Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

January 6, 2025 updated by: Alexion Pharmaceuticals, Inc.

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Multicenter Study to Evaluate the Efficacy and Safety of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

This study will evaluate the efficacy and safety of ALXN2050 (120 milligrams [mg], 180 mg) in participants with generalized myasthenia gravis (gMG).

Safety will be monitored throughout the study.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

The study consists of a blinded 8-week Primary Evaluation Period (PEP) and a blinded 26-week Extended Treatment Period (ETP). After completion of 34 weeks of treatment, participants will enter an Open-label Extension (OLE) Period for up to 1.5 years.

Study Type

Interventional

Enrollment (Actual)

70

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2R7
        • Research Site
    • Ontario
      • London, Ontario, Canada, N6A 4L6
        • Research Site
      • Toronto, Ontario, Canada, M5G 2C4
        • Research Site
  • Germany
      • Berlin, Germany, 10117
        • Research Site
      • Bochum, Germany, 44791
        • Research Site
      • Düsseldorf, Germany, 40225
        • Research Site
      • Hamburg, Germany, 20246
        • Research Site
      • Leipzig, Germany, 04103
        • Research Site
  • Italy
      • Bergamo, Italy, 24127
        • Research Site
      • Milano, Italy, 20133
        • Research Site
      • Napoli, Italy, 80131
        • Research Site
      • Pisa, Italy, 56100
        • Research Site
      • Roma, Italy, 00168
        • Research Site
      • Rome, Italy, 00189
        • Research Site
      • Udine, Italy, 33100
        • Research Site
  • Korea, Republic of
      • Daegu, Korea, Republic of, 41404
        • Research Site
      • Seoul, Korea, Republic of, 03080
        • Research Site
      • Seoul, Korea, Republic of, 06351
        • Research Site
      • Yangsan-si, Korea, Republic of, 50612
        • Research Site
  • Serbia
      • Belgrade, Serbia, 11000
        • Research Site
      • Nis, Serbia, 18000
        • Research Site
  • Spain
      • Barcelona, Spain, 08036
        • Research Site
      • Madrid, Spain, 28046
        • Research Site
      • Malaga, Spain, 29010
        • Research Site
      • Murcia, Spain, 30120
        • Research Site
      • Sevilla, Spain, 41009
        • Research Site
  • Taiwan
      • Hualien City, Taiwan, 97002
        • Research Site
      • Taipei, Taiwan, 11101
        • Research Site
      • Taoyuan City, Taiwan
        • Research Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85028
        • Research Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Research Site
    • Florida
      • Boca Raton, Florida, United States, 33487
        • Research Site
      • Port Charlotte, Florida, United States, 33952
        • Research Site
      • Tampa, Florida, United States, 33612
        • Research Site
    • Kentucky
      • Lexington, Kentucky, United States, 40508
        • Research Site
    • New York
      • New Hyde Park, New York, United States, 11042
        • Research Site
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • Research Site
      • Charlotte, North Carolina, United States, 28207
        • Research Site
    • Ohio
      • West Chester, Ohio, United States, 45069
        • Research Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Research Site
      • Springfield, Oregon, United States, 97477
        • Research Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Research Site
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Research Site
    • Texas
      • Houston, Texas, United States, 77030
        • Research Site
      • San Antonio, Texas, United States, 78229
        • Research Site
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53228
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosed with MG at least 3 months (90 days) prior to the date of the Screening Visit. Confirmation of MG must be made via the following:

    1. Positive serologic test for anti AChR antibodies at the Screening Visit, and
    2. Abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation, or
    3. Positive response to an AChEI test (eg, edrophonium chloride test), or
    4. Improvement of signs or symptoms related to MG during treatment with an oral AChEI, as determined by the treating physician
  • Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at the Screening Visit.
  • MG-ADL total score must be ≥ 5 (with at least 50% of the score attributed to non-ocular elements) at the Screening Visit and at randomization (Day 1).
  • Participants receiving treatment with protocol-specified immunosuppressive therapies, corticosteroids, or acetylcholinesterase inhibitors must have been receiving treatment and on a stable dose prior to the date of the Screening Visit, with no changes to the regimen expected during screening, the PEP, and/or the ETP.

Exclusion Criteria:

  • Estimated glomerular filtration rate ≤ 30 milliliters/minute/1.73 squared meters during Screening calculated by Chronic Kidney Disease Epidemiology Collaboration.
  • History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to the Screening Visit.
  • Any untreated thymic malignancy, carcinoma, or thymoma. Participants with a history of treated thymic malignancy or carcinoma are eligible for enrollment if they meet pre-specified conditions outlined in the protocol.
  • Clinical features consistent with Clinical Deterioration at the time of the Screening Visit or at any time during the Screening Period prior to randomization (Day 1).

  • Use of the following within the time periods specified below:

    1. Intravenous immunoglobulin G or subcutaneous immunoglobulin within the 4 weeks (28 days) prior to the Screening Visit.
    2. Use of tacrolimus or cyclosporine within the 4 weeks (28 days) prior to the Screening Visit.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ALXN2050: 180 mg
Participants will receive ALXN2050.
Drug: ALXN2050
Oral tablet.
Other Names:
  • ACH-0145228 (formerly)
Experimental: ALXN2050: 120 mg
Participants will receive ALXN2050.
Drug: ALXN2050
Oral tablet.
Other Names:
  • ACH-0145228 (formerly)
Placebo Comparator: Placebo
Participants will receive placebo followed by ALXN2050.
Drug: Placebo
Oral tablet.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage Of Participants With a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction Of ≥ 2 Points In Any 4 Consecutive Weeks During The First 8 Weeks And Who Did Not Receive Rescue Therapy
Time Frame: Baseline through Week 8
The MG-ADL profile is an 8-item participant-reported scale that focuses on relevant symptoms and functional performance of ADL in participants with MG. The 8 items of the MG-ADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects of MG. Each response is graded 0 (normal) to 3 (most severe). The MG-ADL total score was calculated as the sum of the scores of the 8 items and ranges from 0 to 24, with higher scores indicating worse function.
Baseline through Week 8

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8
Time Frame: Baseline, Week 8
The QMG Score for Disease Severity is an objective evaluation of therapy for MG and is based on quantitative testing of sentinel muscle groups. The QMG instrument consists of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The QMG total score was calculated as the sum of the scores of the 13 items and ranges from 0 to 39, with higher scores indicating more severe disease. Baseline score at each timepoint as the response variable, treatment group, study visit, and treatment-by-study visit interaction as fixed categorical effects, and baseline QMG total score as a covariate were used to calculate the least square (LS) mean and the standard error.
Baseline, Week 8
Change From Baseline In MG-ADL Total Score At Week 8
Time Frame: Baseline, Week 8
The MG-ADL profile is an 8-item participant-reported scale that focuses on relevant symptoms and functional performance of ADL in participants with MG. The 8 items of the MG-ADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects of MG. Each response is graded 0 (normal) to 3 (most severe). The MG-ADL total score was calculated as the sum of the scores of the 8 items and ranges from 0 to 24, with higher scores indicating worse function. Baseline score at each timepoint as the response variable, treatment group, study visit, and treatment-by-study visit interaction as fixed categorical effects, and baseline MG-ADL total score as a covariate were used to calculate the LS mean and the standard error.
Baseline, Week 8
Change From Baseline In Quality Of Life In Neurological Disorders (Neuro-QoL) Fatigue Score At Week 8
Time Frame: Baseline, Week 8
The Neuro-QoL Fatigue questionnaire is a reliable and validated brief 19-item survey of fatigue, completed by the participant. Each item is scored on a scale of 1-5, with 1 indicating "never" and 5 indicating "sometimes". The Neuro-QoL Fatigue score was calculated as the sum of the scores of the 19 items and ranges from 19-95, with higher scores indicating greater fatigue and greater impact of MG on activities. Baseline score at each timepoint as the response variable, treatment group, study visit, and treatment-by-study visit interaction as fixed categorical effects, and baseline Neuro-QoL Fatigue score as a covariate were used to calculate the LS mean and the standard error.
Baseline, Week 8

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 27, 2022

Primary Completion (Actual)

November 30, 2023

Study Completion (Actual)

April 3, 2024

Study Registration Dates

First Submitted

December 16, 2021

First Submitted That Met QC Criteria

January 28, 2022

First Posted (Actual)

February 1, 2022

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

January 6, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALXN2050-MG-201
  • 2021-001229-26 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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