Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Generalized Myasthenia Gravis
a study on Myasthenia Gravis
Summary
- Eligibility
- for people ages 18-65 (full criteria)
- Location
- at Orange, California and other locations
- Dates
- study startedcompletion around
Description
Summary
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.
Official Title
An Open-label, Multi-center, Phase I/II Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Participants With Treatment-resistant Generalized Myasthenia Gravis
Details
This is an open-label, multi-center, non-confirmatory study intended to assess safety, efficacy, and cellular kinetics of YTB323 treatment in participants with treatment-resistant generalized myasthenia gravis in order to enable a benefit to risk assessment for further development in generalized myasthenia gravis (gMS). The study plans to enroll approximately 15 participants with treatment-resistant gMG. The study utilizes a single dose design across 2 cohorts, consisting of a sentinel cohort of 3 patients followed by an expansion cohort of an additional 12 patients.
All participants dosed with YTB323 will be followed until 15 years after YTB323 administration in the Long-Term Follow-up (LTFU).
Keywords
Generalized Myasthenia Gravis, myasthenia gravis, YTB323, CAR-T, Muscle Weakness
Eligibility
You can join if…
Open to people ages 18-65
- 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 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, 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 CAN'T join 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
Locations
- Univ Cali Irvine ALS Neuromuscular
accepting new patients
Orange California 92868 United States - Novartis Investigative Site
accepting new patients
Kyoto 606 8507 Japan
Details
- Status
- accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- Novartis Pharmaceuticals
- ID
- NCT06704269
- Phase
- Phase 1/2 Myasthenia Gravis Research Study
- Study Type
- Interventional
- Participants
- Expecting 15 study participants
- Last Updated
Please contact me about this study
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