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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

 No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

 Interventional
Eligible Ages 18 Years - 65 Years
Gender All
More Inclusion & Exclusion Criteria

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 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, 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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

 NCT06704269
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

 Phase 1/Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

 Novartis Pharmaceuticals
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

 Novartis Pharmaceuticals
Principal Investigator Affiliation Novartis Pharmaceuticals
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

 Industry
Overall Status Recruiting
Countries Japan, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

 Generalized Myasthenia Gravis
Additional 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).

Arms & Interventions

Arms

Experimental: YTB323

YTB323 single intravenous (i.v.) infusion

Interventions

Genetic: - YTB323

CAR-T cell suspension for intravenous infusion

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Univ Cali Irvine ALS Neuromuscular, Orange, California

Status

Recruiting

Address

Univ Cali Irvine ALS Neuromuscular

Orange, California, 92868

Site Contact

Mihaela Nistor

[email protected]

1-888-669-6682

Houston Methodist Hospital, Houston, Texas

Status

Recruiting

Address

Houston Methodist Hospital

Houston, Texas, 77030

Site Contact

Teresa Martinez

[email protected]

1-888-669-6682

International Sites

Novartis Investigative Site, Chiba, Japan

Status

Recruiting

Address

Novartis Investigative Site

Chiba, , 2608677

Novartis Investigative Site, Kyoto, Japan

Status

Recruiting

Address

Novartis Investigative Site

Kyoto, , 606 8507

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