Rapcabtagene Autoleucel for Myositis

Recruiting at 33 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Disqualifiers: Other myopathies, Organ dysfunction, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

123 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the effectiveness and safety of rapcabtagene autoleucel, a new treatment, compared to other standard treatments for people with severe myositis. Myositis causes muscle inflammation and weakness. The trial involves a single infusion of the experimental treatment. It seeks participants diagnosed with myositis who have not responded to previous treatments and are experiencing active and severe symptoms.

As a Phase 2 trial, this research focuses on measuring how well the treatment works in an initial, smaller group, offering participants a chance to contribute to important advancements in myositis treatment.

Will I have to stop taking my current medications?

The trial requires a complete washout of medications (a period without taking certain medications) before participating, so you may need to stop taking your current medications.

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

Research has shown that rapcabtagene autoleucel, a type of CAR T-cell therapy, has been safe in previous studies. Early results suggest that most people tolerate this treatment well. The treatment increases CAR T-cells and reduces B-cells, crucial actions for its effectiveness, without major safety issues.

This treatment is currently in Phase 2 trials for myositis, indicating it has passed initial safety tests in earlier phases. This phase focuses on further assessing its safety. While some unknowns remain, the treatment has shown promise in earlier studies. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for myositis?

Unlike standard treatments for myositis, which typically include corticosteroids and immunosuppressants to reduce inflammation, Rapcabtagene autoleucel uses a novel approach by employing CAR-T cell therapy. This treatment involves reprogramming a patient's own immune cells to specifically target and attack the cells causing the disease. Researchers are excited about this because it offers a highly targeted alternative that could potentially provide longer-lasting effects and reduce the need for chronic medication use. Additionally, this approach may offer hope for patients who do not respond well to traditional treatments.

What evidence suggests that rapcabtagene autoleucel might be an effective treatment for myositis?

This trial will compare rapcabtagene autoleucel, a type of CAR T-cell therapy, with an investigator's choice of treatment. Research has shown that rapcabtagene autoleucel might help treat severe, hard-to-treat muscle inflammation diseases. Early data suggest that this treatment can reduce certain immune cells, which may help decrease inflammation in these conditions. Initial findings also indicate that rapcabtagene autoleucel is safe and can increase T-cells in the body, potentially improving muscle strength and function. While research is still ongoing, these early results are promising for those with difficult-to-treat muscle inflammation. ^{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 men and women aged 18 to 65 with a confirmed diagnosis of severe myositis, who haven't responded well to previous treatments. They must have active disease symptoms and meet specific criteria for severity based on ACR/EULAR 2017 standards.

Inclusion Criteria

My disease is currently active.

My previous treatment did not work well.

I have been diagnosed with severe muscle inflammation.

Exclusion Criteria

Severe muscle damage at Screening

My organs are not functioning properly.

I have no unrelated health issues that would affect my tolerance to CAR-T cell therapy.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either rapcabtagene autoleucel or a comparator treatment for up to 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

Long-term follow-up

Participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up period

Up to 15 years

What Are the Treatments Tested in This Trial?

Interventions

• Rapcabtagene Autoleucel

Trial Overview The study compares the effectiveness and safety of a new therapy called Rapcabtagene autoleucel against an active comparator in patients with severe refractory idiopathic inflammatory myopathies (IIM). It's a Phase 2 trial where participants are randomly assigned to different treatment groups.

How Is the Trial Designed?

Group I: Rapcabtagene autoleucel Experimental Treatment 1 Intervention

Single infusion of rapcabtagene autoleucel (YTB323)

Group II: Comparator Active Control 1 Intervention

Investigator choice of treatment as per protocol

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

Published Research Related to This Trial

Brexucabtagene autoleucel (KTE-X19) CAR T-cell therapy showed a remarkable 91% objective response rate and 68% complete response rate in 68 patients with relapsed/refractory mantle cell lymphoma after a median follow-up of 35.6 months, indicating its efficacy in this challenging patient population.

The therapy demonstrated durable long-term responses with manageable safety, as late-onset toxicities were infrequent, occurring in only 3% of patients, suggesting that KTE-X19 is a safe and effective treatment option even for those with high-risk characteristics.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Three-Year Follow-Up of KTE-X19 in Patients With Relapsed/Refractory Mantle Cell Lymphoma, Including High-Risk Subgroups, in the ZUMA-2 Study. Wang, M., Munoz, J., Goy, A., et al.[2023]

In the updated ZUMA-3 study with a median follow-up of 26.8 months, brexucabtagene autoleucel (KTE-X19) achieved a 71% complete remission rate in adults with relapsed or refractory B-precursor acute lymphoblastic leukemia, indicating its efficacy as a treatment option.

Patients treated with KTE-X19 had a median overall survival of 25.4 months, significantly longer than the 5.5 months observed in matched historical controls, demonstrating a meaningful survival benefit with manageable safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Two-year follow-up of KTE-X19 in patients with relapsed or refractory adult B-cell acute lymphoblastic leukemia in ZUMA-3 and its contextualization with SCHOLAR-3, an external historical control study. Shah, BD., Ghobadi, A., Oluwole, OO., et al.[2023]

CAR-T therapies, specifically idecabtagene vicleucel (ide-cel) and ciltacabtagene autoleucel (cilta-cel), show significant potential in treating heavily pretreated relapsed/refractory multiple myeloma (RRMM), with updated clinical trial data indicating they can elicit responses in these patients.

Indirect comparisons suggest that cilta-cel may provide deeper and more durable responses than ide-cel in patients with triple class-exposed RRMM, although direct head-to-head trials are necessary to confirm these findings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Recent Advances in the Use of Chimeric Antigen Receptor-Expressing T-Cell Therapies for Treatment of Multiple Myeloma. Martin, T., Jackson, CC., Pacaud, L., et al.[2023]

Tisagenlecleucel (Kymriah™) and axicabtagene ciloleucel (Yescarta™) are pioneering CAR-T cell therapies that involve modifying a patient's own T cells to target cancer, marking a significant advancement in gene therapy.

The establishment of multiple CAR-T cell databases in France aims to enhance data collection and quality for patients treated with these therapies, which is crucial for ongoing research and improving treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[A standardized medical report template for CAR T-Cell therapy patients: Guidelines of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)]. Broussais, F., Vasseur, A., Karam, M., et al.[2022]

Equecabtagene autoleucel (Fucaso®) is a CAR-T cell therapy specifically designed to target B cell maturation antigen (BCMA) and has been conditionally approved in China for treating adults with relapsed or refractory multiple myeloma (RRMM) after at least three prior therapies.

This therapy represents a significant advancement in treatment options for RRMM patients, particularly those who have not responded to other treatments, highlighting its potential efficacy in a challenging patient population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Equecabtagene Autoleucel: First Approval. Keam, SJ.[2023]

Citations

1. novartis.com novartis.com/clinicaltrials/study/nct06665256

Phase 2 Study of Rapcabtagene Autoleucel in Myositis A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06665256?term=AREA%5BConditionSearch%5D(Acquired%20idiopathic%20inflammatory%20myopathy%20OR%20%22idiopathic%20inflammatory%20myopathies%22%20OR%20%22%20idiopathic%20inflammatory%20myositis%22%20OR%20%22%20IIm%22%20OR%20%22%20IMM%22)%20AND%20AREA%5BOverallStatus%5D(NOT_YET_RECRUITING%20OR%20RECRUITING)&rank=2

Phase 2 Study of Rapcabtagene Autoleucel in Myositis The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure.

3. novartis.com novartis.com/clinicaltrials/study/nct06655896

Phase 2 Study Evaluating Rapcabtagene Autoleucel in ... The purpose of this study is to evaluate the efficacy, safety and tolerability of rapcabtagene autoleucel (administered once following lymphodepletion)

4. ard.bmj.com ard.bmj.com/content/annrheumdis/83/Suppl_1/327.1.full.pdf

Scientific Abstracts 327 Conclusion: Preliminary data from this clinical trial suggest favourable safety,. CAR T-cell expansion, B-cell depletion and initial efficacy of ...

5. trial.medpath.com trial.medpath.com/clinical-trial/469407342d6c8b46/2024-514137-38-00-rapcabtagene-autoleucel-severe-refractory-inflammatory-myopathies

A clinical trial to learn about the effects of YTB323 in people ... How does rapcabtagene autoleucel compare to standard-of-care therapies in severe refractory idiopathic inflammatory myopathies? Which biomarkers are used to ...

6. healio.com healio.com/news/rheumatology/20250625/car-tcell-therapies-show-promise-immune-reset-in-multiple-diseases-at-eular-2025

CAR T-cell therapies show promise, immune reset in ... Patients with systemic lupus erythematosus who received autologous chimeric antigen receptor T-cell therapy enjoyed potent B-cell depletion and recovery.