Phase
Condition
Neuropathy
Polymyositis (Inflammatory Muscle Disease)
Sarcopenia
Treatment
YTB323
Clinical Study ID
Ages 18-65 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
- Confirmed gMG diagnosis supported by the following:
Documented report of positive serology testing for either AChR antibodies orMuSK antibodies at screening AND at least one of the following:
History of abnormal neuromuscular transmission test demonstrated by repetitivenerve stimulation or single-fiber electromyography
History of positive acetylcholinesterase inhibitor test
Improvement in MG signs on an oral acetylcholinesterase inhibitor as assessedby the treating physician
MGFA Class III-IVa (gMG) at screening
Treatment-resistant gMG as defined by: MG-ADL score ≥ 6 at screening despiteadequate treatment trials with at least two different non-steroidalimmunosuppressive drugs given at adequate doses and duration of therapy.
If on chronic corticosteroids, the ability and willingness to taper to a maximumdose of 10 mg prednisolone daily or equivalent at least one week beforeleukapheresis
If treated with cholinesterase inhibitors, patients must be on a stable dose for atleast two weeks prior to screening
Exclusion
Exclusion Criteria:
Exclusively ocular myasthenia gravis (MGFA I), mild symptoms (MGFA II), or severebulbar disease or MG crisis, MGFA Class IVb or V at screening
History of bone marrow/hematopoietic stem cell or solid organ transplantation.
Clinically significant active, opportunistic, chronic or recurrent infection (including positive for hepatitis B or hepatitis C) confirmed by clinical evidence,imaging, or positive laboratory tests one month prior to leukapheresis
Other uncontrolled disease states, such as asthma, or inflammatory bowel disease,where flares are commonly treated with oral or parenteral corticosteroids, atscreening
Participants with a known immunodeficiency syndrome (AIDS, hereditary immunedeficiency, drug induced immune deficiency), or tested positive for HIV antibody, atscreening
Prior treatment with anti-CD19 therapy, adoptive T cell therapy or any prior genetherapy product (e.g. CAR-T cell therapy).
Other protocol-defined inclusion/exclusion criteria may apply
Study Design
Study Description
Connect with a study center
Novartis Investigative Site
Chiba, 2608677
JapanActive - Recruiting
Novartis Investigative Site
Kyoto, 606 8507
JapanActive - Recruiting
Univ Cali Irvine ALS Neuromuscular
Orange, California 92868
United StatesActive - Recruiting
Houston Methodist Hospital
Houston, Texas 77030
United StatesActive - Recruiting
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