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

NCT ID: NCT06704269

Last Updated: 2026-02-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-04-22

Study Completion Date

2029-10-26

Brief Summary

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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.

Detailed Description

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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 (gMG). 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).

Conditions

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Generalized Myasthenia Gravis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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YTB323

YTB323 single intravenous (i.v.) infusion

Group Type EXPERIMENTAL

YTB323

Intervention Type GENETIC

CAR-T cell suspension for intravenous infusion

Interventions

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YTB323

CAR-T cell suspension for intravenous infusion

Intervention Type GENETIC

Eligibility Criteria

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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).
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Univ Cali Irvine ALS Neuromuscular

Orange, California, United States

Site Status RECRUITING

Wake Forest Univ School of Medicine

Winston-Salem, North Carolina, United States

Site Status RECRUITING

Houston Methodist Hospital

Houston, Texas, United States

Site Status RECRUITING

Novartis Investigative Site

Bordeaux, , France

Site Status RECRUITING

Novartis Investigative Site

Brest, , France

Site Status RECRUITING

Novartis Investigative Site

Lille, , France

Site Status RECRUITING

Novartis Investigative Site

Chiba, , Japan

Site Status RECRUITING

Novartis Investigative Site

Kyoto, , Japan

Site Status RECRUITING

Novartis Investigative Site

Sheffield, South Yorkshire, United Kingdom

Site Status RECRUITING

Novartis Investigative Site

London, , United Kingdom

Site Status RECRUITING

Countries

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United States France Japan United Kingdom

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

1-888-669-6682

Novartis Pharmaceuticals

Role: CONTACT

+41613241111

Facility Contacts

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UCI Alpha Clinic

Role: primary

949-824-3990

Katie Hoots

Role: primary

336-716-1049

Jennifer Garrett

Role: primary

346-238-4516

Other Identifiers

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CYTB323O12101

Identifier Type: -

Identifier Source: org_study_id

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