SYF6d807638180e48a693629dacd9f67fb6Registry ID: 2024-518939-12-00|Active Not RecruitingPhase 3
Single-cell deep phenotyping of B lymphocytes to personalize immunotherapy in patients with Myasthenia Gravis: clinical trial to evaluate the efficacy and safety of Rituximab in generalized AChR-antibody positive Myasthenia Gravis
Written informed consent and European Union Data Privacy Directive obtained from the subject prior to performing any protocol-related procedures, including screening evaluations
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Diagnosis of MG defined as: a. Positive serologic test for anti-AChR or anti-MuSK antibody titers as confirmed at screening (one retest allowed), and b. At least one of the following: History of abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; or History of positive anticholinesterase test (eg, edrophonium chloride test); or Patient demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician; or Clinical syndrome consistent with a diagnosis of MG, and not otherwise explained by another condition
MGFA Clinical Classification Class II, III, or IV at the time of screening and randomization
MG-ADL score of 5 or greater at screening and at randomization with > 50% of this score attributed to non-ocular items
QMG score of 11 or greater at screening and at randomization
Subjects must be on corticosteroids only, with no dose increase within 4 weeks prior to randomization and at least 20 mg prednisone per day (or equivalent dose on alternate day regimen)
Willing and able to comply with the protocol, complete study assessments, and return for follow-up visits.
Females of childbearing potential who are sexually active with a non-sterilized male partner must use at least one highly effective contraception method (Table 1) from the time of screening and for 12 months after the final dose of IP. Periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of contraception. Females of childbearing potential are defined as those who are not surgically sterile (ie, bilateral tubal ligation, bilateral oophorectomy, or complete hysterectomy) or those who are not postmenopausal (defined as 12 months with no menses without an alternative medical cause).
Non-sterilized males who are sexually active with a female partner of childbearing potential must use a condom from Day 1 for the duration of the study and for 3 months after the last dose of IP. Because male condom is not a highly effective contraception method, it is strongly recommended that female partners of a male study subject also use a highly effective method of contraception throughout this period (table 1 of the protocol)
Vital signs, electrocardiogram (ECG), and laboratory parameters within the normal ranges at screening, or, if outside normal ranges, deemed not clinically significant by the Investigator
You may not qualify if:
Any condition that, in the opinion of the Investigator, would place the patient at unacceptable risk of complications, interfere with evaluation of the IP, or confound the interpretation of patient safety or study results
Lactating or pregnant females, or females who intend to become pregnant anytime from signing the informed consent form (ICF) throughout the RCP plus 6 months following last dose of IP
History of drug or alcohol abuse within < 1 year prior to screening, or any condition associated with poor compliance as judged by the Investigator
Site staff and their family members
Currently committed to an institution by way of official or judicial order
Subjects diagnosed with congenital myasthenic syndromes
Known immunodeficiency disorder, including human immunodeficiency virus (HIV) infection
Thymectomy within ≤ 12 months prior to baseline (Day 1) visit or planned thymectomy during the duration of the RCP.
Receipt of the following medications or treatments at any time prior to randomization: a. Alemtuzumab (Lemtrada®, Campath®) b. Total lymphoid irradiation c. Bone marrow transplant d. T-cell vaccination therapy e. Natalizumab (Tysabri®)
Receipt of ANY immunosuppressive treatment (excluding corticosteroids) at ANY time prior to randomization (such as Azathioprine, Mycophenolate mofetil or Mycophenolic acid, Cyclosporine (except eye drop), Tacrolimus (except topical), Methotrexate, Cyclophosphamide, Tocilizumab (Actemra®), Belimumab (Benlysta®), Eculizumab (Soliris®), rituximab (MabThera®, Rituxan®), ocrelizumab (Ocrevus®), ofatumumab (Arzerra®), obinutuzumab (Gazyva®), inebilizumab, or any experimental B-cell depleting agent)
Receipt within the 4 weeks prior to Day 1: a. Intravenous immunoglobulin (IVIg) b. Plasma exchange (PLEX) treatment
Current use of: a. Prednisone < 20 mg/day or < 40 mg over a 2-day period (or equivalent dose of other corticosteroids) b. Pyridostigmine > 480 mg/day or unstable dose in the 2 weeks prior to Day 1
Concurrent/previous enrollment in another clinical study involving an investigational treatment within 4 weeks or 5 half-lives of the investigational treatment, whichever is longer, prior to Day 1
Receipt of a live attenuated vaccine within 4 weeks prior to randomization. Administration of inactivated (killed) vaccines is acceptable
History of severe allergic or anaphylactic reactions to biologic agents or known allergy to any component of the IP formulation
+12 more criteria
Contact the study team to confirm eligibility.
D1_Protocol synopsis IT_EU CT Number 2024-518939-12-00
E2_SmPC_Rituximab
K1_Recruitment arrangements
L2_Other subject information material_Consenso trattamento dati