Generalized Myasthenia GravisPhase 2NCT05218096

Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

A Phase 2 study testing ALXN2050 in people with Generalized Myasthenia Gravis and Myasthenia Gravis, run by Alexion Pharmaceuticals, Inc. at 46 sites in 8 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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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. Read more about this study

Who is it for?: Ages 18 to 130 years
Conditions the study is for: Generalized Myasthenia Gravis, Myasthenia Gravis
What is being tested?: ALXN2050
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 70 participants
Where: 46 sitesin 8 countries
Study dates: 27 Apr 2022to 3 Apr 2024
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 130 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

46 sites in 8 countries.

Country

City, postcode or hospital

46 sites in 8 countries

United States17 sites

Research Site
Aurora, Colorado, 80045
Research Site
Boca Raton, Florida, 33487
Research Site
Chapel Hill, North Carolina, 27514
Research Site
Charlotte, North Carolina, 28207
Research Site
Houston, Texas, 77030
Research Site
Lexington, Kentucky, 40508
Research Site
Milwaukee, Wisconsin, 53228
Research Site
Nashville, Tennessee, 37232
Research Site
New Hyde Park, New York, 11042
Research Site
Philadelphia, Pennsylvania, 19104
Research Site
Phoenix, Arizona, 85028
Research Site
Port Charlotte, Florida, 33952
Research Site
Portland, Oregon, 97239
Research Site
San Antonio, Texas, 78229
Research Site
Springfield, Oregon, 97477
Research Site
Tampa, Florida, 33612
Research Site
West Chester, Ohio, 45069

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Italy7 sites

Research Site
Bergamo, 24127
Research Site
Milan, 20133
Research Site
Naples, 80131
Research Site
Pisa, 56100
Research Site
Roma, 00168
Research Site
Rome, 00189
Research Site
Udine, 33100

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Germany5 sites

Research Site
Berlin, 10117
Research Site
Bochum, 44791
Research Site
Düsseldorf, 40225
Research Site
Hamburg, 20246
Research Site
Leipzig, 04103

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Spain5 sites

Research Site
Barcelona, 08036
Research Site
Madrid, 28046
Research Site
Málaga, 29010
Research Site
Murcia, 30120
Research Site
Seville, 41009

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South Korea4 sites

Research Site
Daegu, 41404
Research Site
Seoul, 03080
Research Site
Seoul, 06351
Research Site
Yangsan, 50612

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Canada3 sites

Research Site
Edmonton, Alberta, T6G 2R7
Research Site
London, Ontario, N6A 4L6
Research Site
Toronto, Ontario, M5G 2C4

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Taiwan3 sites

Research Site
Hualien City, 97002
Research Site
Taipei, 11101
Research Site
Taoyuan City

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Serbia2 sites

Research Site
Belgrade, 11000
Research Site
Niš, 18000

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About this study

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

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.

Read the 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.

Conditions: Generalized Myasthenia Gravis, Myasthenia Gravis
Being tested: ALXN2050, Placebo
Keywords: ALXN2050, gMG
Medical subject headings: Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ALXN2050Drug
Also called ACH-0145228 (formerly)
Oral tablet.
PlaceboDrug
Oral tablet.

Study groups in NCT05218096
Study group	Role	What they receive
ALXN2050: 180 mg	Experimental	Participants will receive ALXN2050. Receives: Drug: ALXN2050
ALXN2050: 120 mg	Experimental	Participants will receive ALXN2050. Receives: Drug: ALXN2050
Placebo	Placebo comparator	Participants will receive placebo followed by ALXN2050. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline through Week 8

Secondary outcomes (3)

Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8
The QMG Score for Disease Severity is an objective evaluation of therapy for MG and is based on quantitative testing of sentinel muscle groups. The QMG instrument consists of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The QMG total score was calculated as the sum of the scores of the 13 items and ranges from 0 to 39, with higher scores indicating more severe disease. Baseline score at each timepoint as the response variable, treatment group, study visit, and treatment-by-study visit interaction as fixed categorical effects, and baseline QMG total score as a covariate were used to calculate the least square (LS) mean and the standard error.
Measured: Baseline, Week 8
Change From Baseline In MG-ADL Total Score At Week 8
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 score at each timepoint as the response variable, treatment group, study visit, and treatment-by-study visit interaction as fixed categorical effects, and baseline MG-ADL total score as a covariate were used to calculate the LS mean and the standard error.
Measured: Baseline, Week 8
Change From Baseline In Quality Of Life In Neurological Disorders (Neuro-QoL) Fatigue Score At Week 8
The Neuro-QoL Fatigue questionnaire is a reliable and validated brief 19-item survey of fatigue, completed by the participant. Each item is scored on a scale of 1-5, with 1 indicating "never" and 5 indicating "sometimes". The Neuro-QoL Fatigue score was calculated as the sum of the scores of the 19 items and ranges from 19-95, with higher scores indicating greater fatigue and greater impact of MG on activities. Baseline score at each timepoint as the response variable, treatment group, study visit, and treatment-by-study visit interaction as fixed categorical effects, and baseline Neuro-QoL Fatigue score as a covariate were used to calculate the LS mean and the standard error.
Measured: Baseline, Week 8

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

27 Apr 20223 Apr 2024

First posted1 Feb 2022
Study start27 Apr 2022
Primary completion30 Nov 2023
Study completion3 Apr 2024

Why it stopped: Sponsor decision

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Generalized Myasthenia GravisStudies of the same condition, open and under way.
More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 70 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05218096
Sponsor's protocol ID: ALXN2050-MG-201
Other IDs: 2021-001229-26
Status last verified: Jan 2025
First submitted: 16 Dec 2021
Last updated: 9 Jan 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Publications

Sacca F, Gialdini G, Howard JF Jr, Vu TH, Meisel A, Peric S, Lunemann JD, Sgarzi M, Shaibani A, Bril V, Yeh JH, Reyes-Garrido V, Liao S, Metais C, van der Valk RJP; ALXN2050-MG-201 Investigators. Efficacy and Safety of Vemircopan in Generalized Myasthenia Gravis: A Randomized Clinical Trial. JAMA Neurol. 2026 Jun 1;83(6):544-553. doi: 10.1001/jamaneurol.2026.0902.
PubMed 42043793

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 9 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT05218096
Acronym / sponsor ID: ALXN2050-MG-201
Conditions: Generalized Myasthenia Gravis, Myasthenia Gravis
Keywords: ALXN2050, gMG
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Sponsor decision
Enrolment: 70 participants (actual)
Lead sponsor: Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
Study start: 27 Apr 2022 (actual)
Primary completion: 30 Nov 2023 (actual)
Study completion: 3 Apr 2024 (actual)
First posted: 1 Feb 2022 (actual)
Results: Posted 9 Jan 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 3

Arm	Type	Receives
ALXN2050: 180 mg	Experimental	Drug: ALXN2050Participants will receive ALXN2050.
ALXN2050: 120 mg	Experimental	Drug: ALXN2050Participants will receive ALXN2050.
Placebo	Placebo comparator	Drug: PlaceboParticipants will receive placebo followed by ALXN2050.

Who may qualify

Ages: 18 to 130 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

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:
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 (7 listed)

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:

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ALXN2050Also: ACH-0145228 (formerly)Oral tablet.	Drug	ALXN2050: 120 mg, ALXN2050: 180 mg
PlaceboOral tablet.	Drug	Placebo

Primary outcome

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
Time frame: Baseline through Week 8
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.

3 secondary outcomes

Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8
Time frame: Baseline, Week 8
Change From Baseline In MG-ADL Total Score At Week 8
Time frame: Baseline, Week 8
Change From Baseline In Quality Of Life In Neurological Disorders (Neuro-QoL) Fatigue Score At Week 8
Time frame: Baseline, Week 8

Results and safety

Results posted to the registry on 9 Jan 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Group 1: ALXN2050 180 mg BID	28	27
Group 2: ALXN2050 120 mg BID	14	14
Group 3: Placebo	28	26
Group 3a: Placebo/ALXN2050 180 mg BID	0	0
Group 3b: Placebo/ALXN2050 120 mg BID	0	0

Primary outcome: 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

Time frame: Baseline through Week 8. Number (90% Confidence Interval). Unit: percentage of participants

Measure	Group 1: ALXN2050 180 mg BID n = 16	Group 2: ALXN2050 120 mg BID n = 8	Group 3: Placebo n = 18
Value	57.1 (40.0 to 73.1)	57.1 (32.5 to 79.4)	64.3 (47.0 to 79.2)

Group 1: ALXN2050 180 mg BID vs Group 3: Placebo: Difference: -7.1 (90% CI -28.8 to 15.0), p = 0.6797, Barnard's Unconditional Exact Test
Group 2: ALXN2050 120 mg BID vs Group 3: Placebo: Difference: -7.1 (90% CI -34.8 to 19.3), p = 0.7341, Barnard's Unconditional Exact Test

Population analysed: Full Analysis Set: all participants who received at least 1 dose of study intervention. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Baseline (Day 1) up to 30 days after last dose of study intervention (approximately 2 years).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Group 1: ALXN2050 180 mg BID (Primary Evaluation)	1 of 28 (3.6%)	17 of 28 (60.7%)	0 of 28
Group 2: ALXN2050 120 mg BID (Primary Evaluation)	1 of 14 (7.1%)	7 of 14 (50%)	0 of 14
Group 3: Placebo (Primary Evaluation)	3 of 28 (10.7%)	15 of 28 (53.6%)	0 of 28
Group 1: ALXN2050 180 mg BID (Extended Treatment & Open-label Extension)	4 of 27 (14.8%)	17 of 27 (63%)	0 of 27
Group 2: ALXN2050 120 mg BID (Extended Treatment & Open-label Extension)	4 of 14 (28.6%)	7 of 14 (50%)	0 of 14
Group 3a: Placebo/ALXN2050 180 mg BID (Extended Treatment & Open-label Extension)	3 of 13 (23.1%)	9 of 13 (69.2%)	1 of 13
Group 3b: Placebo/ALXN2050 120 mg BID (Extended Treatment & Open-label Extension)	2 of 13 (15.4%)	10 of 13 (76.9%)	0 of 13

Limitations and caveats (from the sponsor): This study did not meet its primary efficacy endpoint and was early terminated by the Sponsor.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 42043793Sacca F, Gialdini G, Howard JF Jr, Vu TH, Meisel A, Peric S, Lunemann JD, Sgarzi M, Shaibani A, Bril V, Yeh JH, Reyes-Garrido V, Liao S, Metais C, van der Valk RJP; ALXN2050-MG-201 Investigators. Efficacy and Safety of Vemircopan in Generalized Myasthenia Gravis: A Randomized Clinical Trial. JAMA Neurol. 2026 Jun 1;83(6):544-553. doi: 10.1001/jamaneurol.2026.0902.
Related InfoLink provided by the sponsor.
Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Related trials for Generalized Myasthenia Gravis

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

46 sites in 8 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ALXN2050 in people with Generalized Myasthenia Gravis and Myasthenia Gravis, run by Alexion Pharmaceuticals, Inc. at 46 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 9 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 46 sites in United States, Italy, Germany, Spain, South Korea and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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 (time frame: Baseline through Week 8). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05218096, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jan 2025.