day 1 |
1945.467; 4858.643 | — |
| SECONDARY Main Portion: Time Corresponding to Cmax (Tmax) on Day 1 |
4.550; 4.700 | — |
| SECONDARY Main Portion: Metabolites (RA102758 and RA103488) to Parent Ratio |
NA; NA; 0.000; 0.000; 0.000; 0.000 | — |
Summary
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IVa] at Screening
- Positive serology for acetylcholine receptor (AChR) autoantibodies
- QMG score ≥ 12 at Screening and Randomization
- No change in corticosteroid dose for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
- No change in immunosuppressive therapy, including dose, for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
Exclusion Criteria
- Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12 week Treatment Period
- History of meningococcal disease
- Current or recent systemic infection within 2 weeks prior to Randomization or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization
Data sourced from ClinicalTrials.gov (NCT03315130). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.