Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis

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

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).

Study Overview

• Status: Withdrawn
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Drug: ALXN1830; Other: Placebo

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).

• Study Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85028
      • Clinical Study Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ALXN1830 Dosing Arm 1; Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.	Drug: ALXN1830; Administered as an SC infusion.
Experimental: ALXN1830 Dosing Arm 2; Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks.	Drug: ALXN1830; Administered as an SC infusion.
Experimental: ALXN1830 Dosing Arm 3; Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks.	Drug: ALXN1830; Administered as an SC infusion.; Other: Placebo; Administered as an SC infusion.

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome Measure	Time Frame
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
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
Serum Trough Concentrations Of ALXN1830	Up to Week 24
Change From Baseline In IgG Subtypes	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

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Anticipated): January 1, 2022
• Primary Completion (Anticipated): January 31, 2023
• Study Completion (Anticipated): January 31, 2024

Study Registration Dates

• First Submitted: June 21, 2021
• First Submitted That Met QC Criteria: July 20, 2021
• First Posted (Actual): July 29, 2021

Study Record Updates

• Last Update Posted (Actual): February 11, 2022
• Last Update Submitted That Met QC Criteria: February 2, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Keywords: Generalized Myasthenia Gravis
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis
• Other Study ID Numbers: ALXN1830-MG-201

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No