Engineered immune cells take on tough Muscle-Weakening disease
NCT ID NCT06704269
First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 1 time
Summary
This early-stage trial is testing a new treatment called YTB323 for people with generalized myasthenia gravis that hasn't responded to standard therapies. YTB323 is a CAR-T cell therapy, meaning a patient's own immune cells are modified in a lab to better attack the disease. The study will enroll 15 participants to check safety, how long the cells last in the body, and whether they improve muscle weakness.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- YTB323 (a CAR-T cell therapy given by infusion)
- What this could lead to
- If successful, this could point toward a new treatment option for people with hard-to-control myasthenia gravis, potentially reducing symptoms and reliance on other medications.
- What could go wrong
- This is an early-phase trial with only 15 participants, so results may not apply to everyone. CAR-T therapies can cause serious side effects like cytokine release syndrome and nerve problems.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 15 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2025
- Expected to finish
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Oct 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. 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 2. MGFA Class III-IVa (gMG) at screening 3. 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. 4. 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 5. If treated with cholinesterase inhibitors, patients must be on a stable dose for at least two weeks prior to screening Exclusion Criteria: 1. Exclusively ocular myasthenia gravis (MGFA I), mild symptoms (MGFA II), or severe bulbar disease or MG crisis, MGFA Class IVb or V at screening 2. History of bone marrow/hematopoietic stem cell or solid organ transplantation. 3. 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 4. Other uncontrolled disease states, such as asthma, or inflammatory bowel disease, where flares are commonly treated with oral or parenteral corticosteroids, at screening 5. Participants with a known immunodeficiency syndrome (AIDS, hereditary immune deficiency, drug induced immune deficiency), or tested positive for HIV antibody, at screening 6. 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Houston Methodist Hospital
Houston, Texas, 77030, United States
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Novartis Investigative Site
Bordeaux, 33076, France
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Novartis Investigative Site
Brest, 29200, France
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Novartis Investigative Site
Lille, 59037, France
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Novartis Investigative Site
Chiba, Chiba, 2608677, Japan
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Novartis Investigative Site
Kyoto, 6068507, Japan
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Novartis Investigative Site
Sheffield, South Yorkshire, S10 2JF, United Kingdom
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Novartis Investigative Site
London, SE5 9RS, United Kingdom
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Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
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Univ Cali Irvine ALS Neuromuscular
Orange, California, 92868, United States
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Wake Forest Univ School of Medicine
Winston-Salem, North Carolina, 27157-1052, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new drug offer Long-Term relief for muscle weakness?
- Can a new drug ease the muscle weakness of myasthenia gravis?
- Engineered immune cells take aim at debilitating muscle weakness
- New hope for myasthenia gravis: experimental drug CNP-106 enters human trials
- New hope for myasthenia gravis patients: experimental drug enters phase 2 trial
- New drug trial aims to ease muscle weakness in rare disease