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Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

Sponsor
Alexion Pharmaceuticals (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05218096
Collaborator
(none)
70
Enrollment
31
Locations
3
Arms
38
Anticipated Duration (Months)
2.3
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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
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

Study Type:
Interventional
Anticipated 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 :
Dec 30, 2021
Anticipated Primary Completion Date :
Feb 28, 2023
Anticipated Study Completion Date :
Feb 28, 2025

Arms and Interventions

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.

    Outcome Measures

    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 [Baseline through Week 8]

    Secondary Outcome Measures

    1. Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8 [Baseline, Week 8]

    2. Proportion Of Participants Meeting Prespecified Threshold In The QMG Total Score At Week 8 [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 [Baseline through Week 8]

    4. Change From Baseline In MG-ADL Total Score At Week 8 [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 [Baseline through Week 8]

    6. Proportion Of Participants Meeting Prespecified Threshold In The MG-ADL Total Score At Week 8 [Week 8]

    7. Change From Baseline In Quality Of Life In Neurological Disorders Fatigue Questionnaire (Neuro-QoL) Fatigue Score At Week 8 [Baseline, Week 8]

    8. Maximum Peak Plasma Concentration (Cmax) Of ALXN2050 Over Time [Baseline through Week 8]

    9. Pre-dose Concentration (Ctrough) Of ALXN2050 Over Time [Baseline through Week 8]

    10. Absolute Values And Change From Baseline In Plasma Concentration Of Bb Fragment Of Complement Factor B At Week 8 [Baseline, Week 8]

    11. Absolute Values And Change From Baseline In Serum Alternative Pathway (AP) Activity At Week 8 As Measured By Wieslab Assay [Baseline, Week 8]

    12. Plasma Factor D Concentration Over Time [Baseline through Week 8]

    13. Serum Complement Component 3 Concentration Over Time [Baseline through Week 8]

    14. Serum Classical Pathway Activity Over Time As Measured By CH50 [Baseline through Week 8]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:

    • Diagnosed with MG at least 3 months (90 days) prior to the date of the Screening Visit.

    • 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

    Locations

    Site City State Country Postal Code
    1 Clinical Trial Site Boca Raton Florida United States 33487-5712
    2 Clinical Trial Site Tampa Florida United States 33612
    3 Clinical Trial Site Louisville Kentucky United States 40202-1675
    4 Clinical Trial Site Saint Louis Missouri United States 63110-1010
    5 Clinical Trial Site Albuquerque New Mexico United States 87106
    6 Clinical Trial Site New Hyde Park New York United States 11042-2062
    7 Clinical Trial Site New Hyde Park New York United States 11042
    8 Clinical Trial Site Chapel Hill North Carolina United States 27514
    9 Clinical Trial Site Springfield Oregon United States 97477-1347
    10 Clinical Trial Site Allentown Pennsylvania United States 18103
    11 Clinical Trial Site Houston Texas United States 77030-1501
    12 Clinical Trial Site Houston Texas United States 77030-2312
    13 Clinical Trial Site Milan Milano Italy 20133
    14 Clinical Trial Site Naples Napoli Italy 80131
    15 Clinical Trial Site Rome Roma Italy 00168
    16 Clinical Trial Site Rome Roma Italy 00189
    17 Clinical Trial Site Udine Italy 33100
    18 Clinical Trial Site Daegu Gyeongsangbuk-do Korea, Republic of 41404
    19 Clinical Trial Site Yangsan Gyeongsangnam-do Korea, Republic of 50612
    20 Clinical Trial Site Seoul Korea, Republic of 03080
    21 Clinical Trial Site Seoul Korea, Republic of 06351
    22 Clinical Trial Site Barakaldo Biscay Spain 48903
    23 Clinical Trial Site Barcelona Spain 08035
    24 Clinical Trial Site Barcelona Spain 08036
    25 Clinical Trial Site Madrid Spain 28040
    26 Clinical Trial Site Madrid Spain 28046
    27 Clinical Trial Site Malaga Spain 29010
    28 Clinical Trial Site Murcia Spain 30120
    29 Clinical Trial Site Sevilla Spain 41009
    30 Clinical Trial Site Taipei City Taiwan 111
    31 Clinical Trial Site Taoyuan City Taiwan 333

    Sponsors and Collaborators

    • Alexion Pharmaceuticals

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Alexion Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT05218096
    Other Study ID Numbers:
    • ALXN2050-MG-201
    First Posted:
    Feb 1, 2022
    Last Update Posted:
    Aug 23, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Alexion Pharmaceuticals
    ALXN2050
    gMG
    Additional relevant MeSH terms:
    Myasthenia Gravis
    Muscle Weakness

    Study Results

    No Results Posted as of Aug 23, 2022