YTB323 for Myasthenia Gravis

Recruiting at 8 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

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 All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called YTB323 for individuals with generalized myasthenia gravis (gMG) who haven't found success with other treatments. YTB323 is a cell therapy that uses specially engineered cells to potentially enhance immune system function. The main goals are to assess the treatment's safety, effectiveness, and behavior in the body. Suitable candidates for this trial have a confirmed gMG diagnosis, face significant daily challenges due to the condition, and have not improved with at least two other treatments. As a Phase 1 trial, this research aims to understand how YTB323 works in people, offering participants the opportunity to be among the first to receive this innovative treatment.

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.

Is there any evidence suggesting that YTB323 is likely to be safe for humans?

Research has shown that YTB323, a type of CAR-T cell therapy, is being tested for safety in patients with treatment-resistant generalized myasthenia gravis. In other studies with similar treatments, patients have generally tolerated them well. However, similar CAR-T therapies have reported side effects like fevers or low blood pressure. Since YTB323 remains in the early testing stages, complete safety information is still being gathered. While some evidence suggests it is safe, further research is necessary to confirm this. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about YTB323 for myasthenia gravis because it represents a novel approach compared to existing treatments. Current options often include medications like acetylcholinesterase inhibitors, corticosteroids, and immunosuppressants, which can come with significant side effects and require long-term management. Unlike these treatments, YTB323 is a single intravenous infusion that leverages a different mechanism of action, potentially offering a more targeted and efficient way to manage symptoms. This innovative approach could lead to quicker relief and fewer side effects, making it a promising option for patients.

What evidence suggests that YTB323 might be an effective treatment for myasthenia gravis?

Research has shown that YTB323, a type of CAR-T cell therapy, could help treat generalized myasthenia gravis, particularly in patients unresponsive to other treatments. CAR-T cell therapy modifies a person's immune cells to enhance their ability to combat the disease. Early results suggest a significant reduction in symptoms, with improvements lasting at least six months. This method builds on positive outcomes from similar treatments targeting immune system cells. Although further research is necessary, this therapy's unique mechanism offers hope for those with difficult-to-treat conditions. ^{1 2 3 4 6}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

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.

Exclusion Criteria

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

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

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

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• YTB323

Trial Overview 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.

How Is the Trial Designed?

Group I: YTB323 Experimental Treatment 1 Intervention

YTB323 single intravenous (i.v.) infusion

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Citations

1. novartis.com novartis.com/clinicaltrials/study/nct06704269

Study to Assess Safety, Efficacy, and Cellular Kinetics of ... This is a phase I/II study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia gravis.

2. neurologylive.com neurologylive.com/view/early-stage-study-test-cd19-car-t-cell-therapy-ytb323-myasthenia-gravis

Early-Stage Study to Test CD19 CAR T-Cell Therapy ... The trial assesses YTB323's safety, efficacy, and cellular kinetics in 15 patients with treatment-resistant generalized myasthenia gravis (gMG).

3. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/559015

Study to Assess Safety, Efficacy, and Cellular Kinetics of ... This is a phase I/II study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05798117

NCT05798117 | An Open-label, Study to Assess Safety ... The study is intended to assess safety, efficacy and cellular kinetics of YTB323 treatment in participants with severe refractory systemic lupus erythematosus.

5. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ytb323-and-drug-combination-for-patients-with-treatment-resistant-generalized-myasthenia-gravis/

Study on the Safety and Effects of YTB323 and Drug ... The study aims to determine if participants achieve a significant reduction in symptoms and maintain this improvement for six months.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06617793

NCT06617793 | An Open-label Study to Assess the Safety, ... This is an open-label, multi-center, non-confirmatory study to assess the safety, efficacy, and cellular kinetics of YTB323 in approximately 28 participants ...