Clinical trial NCT04982289: Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis
Official title: A Phase 2a Randomized, Double-blind, Placebo-controlled, Parallel Groups, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALXN1830 Administered Subcutaneously in Adult Patients With Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Generalized Myasthenia Gravis
Keywords: Generalized Myasthenia Gravis
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-07-29
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

Eligibility 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.

Primary outcomes:
measure: Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24
timeFrame: Up to Week 24
measure: AEs And SAEs Up To Week 82
timeFrame: Up to Week 82 (OLE)
measure: Change From Baseline In Serum Total Immunoglobulin G (IgG)
timeFrame: Up to Week 24
Secondary outcomes:
measure: Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score
timeFrame: Up to Week 24
measure: Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score
timeFrame: Up to Week 24
measure: Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks
timeFrame: Up to Week 24
measure: Number Of Participants With At Least A 3-point Improvement In The QMG Score Over 4 Consecutive Weeks
timeFrame: Up to Week 8
measure: Change From Baseline In Neurological Disorders Fatigue Questionnaire (Neuro-Qol) Fatigue Score
timeFrame: Up to Week 24
measure: Serum Trough Concentrations Of ALXN1830
timeFrame: Up to Week 24
measure: Change From Baseline In IgG Subtypes
timeFrame: Up to Week 24
measure: Incidence Of Anti-drug Antibodies (ADA) And Neutralizing Antibodies (Nab) Against ALXN1830
timeFrame: Up to Week 24
measure: Titers Of ADA And Nab Against ALXN1830
timeFrame: Up to Week 24
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Clinical Study Site
state: Arizona
Study officials:
References: