Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

NCT05218096 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: ALXN2050-MG-2012021-001229-26
• Study Type: interventional
• Target: 70
• Locations: 8 countries
• Sites: 46

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.

Conditions

Generalized Myasthenia Gravis; Myasthenia Gravis

Keywords

ALXN2050; gMG

Outcome Measures

Primary Outcomes (1)

• 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

  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 Outcomes (3)

• Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8

  Baseline, Week 8

• Change From Baseline In MG-ADL Total Score At Week 8

  Baseline, Week 8

• Change From Baseline In Quality Of Life In Neurological Disorders (Neuro-QoL) Fatigue Score At Week 8

  Baseline, Week 8

Study Arms (3)

ALXN2050: 180 mg

EXPERIMENTAL

Participants will receive ALXN2050.

Drug: ALXN2050

ALXN2050: 120 mg

EXPERIMENTAL

Participants will receive ALXN2050.

Drug: ALXN2050

Placebo

PLACEBO COMPARATOR

Participants will receive placebo followed by ALXN2050.

Drug: Placebo

Interventions

ALXN2050 DRUG

Oral tablet.

Also known as: ACH-0145228 (formerly)

ALXN2050: 120 mg; ALXN2050: 180 mg

Placebo DRUG

Oral tablet.

Placebo

Eligibility Criteria

• Age: 18 Years - 130 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Positive serologic test for anti AChR antibodies at the Screening Visit, and
• Abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation, or
• Positive response to an AChEI test (eg, edrophonium chloride test), or
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (46)

Research Site

Phoenix, Arizona, 85028, United States

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Aurora, Colorado, 80045, United States

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Boca Raton, Florida, 33487, United States

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Port Charlotte, Florida, 33952, United States

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Tampa, Florida, 33612, United States

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Lexington, Kentucky, 40508, United States

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New Hyde Park, New York, 11042, United States

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Chapel Hill, North Carolina, 27514, United States

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Charlotte, North Carolina, 28207, United States

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West Chester, Ohio, 45069, United States

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Portland, Oregon, 97239, United States

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Springfield, Oregon, 97477, United States

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Philadelphia, Pennsylvania, 19104, United States

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Nashville, Tennessee, 37232, United States

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Houston, Texas, 77030, United States

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San Antonio, Texas, 78229, United States

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Milwaukee, Wisconsin, 53228, United States

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Edmonton, Alberta, T6G 2R7, Canada

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London, Ontario, N6A 4L6, Canada

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Toronto, Ontario, M5G 2C4, Canada

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Berlin, 10117, Germany

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Bochum, 44791, Germany

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Düsseldorf, 40225, Germany

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Hamburg, 20246, Germany

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Leipzig, 04103, Germany

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Bergamo, 24127, Italy

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Milan, 20133, Italy

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Napoli, 80131, Italy

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Pisa, 56100, Italy

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Roma, 00168, Italy

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Rome, 00189, Italy

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Udine, 33100, Italy

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Belgrade, 11000, Serbia

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Niš, 18000, Serbia

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Daegu, 41404, South Korea

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Seoul, 03080, South Korea

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Seoul, 06351, South Korea

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Yangsan, 50612, South Korea

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Barcelona, 08036, Spain

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Madrid, 28046, Spain

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Málaga, 29010, Spain

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Murcia, 30120, Spain

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Seville, 41009, Spain

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Hualien City, 97002, Taiwan

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Taipei, 11101, Taiwan

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Taoyuan, Taiwan

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Related Links

• Related Info

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

rhoA GTP-Binding Protein

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

rho GTP-Binding Proteins; Monomeric GTP-Binding Proteins; GTP-Binding Proteins; GTP Phosphohydrolases; Acid Anhydride Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Carrier Proteins; Proteins; Amino Acids, Peptides, and Proteins; Intracellular Signaling Peptides and Proteins

Limitations and Caveats

This study did not meet its primary efficacy endpoint and was early terminated by the Sponsor.

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

+1.855.752.2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Masking of treatment allocation will be observed until at least Week 34.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 16, 2021
• First Posted: February 1, 2022
• Study Start: April 27, 2022
• Primary Completion: November 30, 2023
• Study Completion: April 3, 2024
• Last Updated: January 9, 2025
• Results First Posted: January 9, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

TW (3); IT (7); DE (5); SE (2); KR (4); CA (3); US (17); ES (5)