YTB323 for Myasthenia Gravis

Recruiting in Palo Alto (17 mi)

+3 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Novartis Pharmaceuticals

Must be taking: Cholinesterase inhibitors

Must not be taking: Anti-CD19 therapy

Disqualifiers: Ocular MG, Transplant history, Infections, others

No Placebo Group

Trial Summary

What is the purpose of this trial?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.

Will I have to stop taking my current medications?

The trial requires that if you are on chronic corticosteroids, you must be willing to reduce your dose to a maximum of 10 mg of prednisolone daily at least one week before a procedure called leukapheresis. If you are taking cholinesterase inhibitors, you need to be on a stable dose for at least two weeks before screening.

Eligibility Criteria

This trial is for people with generalized myasthenia gravis (gMG) who haven't improved after trying at least two different non-steroidal immunosuppressive drugs. They should have a certain level of muscle weakness, a history of abnormal neuromuscular tests, and be able to reduce their use of steroids or maintain a stable dose of cholinesterase inhibitors.

Inclusion Criteria

My myasthenia gravis is severe and hasn't improved with at least two different treatments.

I can reduce my steroid use to 10 mg of prednisolone daily before a specific procedure.

I've been on a steady dose of memory medications for over two weeks.

+2 more

Exclusion Criteria

My myasthenia gravis ranges from eye-related symptoms to severe with crisis.

Known immunodeficiency syndrome, tested positive for HIV antibody, or prior treatment with specific therapies

I have had a bone marrow, stem cell, or organ transplant.

+2 more

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous infusion of YTB323

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 years

Regular visits (frequency not specified)

Long-Term Follow-up

Participants are followed until 15 years after YTB323 administration

15 years

Participant Groups

The study is testing YTB323, which is a CAR-T cell therapy—a type of treatment that modifies the patient's immune cells to fight the disease. It aims to see if this new therapy is safe, effective, and how it affects the body's cells in those with treatment-resistant gMG.

1Treatment groups

Experimental Treatment

Group I: YTB323Experimental Treatment1 Intervention

YTB323 single intravenous (i.v.) infusion