Clinical trial NCT05218096: Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Generalized Myasthenia Gravis
Myasthenia Gravis
Keywords: ALXN2050
gMG
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2022-02-01
Enrollment: 70
Sex: ALL
Minimum age (years): 18
Maximum age (years): 130
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
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
timeFrame: Baseline through Week 8
Secondary outcomes:
measure: Change From Baseline In Quantitative Myasthenia Gravis (QMG) Total Score At Week 8
timeFrame: Baseline, Week 8
measure: Change From Baseline In MG-ADL Total Score At Week 8
timeFrame: Baseline, Week 8
measure: Change From Baseline In Quality Of Life In Neurological Disorders (Neuro-QoL) Fatigue Score At Week 8
timeFrame: Baseline, Week 8
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Research Site
state: Arizona
city: Aurora
country: United States
facility: Research Site
state: Colorado
city: Boca Raton
country: United States
facility: Research Site
state: Florida
city: Port Charlotte
country: United States
facility: Research Site
state: Florida
city: Tampa
country: United States
facility: Research Site
state: Florida
city: Lexington
country: United States
facility: Research Site
state: Kentucky
city: New Hyde Park
country: United States
facility: Research Site
state: New York
city: Chapel Hill
country: United States
facility: Research Site
state: North Carolina
city: Charlotte
country: United States
facility: Research Site
state: North Carolina
city: West Chester
country: United States
facility: Research Site
state: Ohio
city: Portland
country: United States
facility: Research Site
state: Oregon
city: Springfield
country: United States
facility: Research Site
state: Oregon
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Nashville
country: United States
facility: Research Site
state: Tennessee
city: Houston
country: United States
facility: Research Site
state: Texas
city: San Antonio
country: United States
facility: Research Site
state: Texas
city: Milwaukee
country: United States
facility: Research Site
state: Wisconsin
city: Edmonton
country: Canada
facility: Research Site
state: Alberta
city: London
country: Canada
facility: Research Site
state: Ontario
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Berlin
country: Germany
facility: Research Site
city: Bochum
country: Germany
facility: Research Site
city: Düsseldorf
country: Germany
facility: Research Site
city: Hamburg
country: Germany
facility: Research Site
city: Leipzig
country: Germany
facility: Research Site
city: Bergamo
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Napoli
country: Italy
facility: Research Site
city: Pisa
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Rome
country: Italy
facility: Research Site
city: Udine
country: Italy
facility: Research Site
city: Daegu
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Yangsan-si
country: Korea, Republic of
facility: Research Site
city: Belgrade
country: Serbia
facility: Research Site
city: Nis
country: Serbia
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
city: Madrid
country: Spain
facility: Research Site
city: Malaga
country: Spain
facility: Research Site
city: Murcia
country: Spain
facility: Research Site
city: Sevilla
country: Spain
facility: Research Site
city: Hualien City
country: Taiwan
facility: Research Site
city: Taipei
country: Taiwan
facility: Research Site
city: Taoyuan City
country: Taiwan
facility: Research Site
Study officials:
References: