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Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

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ClinicalTrials.gov Identifier: NCT05218096
Recruitment Status : Recruiting
First Posted : February 1, 2022
Last Update Posted : March 20, 2023
Sponsor:
Information provided by (Responsible Party):
Alexion

Study Description
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Brief Summary:

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.


Condition or disease Intervention/treatment Phase
Generalized Myasthenia Gravis Myasthenia Gravis Drug: ALXN2050 Drug: Placebo Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Masking of treatment allocation will be observed until at least Week 34.
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : December 30, 2021
Estimated Primary Completion Date : June 30, 2023
Estimated Study Completion Date : September 26, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: ALXN2050: 180 mg
Participants will receive ALXN2050.
 Drug: ALXN2050
Oral tablet.
Other Name: ACH-0145228 (formerly)
Experimental: ALXN2050: 120 mg
Participants will receive ALXN2050.
 Drug: ALXN2050
Oral tablet.
Other Name: ACH-0145228 (formerly)
Placebo Comparator: Placebo
Participants will receive placebo followed by ALXN2050.
 Drug: ALXN2050
Oral tablet.
Other Name: ACH-0145228 (formerly)

Drug: Placebo
Oral tablet.


Outcome Measures
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Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8 [ Time Frame: Baseline, Week 8 ]
  2. Proportion Of Participants Meeting Prespecified Threshold In The QMG Total Score At Week 8 [ Time Frame: Week 8 ]
  3. 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 ]
  4. Change From Baseline In MG-ADL Total Score At Week 8 [ Time Frame: Baseline, Week 8 ]
  5. 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 ]
  6. Proportion Of Participants Meeting Prespecified Threshold In The MG-ADL Total Score At Week 8 [ Time Frame: Week 8 ]
  7. Change From Baseline In Quality Of Life In Neurological Disorders Fatigue Questionnaire (Neuro-QoL) Fatigue Score At Week 8 [ Time Frame: Baseline, Week 8 ]
  8. Maximum Peak Plasma Concentration (Cmax) Of ALXN2050 Over Time [ Time Frame: Baseline through Week 8 ]
  9. Pre-dose Concentration (Ctrough) Of ALXN2050 Over Time [ Time Frame: Baseline through Week 8 ]
  10. Absolute Values And Change From Baseline In Plasma Concentration Of Bb Fragment Of Complement Factor B At Week 8 [ Time Frame: Baseline, Week 8 ]
  11. 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 ]
  12. Plasma Factor D Concentration Over Time [ Time Frame: Baseline through Week 8 ]
  13. Serum Complement Component 3 Concentration Over Time [ Time Frame: Baseline through Week 8 ]
  14. Serum Classical Pathway Activity Over Time As Measured By CH50 [ Time Frame: Baseline through Week 8 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05218096


Contacts
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Contact: Alexion Pharmaceuticals, Inc. 1-855-752-2356 clinicaltrials@alexion.com

Locations
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Sponsors and Collaborators
Alexion
More Information
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Responsible Party: Alexion
ClinicalTrials.gov Identifier: NCT05218096    
Other Study ID Numbers: ALXN2050-MG-201
First Posted: February 1, 2022    Key Record Dates
Last Update Posted: March 20, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion:
ALXN2050
gMG
Additional relevant MeSH terms:
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Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
 Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases