PREDNISONE for Ocular myasthenia gravis | Myasthenia gravis

Inclusion criteria

• {"criterion_text":"- Patients over 18 years old\n- Diagnosis of ocular myasthenia within the last 6 months, defined : either by a typical clinical examination objectified by an expert clinician: ptosis and/or binocular diplopia, with a variable and fluctuating character (either spontaneous or provoked by effort or rest) ; or by positive anti-AChR antibodies or the presence of a decrement on repetitive nerve stimulation or a positive edrophonium test\n- Myasthenic symptoms limited to ocular and/or orbicular muscles muscles (no non-ocular symptoms on MMS, MGC and MG-ADL).\n- Myasthenic symptoms for at least one month (to rule out generalized myasthenia at the outset)\n- Immunosuppressive therapy-naive management of ocular myasthenia gravis\n- Having received information about the study and having signed a consent to participate in the study\n- Affiliated to a social security system"}

Exclusion criteria

• {"criterion_text":"- Thymoma\n- Risk of angle-closure glaucoma\n- Risk of urinary retention due to urethro-prostatic disorders\n- Alternative diagnosis to ocular involvement (pupillary abnormality other than that resulting from previous local disease or surgery abduction myopathy due to dysthyroid ophthalmopathy or dysimmune orbitopathy)\n- Vaccination with live attenuated vaccine required during study and up to 6 months after rituximab discontinuation\n- Women of childbearing age* who do not wish to use effective contraception (effective contraception includes oral contraception, intrauterine devices and other forms of contraception with a failure rate <1%) during their participation and at least 12 months after the last dose (oral commitment by the patient recorded in the file by the investigator)\n- Pregnant or breast-feeding women\n- Persons deprived of their liberty by judicial or administrative administrative decision, persons under psychiatric care by virtue of articles L.3212-1 and L.3213-1 and persons admitted to a health or social establishment for purposes purposes other than research (L.1121-6)\n- Adults under legal protection (L.1121-8)\n- History of immunosuppressive treatment or current immunosuppressive therapy for the management of a chronic pathology\n- Onset of ocular symptoms more than one year prior to diagnosis\n- Hypersensitivity to paracetamol\n- Hypersensitivity to rituximab or murine proteins\n- Hypersensitivity to prednisone and/or methylprednisone\n- Hypersensitivity to aziathioprine or 6-mercaptopurine\n- Hypersensitivity to dexchlorpheniramine\n- Any infectious condition (including hepatitis b)\n- Patients with severe immune deficiency\n- Severe heart failure (New York Heart Association (NYHA) Class IV) or severe uncontrolled heart disease\n- Severe liver failure\n- Psychotic state not yet controlled by treatment\n- Hyperuricemia on xanthine oxidase inhibitors (allopurinol and febuxostat)"}

Primary endpoints

• {"endpoint_text":"- Comparison of the proportion of patients who progressed to generalized myasthenia within 2 years of follow-up between the standardized experimental group and the control group. Generalization is defined by a loss of 5 points or more on any Myasthenic Muscle Score (MMS) item, excluding \"eyelid occlusion\" and \"extrinsic ocular musculature\".","definition_or_measurement_approach":"Generalization is defined by a loss of 5 points or more on any Myasthenic Muscle Score (MMS) item, excluding \"eyelid occlusion\" and \"extrinsic ocular musculature\"; endpoint compares proportion progressing to generalized myasthenia within 2 years between experimental and control groups."}

Secondary endpoints

• {"endpoint_text":"- Comparison of the proportion of patients who progressed to generalized myasthenia gravis in the first year of follow-up between the standardized experimental group and the control group. Generalization is defined as for the primary endpoint.\n- Comparison between the standardized experimental group and the control group of the severity of generalization symptoms according to: a. Myasthenic Muscle Score (MMS), b. Myasthenia Gravis Composite Scale (MGC), c. MG Activities of Daily Living (MG-ADL) score.\n- Comparison between standardized experimental group and control group of number of hospitalizations and ICU admissions due to myasthenia exacerbation or adverse event\n- Comparison of the proportion of patients treated with Ig-IV or plasma exchange between the standardized experimental group and the control group.\n- Comparison between the standardized experimental group and the control group of the proportion of patients achieving remission of ocular symptoms, as defined by the first three items of the MGC equal to zero\n- Comparison between the standardized experimental group and the control group of the proportion of patients presenting an ocular relapse, defined by a loss of 5 points or more on the sub-score comprising the 2 ocular items (diplopia and ptosis) of the MMS, without generalization of symptoms.\n- Comparison between standardized experimental group and control group of quality of life measured by: a. Myasthenia gravis quality of life score (MG-QoL15), b. National Eye Institute Visual Functioning Questionnaire with neuro-ophthalmological supplement (NEI VFQ 25 + supplement)\n- Comparison between standardized experimental group and control group, of cumulative dose of prednisone equivalent prescribed\n- Comparison between the standardized experimental group and the control group of changes in the following clinical scores: MMS, MGC, MG-ADL, MGFA-PIS.","definition_or_measurement_approach":"Secondary endpoints use proportions, clinical scale scores (MMS, MGC, MG-ADL, MG-QoL15, NEI VFQ-25 + supplement, MGFA-PIS), counts of hospitalizations/ICU admissions, treatment exposure (Ig-IV/plasma exchange), cumulative prednisone-equivalent dose, and predefined definitions for generalization/ocular relapse consistent with primary endpoint definitions."}

France

Earliest CTIS Part Ii Submission Date

01-03-2024

Latest Decision Or Authorization Date

06-06-2025

Processing Time Days

462

Number Of Sites

11

Number Of Participants

128

Primary sponsor

Full Name

Hopital Fondation Adolphe De Rothschild

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France