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Immediate Corticosteroid Therapy and Rituximab to Prevent Generalization in Ocular Myasthenia: a PROBE Multicenter Open-label Randomized Controlled Trial.

RecruitingPhase 3Ages 18 Years - anyAllFondation Ophtalmologique Adolphe de Rothschild
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In plain words

What the trial is, and whether it could suit you

Myasthenia is an autoimmune disease causing dysfunction of the neuromuscular junction, resulting in fluctuating and variable muscle weakness. In the initial phase of the disease, 70% of patients present with ocular onset myasthenia (OMG), i.e. weakness limited to the oculomotor muscles. Generalization to skeletal, bulbar and axial muscles occurs in 20-40% of cases, with a higher frequency in the first and second years, respectively 46% and 60% of generalizations. This reflects the maturation of the autoimmune response in the early years of the disease, and represents a therapeutic window of opportunity to modify the course of the disease. Generalization is a critical event, putting the patient at risk of admission to an intensive care unit and necessitating the use of long-term immunosuppressants. There is currently no validated strategy for preventing generalization. On the one hand,

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📋 Eligibility rules (as published)
Inclusion Criteria: * Patients over 18 years of age * 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 decrement on repetitive nerve stimulation or a positive edrophonium test * Ocular symptoms lasting at least one month and limited to extra-ocular muscles (weakness in one or both orbicularis oculi) * No non-ocular symptoms on MMS, MGC and MG-ADL. * Naïve to immunosuppressive therapy for ocular myasthenia gravis. Exclusion Criteria: * Thymoma * Pupillary anomaly other than that resulting from previous local disease or surgery. * Signs of restrictive abduction or supraduction myopathy due to dysthyroid ophthalmopathy. * Graves' ophthalmopathy * Onset of ocular symptoms more than one year before screening date * Hypersensitivity to rituximab, murine proteins, prednisone, methylprednisone, aziathioprine or 6-mercaptopurine, paracetamol, dexchlorpheniramine. * Any infectious condition * Patients with severe immune deficiency * Severe heart failure (New York Heart Association (NYHA) Class IV) or severe uncontrolled heart disease * Severe hepatic insufficiency * Psychotic states not yet controlled by treatment * Hyperuricemia on xanthine oxidase inhibitors (allopurinol and febuxostat) * Risk of angle-closure glaucoma * Risk of urinary retention due to urethro-prostatic disorders * Vaccination with live attenuated vaccine required during study and up to 6 months after rituximab discontinuation * Women of childbearing age who do not wish to use effective contraception during their participation and at least 12 months after * Pregnant or breast-feeding women
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What it tests

  • immediate treatment with corticosteroids
  • addition of rituximab if recurrence
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Where

11 sites
Centre Hospitalier Universitaire De Bordeaux
Bordeaux, France
Hôpital Raymond Pointcarre
Garches, France
Centre Hospitalier Universitaire De Lille
Lille, France
Hospices Civils De Lyon
Lyon, France
Centre Hospitalier Universitaire De Nice
Nice, France
CHNO
Paris, France
Hôpital de la Pitié-Salpêtrière
Paris, France
Centre Hospitalier Sainte Anne
Paris, France

…and 3 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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