Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

November 7, 2023 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

Active, not recruiting

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 (Estimated)

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 Contact

Study Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 1Z1
        • Clinical Trial Site
    • Ontario
      • London, Ontario, Canada, N6A 5A5
        • Clinical Trial Site
      • Toronto, Ontario, Canada, M5G 2C4
        • Clinical Trial Site
  • Germany
      • Berlin, Germany, 10117
        • Clinical Trial Site
    • Niedersachsen
      • Hamburg, Niedersachsen, Germany, 20246
        • Clinical Trial Site
    • Nordrhein-Westfalen
      • Bochum, Nordrhein-Westfalen, Germany, 44791
        • Clinical Trial Site
      • Düsseldorf, Nordrhein-Westfalen, Germany, 40225
        • Clinical Trial Site
      • Münster, Nordrhein-Westfalen, Germany, 48149
        • Clinical Trial Site
    • Saxony
      • Leipzig, Saxony, Germany, 04103
        • Clinical Trial Site
  • Italy
      • Bergamo, Italy, 24127
        • Clinical Trial Site
      • Pisa, Italy, 56126
        • Clinical Trial Site
      • Udine, Italy, 33100
        • Clinical Trial Site
    • Milano
      • Milan, Milano, Italy, 20133
        • Clinical Trial Site
    • Napoli
      • Naples, Napoli, Italy, 80131
        • Clinical Trial Site
    • Roma
      • Rome, Roma, Italy, 00168
        • Clinical Trial Site
      • Rome, Roma, Italy, 00189
        • Clinical Trial Site
  • Korea, Republic of
      • Seoul, Korea, Republic of, 03080
        • Clinical Trial Site
      • Seoul, Korea, Republic of, 06351
        • Clinical Trial Site
    • Gyeongsangbuk-do
      • Daegu, Gyeongsangbuk-do, Korea, Republic of, 41404
        • Clinical Trial Site
    • Gyeongsangnam-do
      • Yangsan, Gyeongsangnam-do, Korea, Republic of, 50612
        • Clinical Trial Site
  • Serbia
      • Belgrade, Serbia, 11000
        • Clinical Trial Site
    • Nišava District
      • Nis, Nišava District, Serbia, 18000
        • Clinical Trial Site
  • Spain
      • Barcelona, Spain, 08035
        • Clinical Trial Site
      • Barcelona, Spain, 08036
        • Clinical Trial Site
      • Madrid, Spain, 28040
        • Clinical Trial Site
      • Madrid, Spain, 28046
        • Clinical Trial Site
      • Malaga, Spain, 29010
        • Clinical Trial Site
      • Murcia, Spain, 30120
        • Clinical Trial Site
      • Sevilla, Spain, 41009
        • Clinical Trial Site
    • Biscay
      • Barakaldo, Biscay, Spain, 48903
        • Clinical Trial Site
  • Taiwan
      • Hualien City, Taiwan, 970
        • Clinical Trial Site
      • Taipei City, Taiwan, 111
        • Clinical Trial Site
      • Taoyuan City, Taiwan, 333
        • Clinical Trial Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85028
        • Clinical Trial Site
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Clinical Trial Site
    • California
      • Palo Alto, California, United States, 94304
        • Clinical Trial Site
      • San Diego, California, United States, 92037
        • Clinical Trial Site
      • San Francisco, California, United States, 94109-6920
        • Clinical Trial Site
    • Colorado
      • Aurora, Colorado, United States, 80045-2545
        • Clinical Trial Site
    • Florida
      • Boca Raton, Florida, United States, 33487-5712
        • Clinical Trial Site
      • Maitland, Florida, United States, 32751-4749
        • Clinical Trial Site
      • Port Charlotte, Florida, United States, 33952-9204
        • Clinical Trial Site
      • Tampa, Florida, United States, 33612
        • Clinical Trial Site
    • Kentucky
      • Lexington, Kentucky, United States, 40536-7001
        • Clinical Trial Site
    • Missouri
      • Saint Louis, Missouri, United States, 63110-1010
        • Clinical Trial Site
    • New Mexico
      • Albuquerque, New Mexico, United States, 87106
        • Clinical Trial Site
    • New York
      • New Hyde Park, New York, United States, 11042-2062
        • Clinical Trial Site
      • New York, New York, United States, 10075-1850
        • Clinical Trial Site
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • Clinical Trial Site
      • Charlotte, North Carolina, United States, 28207-1885
        • Clinical Trial Site
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • Clinical Trial Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Clinical Trial Site
      • Springfield, Oregon, United States, 97477-1347
        • Clinical Trial Site
    • Pennsylvania
      • Allentown, Pennsylvania, United States, 18103
        • Clinical Trial Site
      • Philadelphia, Pennsylvania, United States, 19104
        • Clinical Trial Site
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Clinical Trial Site
    • Texas
      • Houston, Texas, United States, 77030
        • Clinical Trial Site
      • Houston, Texas, United States, 77030-2340
        • Clinical Trial Site
      • San Antonio, Texas, United States, 78229-3931
        • Clinical Trial Site
    • Wisconsin
      • Greenfield, Wisconsin, United States, 53228-1321
        • Clinical Trial 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

Key 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.

Key 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: ALXN2050
Oral tablet.
Other Names:
  • ACH-0145228 (formerly)
Drug: Placebo
Oral tablet.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Proportion 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
Baseline through Week 8

Secondary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8
Time Frame: Baseline, Week 8
Baseline, Week 8
Proportion Of Participants Meeting Prespecified Threshold In The QMG Total Score At Week 8
Time Frame: Week 8
Week 8
Proportion Of Participants Meeting Prespecified Threshold In The QMG Total Score In Any 4 Consecutive Weeks During The First 8 Weeks And Who Did Not Receive Rescue Therapy
Time Frame: Baseline through Week 8
Baseline through Week 8
Change From Baseline In MG-ADL Total Score At Week 8
Time Frame: Baseline, Week 8
Baseline, Week 8
Proportion Of Participants Meeting Prespecified Threshold In The MG-ADL Total Score In Any 4 Consecutive Weeks During The First 8 Weeks And Who Did Not Receive Rescue Therapy
Time Frame: Baseline through Week 8
Baseline through Week 8
Proportion Of Participants Meeting Prespecified Threshold In The MG-ADL Total Score At Week 8
Time Frame: Week 8
Week 8
Change From Baseline In Quality Of Life In Neurological Disorders Fatigue Questionnaire (Neuro-QoL) Fatigue Score At Week 8
Time Frame: Baseline, Week 8
Baseline, Week 8
Maximum Peak Plasma Concentration (Cmax) Of ALXN2050 Over Time
Time Frame: Baseline through Week 8
Baseline through Week 8
Pre-dose Concentration (Ctrough) Of ALXN2050 Over Time
Time Frame: Baseline through Week 8
Baseline through Week 8
Absolute Values And Change From Baseline In Plasma Concentration Of Bb Fragment Of Complement Factor B At Week 8
Time Frame: Baseline, Week 8
Baseline, Week 8
Absolute Values And Change From Baseline In Serum Alternative Pathway (AP) Activity At Week 8 As Measured By Wieslab Assay
Time Frame: Baseline, Week 8
Baseline, Week 8
Plasma Factor D Concentration Over Time
Time Frame: Baseline through Week 8
Baseline through Week 8
Serum Complement Component 3 Concentration Over Time
Time Frame: Baseline through Week 8
Baseline through Week 8
Serum Classical Pathway Activity Over Time As Measured By CH50
Time Frame: Baseline through Week 8
Baseline through Week 8

Collaborators and Investigators

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

Sponsor

Alexion Pharmaceuticals, Inc.

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)

December 30, 2021

Primary Completion (Estimated)

November 30, 2023

Study Completion (Estimated)

December 30, 2025

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)

November 8, 2023

Last Update Submitted That Met QC Criteria

November 7, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALXN2050-MG-201

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.

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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