Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Generalized Myasthenia Gravis

Recruiting now · Phase 1/Phase 2

Conditions studied: Generalized Myasthenia Gravis

In brief

This is a phase I/II study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia gravis. YTB323 is a Biological CAR-T cell therapy.

Key facts

Study ID

NCT06704269

Run by

Novartis Pharmaceuticals

People needed

15

Starts

2025-04-22

Expected to finish

2029-10-26

Last updated by the study team

2026-06-26

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed gMG diagnosis supported by the following:
• Documented report of positive serology testing for either AChR antibodies or MuSK antibodies at screening AND at least one of the following:
• History of abnormal neuromuscular transmission test demonstrated by repetitive nerve stimulation or single-fiber electromyography
• History of positive acetylcholinesterase inhibitor test
• Improvement in MG signs on an oral acetylcholinesterase inhibitor as assessed by the treating physician
• MGFA Class III-IVa (gMG) at screening
• Treatment-resistant gMG as defined by: MG-ADL score ≥ 6 (≥50% non-ocular) at screening despite adequate treatment trials with at least two different non-steroidal immunosuppressive drugs given at adequate doses and duration of therapy.
• If on chronic corticosteroids, must be on a stable dose of corticosteroids for ≥1 month prior to screening and have the ability and willingness to taper to a maximum dose of 10 mg prednisolone daily or equivalent at least one week before leukapheresis
• If treated with cholinesterase inhibitors, patients must be on a stable dose for at least two weeks prior to screening

You may not qualify if…

• Exclusively ocular myasthenia gravis (MGFA I), mild symptoms (MGFA II), or severe bulbar 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, at screening
• Participants with a known immunodeficiency syndrome (AIDS, hereditary immune deficiency, drug induced immune deficiency), or tested positive for HIV antibody, at screening
• Prior treatment with anti-CD19 therapy, adoptive T cell therapy or any prior gene therapy product (e.g. CAR-T cell therapy).
• Other protocol-defined inclusion/exclusion criteria may apply

Where it is running

• Univ Cali Irvine ALS Neuromuscular — Orange, California, United States (enrolling)
• Wake Forest Univ School of Medicine — Winston-Salem, North Carolina, United States (enrolling)
• Thomas Jefferson University — Philadelphia, Pennsylvania, United States (enrolling)
• Houston Methodist Hospital — Houston, Texas, United States (enrolling)
• Novartis Investigative Site — Bordeaux, France (enrolling)
• Novartis Investigative Site — Brest, France (enrolling)
• Novartis Investigative Site — Lille, France (enrolling)
• Novartis Investigative Site — Chiba, Chiba, Japan (enrolling)
• Novartis Investigative Site — Kyoto, Japan (enrolling)
• Novartis Investigative Site — Sheffield, South Yorkshire, United Kingdom (enrolling)
• Novartis Investigative Site — London, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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