Clinical trial NCT06967480: Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis
Official title: MG-EVOLUTION. Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis: A Real-World Study With Biomarker Analysis
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Generalized Myasthenia Gravis
Anti-AChR Antibody Positive
Keywords: Anti-AChR Antibody-Positive
Generalized Myasthenia Gravis
gMG
Ravulizumab
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2025-05-13
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with anti-AChR antibody positive, generalized, myasthenia gravis elegible to Ravulizumab treatment with a disease duration from onset equal or less than 3 years.

Brief summary: The primary objective of the study is to evaluate the effectiveness of Ravulizumab in improving MG-ADL in an early-stage AChR+ gMG population.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of MG confirmed by:

  * History of a positive serologic test for anti-AChR antibodies, and
  * One of the following:

    * History of abnormal neuromuscular transmission test demonstrated by singlefibre electromyography or repetitive nerve stimulation
    * History of positive anticholinesterase test (e.g., edrophonium chloride test); or
    * Patient demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician
* Disease duration from MG onset ≤ 3 years before T-4 (Enrolment);
* MGFA class IIb to IV;
* Patient eligible for Ravulizumab treatment based on AIFA reimbursement criteria
* Vaccination cycle for Neisseria meningitidis completed at least two weeks before Ravulizumab initiation or antibiotics chemoprophylaxis according to the SmPC.

Exclusion Criteria:

* Patient unable to understand and sign the informed consent
* Hypersensitivity to the active substance or to any of the excipients of the study product
* Patient for whom the study product is contraindicated according to SmPC
* Previous treatment with C5 inhibitors
* Last rituximab infusion performed \< 6 months before T-4 (Enrolment)
* Last infusion with FcRn blockers performed \< 3 months before T-4 (Enrollment)
* Pregnant or lactating or planning a pregnancy during the study
* Patient who plan to relocate during the study
* Patient who are unsure of following the visit schedule
* Patient unable to complete questionnaires
* Previous or current participation to other interventional studies

Primary outcomes:
measure: Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 50
timeFrame: Week 50
measure: Change From Baseline in MG-ADL Total Score at Week 50
timeFrame: Baseline, Week 50
Secondary outcomes:
measure: MG-ADL Total Score at Weeks 2, 10, and 26
timeFrame: Weeks 2, 10, and 26
measure: Change From Baseline in MG-ADL Total Score at Weeks 2, 10 and 26
timeFrame: Baseline, Weeks 2, 10 and 26
measure: Percentage of Participants With >=2- Point Reduction From Baseline in MG-ADL Total Score at Weeks 2, 10, 26, and 50
timeFrame: Baseline, Weeks 2, 10, 26, and 50
measure: Quantitative Myasthenia Gravis (QMG) Total Score at Weeks 2, 10, 26, and 50
timeFrame: Weeks 2, 10, 26, and 50
measure: Change From Baseline in QMG Total Score at Weeks 2, 10, 26 and 50
timeFrame: Baseline, Weeks 2, 10, 26 and 50
measure: Percentage of Participants With >=3- Point Reduction From Baseline in QMG Total Score at Weeks 2, 10, 26, and 50
timeFrame: Baseline, Weeks 2, 10, 26, and 50
measure: Myasthenia Gravis Composite (MGC) Total Score at Weeks 2, 10, 26, and 50
timeFrame: Weeks 2, 10, 26, and 50
measure: Change From Baseline in MGC Total Score at Weeks 2,10, 26 and 50
timeFrame: Baseline, Weeks 2, 10, 26 and 50
measure: Percentage of Participants With >=3- Point Reduction From Baseline in MGC Total Score at Weeks 2, 10, 26 and 50
timeFrame: Baseline, Weeks 2, 10, 26, and 50
measure: Myasthenia Gravis Quality of Life 15-Item Scale - Revised (MG-QoL15r) Total Score at Weeks 10, 26, and 50
timeFrame: Weeks 10, 26, and 50
measure: Change From Baseline in MG-QoL15r Total Score at Weeks 10, 26 and 50
timeFrame: Baseline, Weeks 10, 26 and 50
measure: Hospital Anxiety and Depression Scale (HADS) Total Score at Weeks 10, 26, and 50
timeFrame: Weeks 10, 26, and 50
measure: Change From Baseline in HADS Total Score at Weeks 10, 26 and 50
timeFrame: Baseline, Weeks 10, 26 and 50
measure: Insomnia Severity Index Scale (ISI) Total Score at Weeks 10, 26, and 50
timeFrame: Weeks 10, 26, and 50
measure: Change From Baseline in ISI Total Score at Weeks 10, 26 and 50
timeFrame: Baseline, Weeks 10, 26 and 50
measure: Pain Visual Analogue Scale (Pain VAS) Total Score at Weeks 2, 10, 26, and 50
timeFrame: Weeks 2, 10, 26, and 50
measure: Change From Baseline in Pain VAS Total Score at Weeks 2, 10, 26 and 50
timeFrame: Baseline, Weeks 2, 10, 26 and 50
measure: Percentage of Participants With Mean Dose Reduction From Baseline of Corticosteroids and Immunosuppressants (ISTs) Treatment at Weeks 2, 10, 26, and 50
timeFrame: Baseline, Weeks 2, 10, 26, and 50
measure: Percentage of Participants in Different ISTs Status at Baseline, and at Weeks 2, 10, 26, and 50
timeFrame: Baseline, Weeks 2, 10, 26, and 50
measure: Number of Clinical Deterioration Events
timeFrame: Baseline, Weeks 2, 10, 26, and 50
measure: Percentage of Participants With Minimal Symptom Expression at Weeks 2, 10, 26, and 50
timeFrame: Weeks 2, 10, 26, and 50
measure: Institute For Medical Technology Assessment (iMTA) Productivity Cost Questionnaire (iPCQ) Score at Weeks 10, 26, and 50
timeFrame: Weeks 10, 26, and 50
measure: Change From Baseline in iMTA iPCQ Score at Weeks 10, 26 and 50
timeFrame: Baseline, Weeks 10, 26 and 50
measure: Biomarkers analyses (complement system proteins, interleukins and miRNA serum levels)
timeFrame: Baseline, Week 10, 26, and 50
Other outcomes:
Locations:
city: Bergamo
country: Italy
facility: Clinical Trial Site
city: Bologna
country: Italy
facility: Clinical Trial Site
city: Brescia
country: Italy
facility: Clinical Trial Site
city: Firenze
country: Italy
facility: Clinical Trial Site
city: Imperia
country: Italy
facility: Clinical Trial Site
city: Lecco
country: Italy
facility: Clinical Trial Site
city: Messina
country: Italy
facility: Clinical Trial Site
city: Milano
country: Italy
facility: Clinical Trial Site
city: Napoli
country: Italy
facility: Clinical Trial Site
city: Novara
country: Italy
facility: Clinical Trial Site
city: Orbassano
country: Italy
facility: Clinical Trial Site
city: Padova
country: Italy
facility: Clinical Trial Site
city: Palermo
country: Italy
facility: Clinical Trial Site
city: Parma
country: Italy
facility: Clinical Trial Site
city: Pavia
country: Italy
facility: Clinical Trial Site
city: Pisa
country: Italy
facility: Clinical Trial Site
city: Roma
country: Italy
facility: Clinical Trial Site
city: Salerno
country: Italy
facility: Clinical Trial Site
city: Sassari
country: Italy
facility: Clinical Trial Site
city: Torino
country: Italy
facility: Clinical Trial Site
Study officials:
References: