Rapcabtagene Autoleucel for Myositis

Recruiting in Palo Alto (17 mi)

+10 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Novartis Pharmaceuticals

Disqualifiers: Other myopathies, Organ dysfunction, others

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)

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.

How is the treatment Rapcabtagene Autoleucel unique for myositis?

Rapcabtagene Autoleucel is a novel treatment for myositis because it uses CAR-T cell therapy, which involves modifying a patient's own immune cells to better target and attack specific cells. This approach is different from traditional treatments for myositis, which often involve immunosuppressive drugs or steroids, as it harnesses the body's immune system in a more targeted way. ^{1 2 3 4 5}

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

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 am between 18 and 65 years old and have been diagnosed with myositis.

Exclusion Criteria

My organs are not functioning properly.

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

I am not allergic to any medications or ingredients in the study.

Trial Timeline

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

Treatment Details

Interventions

• Rapcabtagene Autoleucel (CAR T-cell Therapy)

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.

Participant Groups

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

Findings from Research

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]

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]

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Recent Advances in the Use of Chimeric Antigen Receptor-Expressing T-Cell Therapies for Treatment of Multiple Myeloma. [2023]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Equecabtagene Autoleucel: First Approval. [2023]

3. England 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. [2023]

4. United States 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. [2023]

5. France 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)]. [2022]