Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis
- Registration Number
- NCT04982289
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- 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).
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ALXN1830 Dosing Arm 3 Placebo Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks. ALXN1830 Dosing Arm 2 ALXN1830 Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks. ALXN1830 Dosing Arm 1 ALXN1830 Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks. ALXN1830 Dosing Arm 3 ALXN1830 Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks.
- Primary Outcome Measures
Name Time Method AEs And SAEs Up To Week 82 Up to Week 82 (OLE) Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24 Up to Week 24 Change From Baseline In Serum Total Immunoglobulin G (IgG) Up to Week 24
- Secondary Outcome Measures
Name Time Method Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks Up to Week 24 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 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 Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score Up to Week 24 Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score Up to Week 24 Serum Trough Concentrations Of ALXN1830 Up to Week 24 Change From Baseline In IgG Subtypes Up to Week 24
Trial Locations
- Locations (1)
Clinical Study Site
🇺🇸Phoenix, Arizona, United States