Generalized Myasthenia Gravis
Conditions
Keywords
Generalized Myasthenia Gravis
Brief summary
This study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics (PD), immunogenicity, and efficacy of subcutaneous (SC) ALXN1830 in adults with generalized myasthenia gravis (gMG).
Detailed description
Participants have the option to enroll in an Open-label Extension (OLE) Period to receive ALXN1830 up to 58 weeks (including follow-up period).
Interventions
DRUGALXN1830
Administered as an SC infusion.
OTHERPlacebo
Administered as an SC infusion.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Diagnosis of myasthenia gravis. * Positive serologic test for anti-acetylcholine receptor antibodies. * Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at Screening. * MG-ADL profile must be ≥ 5. * Participants receiving stable treatment with azathioprine; other immunosuppressive therapies. * Total IgG level at Screening ≥ 600 milligrams/deciliter. Key
Exclusion criteria
* History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to Screening. * Any untreated thymic malignancy, carcinoma, or thymoma. * Intravenous immunoglobulin within the 6 weeks, and/or use of plasmapheresis/plasma exchange prior to randomization (Day 1). * Use of rituximab within the 3 months (90 days) prior to Screening. * Participants who have received previous treatment with any biological agent or other anti-neonatal fragment crystallizable receptor therapy within 5 half-lives or 90 days after last dose (whichever is longer). * Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the participant's full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24 | Up to Week 24 |
| AEs And SAEs Up To Week 82 | Up to Week 82 (OLE) |
| Change From Baseline In Serum Total Immunoglobulin G (IgG) | Up to Week 24 |
Secondary
| Measure | Time frame |
|---|---|
| Number Of Participants With At Least A 3-point Improvement In The QMG Score Over 4 Consecutive Weeks | Up to Week 8 |
| Change From Baseline In Neurological Disorders Fatigue Questionnaire (Neuro-Qol) Fatigue Score | Up to Week 24 |
| Serum Trough Concentrations Of ALXN1830 | Up to Week 24 |
| Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score | Up to Week 24 |
| Incidence Of Anti-drug Antibodies (ADA) And Neutralizing Antibodies (Nab) Against ALXN1830 | Up to Week 24 |
| Titers Of ADA And Nab Against ALXN1830 | Up to Week 24 |
| Change From Baseline In IgG Subtypes | Up to Week 24 |
| Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score | Up to Week 24 |
| Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks | Up to Week 24 |
Countries
United States
Outcome results
None listed