Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

Myasthenia Gravis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05218096
• Other study ID #: ALXN2050-gMG-201
• Status: Terminated
• Phase: Phase 2
• Start date: April 27, 2022
• Est. completion date: April 3, 2024

Study information

• Verified date: May 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 70
• Est. completion date: April 3, 2024
• Est. primary completion date: November 30, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 130 Years

Eligibility

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.

Related Conditions & MeSH terms

• Generalized Myasthenia Gravis
• Muscle Weakness
• Myasthenia Gravis

Intervention

Drug:
• ALXN2050
Oral tablet.
• Placebo
Oral tablet.

Locations

Country	Name	City	State
Canada	Research Site	Edmonton	Alberta
Canada	Research Site	London	Ontario
Canada	Research Site	Toronto	Ontario
Germany	Research Site	Berlin
Germany	Research Site	Bochum
Germany	Research Site	Düsseldorf
Germany	Research Site	Hamburg
Germany	Research Site	Leipzig
Italy	Research Site	Bergamo
Italy	Research Site	Milano
Italy	Research Site	Napoli
Italy	Research Site	Pisa
Italy	Research Site	Roma
Italy	Research Site	Rome
Italy	Research Site	Udine
Korea, Republic of	Research Site	Daegu
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Yangsan-si
Serbia	Research Site	Belgrade
Serbia	Research Site	Nis
Spain	Research Site	Barcelona
Spain	Research Site	Madrid
Spain	Research Site	Malaga
Spain	Research Site	Murcia
Spain	Research Site	Sevilla
Taiwan	Research Site	Hualien City
Taiwan	Research Site	Taipei
Taiwan	Research Site	Taoyuan City
United States	Research Site	Aurora	Colorado
United States	Research Site	Boca Raton	Florida
United States	Research Site	Chapel Hill	North Carolina
United States	Research Site	Charlotte	North Carolina
United States	Research Site	Houston	Texas
United States	Research Site	Lexington	Kentucky
United States	Research Site	Milwaukee	Wisconsin
United States	Research Site	Nashville	Tennessee
United States	Research Site	New Hyde Park	New York
United States	Research Site	Philadelphia	Pennsylvania
United States	Research Site	Phoenix	Arizona
United States	Research Site	Port Charlotte	Florida
United States	Research Site	Portland	Oregon
United States	Research Site	San Antonio	Texas
United States	Research Site	Springfield	Oregon
United States	Research Site	Tampa	Florida
United States	Research Site	West Chester	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Italy,  Korea, Republic of,  Serbia,  Spain,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8		Baseline, Week 8
Secondary	Proportion Of Participants Meeting Various Point Improvement In The QMG Total Score At Week 8		Baseline, Week 8
Secondary	Proportion Of Participants Meeting Various Point Improvement 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
Secondary	Change From Baseline In MG-ADL Total Score At Week 8		Baseline, Week 8
Secondary	Proportion Of Participants Meeting Various Point Improvement 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
Secondary	Proportion Of Participants Meeting Various Point Improvement In The MG-ADL Total Score At Week 8		Baseline, Week 8
Secondary	Change From Baseline In Quality Of Life In Neurological Disorders Fatigue Questionnaire (Neuro-QoL) Fatigue Score At Week 8		Baseline, Week 8
Secondary	Maximum Peak Plasma Concentration (Cmax) Of ALXN2050 Over Time		Baseline through Week 8
Secondary	Pre-dose Concentration (Ctrough) Of ALXN2050 Over Time		Baseline through Week 8
Secondary	Absolute Values And Change From Baseline In Plasma Concentration Of Bb Fragment Of Complement Factor B At Week 8		Baseline, Week 8
Secondary	Absolute Values And Change From Baseline In Serum Alternative Pathway (AP) Activity At Week 8 As Measured By Wieslab Assay		Baseline, Week 8
Secondary	Plasma Factor D Concentration Over Time		Baseline through Week 8
Secondary	Serum Complement Component 3 Concentration Over Time		Baseline through Week 8
Secondary	Serum Classical Pathway Activity Over Time As Measured By CH50		Baseline through Week 8