Phase 3
N=175
Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Bottom Line
View on ClinicalTrials.gov: NCT03920293 ↗Enrolled (actual)
175
Serious AEs
26.8%
Results posted
May 2022
Primary outcome: Primary: Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26 — -3.1; -1.4 units on a scale — p==0.0009
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Ravulizumab (Biological); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- May 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26 |
-3.1; -1.4 | =0.0009 sig |
| SECONDARY Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26 |
-2.8; -0.8 | — |
| SECONDARY Percentage of Participants With a Quantitative Myasthenia Gravis (QMG) Total Score Reduction of at Least 5 Points At Week 26 |
30.0; 11.3 | — |
| SECONDARY Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26 |
-3.3; -1.6 | — |
| SECONDARY Change From Baseline in Neurological Quality of Life (Neuro-QoL) Fatigue Score at Week 26 |
-7.0; -4.8 | — |
| SECONDARY Percentage of Participants With a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction of at Least 3 Points At Week 26 |
56.7; 34.1 | — |
Summary
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
Eligibility Criteria
Inclusion Criteria
- Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.
- Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.
- MG-ADL profile must be ≥ 6 at screening and randomization (Day 1).
- Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).
Exclusion Criteria
Medical Conditions
- Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 5 years before screening.
- History of thymectomy within the 12 months prior to screening.
- History of N meningitidis infection.
- Use of the following within the time period specified below:
- IV immunoglobulin within 4 weeks of randomization
- Use of plasma exchange within 4 weeks of randomization
- Use of rituximab within 6 months of screening
- Participants who have received previous treatment with complement inhibitors (for example, eculizumab).
Data sourced from ClinicalTrials.gov (NCT03920293). 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.