CAR-T Therapy for Multiple Myeloma

Recruiting at 6 trial locations

Overseen By Clinical Trials Referral Office

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Mayo Clinic

Disqualifiers: Ineligible for CAR-T, Myeloid neoplasm

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 1 day

82 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on how specific genetic changes might affect blood cell levels after CAR-T therapy (Chimeric Antigen Receptor T-cell Therapy) in individuals with multiple myeloma or certain lymphoproliferative disorders, where white blood cells grow uncontrollably. The research aims to identify gene mutations that could predict which patients risk prolonged low blood cell counts (cytopenia) after treatment. Participants will provide various samples and undergo tests to help researchers explore these genetic influences. This trial suits individuals diagnosed with multiple myeloma or a CD19-positive lymphoproliferative disorder who are willing to provide samples for research and return for follow-up visits. As an unphased trial, this study offers a unique opportunity to contribute to groundbreaking research that could improve future treatments.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that CAR-T Therapy is safe for patients with multiple myeloma or CD19 positive lymphoproliferative disorder?

Research has shown that CAR-T therapy, which uses the body's immune cells to fight cancer, holds promise for some patients. However, it carries risks. Studies have identified cytopenia, a condition of having fewer blood cells than normal, as a common issue. This can occur after receiving CAR-T therapy, with about one-third of patients experiencing it for more than 30 days post-treatment.

While CAR-T therapy is approved for certain cancers, awareness of the risks is important. Patients have reported side effects such as fever, fatigue, and nausea. These side effects are often manageable but can become serious, particularly if blood cell counts drop significantly.

In summary, CAR-T therapy can be effective but comes with potential side effects. It is crucial for anyone considering this treatment to discuss the risks and management strategies with their doctors. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about CAR-T therapy for multiple myeloma because it offers a novel approach by modifying a patient's own T-cells to better target and destroy cancer cells. Unlike standard treatments like chemotherapy or stem cell transplants, which can affect both healthy and cancerous cells, CAR-T therapy is highly specific, targeting the cancer cells directly. This precision not only has the potential to improve treatment outcomes but also minimizes damage to healthy cells, potentially reducing side effects. Additionally, CAR-T therapy could lead to longer-lasting remissions, offering hope for a more durable response in patients with this challenging condition.

What evidence suggests that CAR-T Therapy might be an effective treatment for multiple myeloma?

Research has shown that CAR-T therapy is a strong option for treating multiple myeloma and similar blood cancers. In this trial, CAR-T therapy modifies a patient's own immune cells to better find and attack cancer cells. Studies have found that many patients with B-cell and plasma cell cancers have benefited from this therapy. CAR-T therapy has greatly reduced cancer cells in the body by training the immune system to target and destroy them effectively. Participants in this trial will receive CAR-T therapy, and supportive care will include bone marrow aspiration and CFU analysis to monitor treatment effects. ^{1 3 4 6 7}

Who Is on the Research Team?

Mithun Shah, MD

Principal Investigator

Mayo Clinic in Rochester

Are You a Good Fit for This Trial?

This trial is for patients with multiple myeloma or CD19+ lymphoproliferative disorders who have undergone CAR-T therapy and are experiencing low blood cell counts. The study aims to understand the genetic factors contributing to this condition.

Inclusion Criteria

Willingness to provide mandatory bone marrow aspirate specimens for correlative research

Willingness to provide mandatory hair follicle specimens for correlative research

Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study)

Exclusion Criteria

I was diagnosed with a blood cancer before receiving CAR-T therapy.

I am not eligible for CAR-T cell therapy.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

1 visit (in-person)

Pre-Treatment Sample Collection

Patients undergo bone marrow aspiration and hair, buccal, and saliva sample collection up to 14 days prior to lymphodepleting therapy

2 weeks

1 visit (in-person)

Treatment

Patients receive CAR-T therapy

1 day

1 visit (in-person)

Follow-up

Patients undergo clinical follow-up on day 90 post-CAR-T therapy. Patients with unexplained cytopenia are followed up every 90 days for up to 2 years until resolution. Patients without unexplained cytopenia are followed clinically for up to 2 years.

Up to 2 years

Multiple visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• CAR-T Therapy

Trial Overview The trial involves collecting biospecimens, genetic counseling and testing, bone marrow aspiration, reviewing electronic health records, and follow-up visits to investigate how gene variants affect blood cell count after CAR-T therapy.

How Is the Trial Designed?

Group I: Supportive care (bone marrow aspiration, CFU) Experimental Treatment 6 Interventions

Patients undergo bone marrow aspiration and hair, buccal, saliva sample collection up to 14 days prior to LD therapy. Patients undergo CFU on day 90 post-CAR-T therapy. Patients with unexplained cytopenia also undergo bone marrow aspiration for sequencing analysis on day 90 and at development of MN-pCT during CFU. Patients also undergo bone marrow aspiration at determination of clonal evolution or myeloid neoplasm if not done during on day 90.

CAR-T Therapy is already approved in United States, European Union for the following indications:

Approved in United States as Kymriah for:

• B-cell precursor acute lymphoblastic leukemia (ALL)
• Relapsed or refractory large B-cell lymphoma
• Diffuse large B-cell lymphoma (DLBCL)

Approved in European Union as Kymriah for:

• B-cell precursor acute lymphoblastic leukemia (ALL)
• Relapsed or refractory large B-cell lymphoma
• Diffuse large B-cell lymphoma (DLBCL)

Approved in United States as Yescarta for:

• Relapsed or refractory large B-cell lymphoma
• Diffuse large B-cell lymphoma (DLBCL)

Approved in European Union as Yescarta for:

• Relapsed or refractory large B-cell lymphoma
• Diffuse large B-cell lymphoma (DLBCL)

Approved in United States as Tecartus for:

• Relapsed or refractory mantle cell lymphoma
• Relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)

Approved in European Union as Tecartus for:

• Relapsed or refractory mantle cell lymphoma
• Relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

Published Research Related to This Trial

Two CAR T cell therapies, Tisagenlecleucel and Axicabtagene ciloleucel, have been approved for treating specific types of blood cancers, including B-cell acute lymphoblastic leukemia and large B-cell lymphoma, in patients who have not responded to other treatments.

This review emphasizes the importance of recognizing and managing the toxicities associated with CAR T cell therapies, while also suggesting future strategies to reduce these side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

CAR T Cell Toxicity: Current Management and Future Directions. Yáñez, L., Sánchez-Escamilla, M., Perales, MA.[2020]

Ciltacabtagene autoleucel (cilta-cel) significantly improved progression-free survival in patients with lenalidomide-refractory multiple myeloma, with a median progression-free survival not reached in the cilta-cel group compared to 11.8 months in the standard-care group, based on a phase 3 trial with 419 participants.

The cilta-cel group also showed higher overall response rates (84.6% vs. 67.3%) and complete response rates (73.1% vs. 21.8%) compared to standard care, although most patients experienced grade 3 or 4 adverse events, including cytokine release syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cilta-cel or Standard Care in Lenalidomide-Refractory Multiple Myeloma. San-Miguel, J., Dhakal, B., Yong, K., et al.[2023]

CAR-T cell therapies, specifically axicabtagene ciloleucel and tisagenlecleucel, have significantly improved outcomes for patients with relapsed or refractory aggressive B-cell lymphomas, with approvals based on pivotal trials demonstrating their efficacy after multiple prior treatments.

Despite their similar CAR technologies, differences in manufacturing and clinical trial designs exist between these therapies, highlighting the need for ongoing monitoring of patient responses and potential long-term side effects in real-world settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of CD19-directed CAR-T cell therapies in patients with relapsed/refractory aggressive B-cell lymphomas: Observations from the JULIET, ZUMA-1, and TRANSCEND trials. Westin, JR., Kersten, MJ., Salles, G., et al.[2022]

CAR T-cell therapies, such as tisagenlecleucel (Kymriah™) and axicabtagene ciloleucel (Yescarta™), are effective treatments for relapsed/refractory B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma, utilizing genetically modified T cells that target the CD19 antigen.

These therapies are classified as 'living drugs' because they involve the genetic engineering of a patient's own T cells, highlighting the importance of proper collection and manufacturing processes for effective treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[How to perform leukapheresis for procurement of the staring material used for commercial CAR T-cell manufacturing: A consensus from experts convened by the SFGM-TC]. Carnoy, S., Beaumont, JL., Kanouni, T., et al.[2021]

A survey of 41 experienced clinicians revealed that many do not consider a history of malignancy or prior treatments as contraindications for CAR T-cell therapy, indicating a broader eligibility for patients than previously thought.

There is significant variability in follow-up care models after CAR T therapy, with most clinicians recommending frequent hospital visits in the initial month, highlighting the need for standardized guidelines in patient management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An international survey on the management of patients receiving CAR T-cell therapy for haematological malignancies on behalf of the Chronic Malignancies Working Party of EBMT. Hayden, PJ., Sirait, T., Koster, L., et al.[2021]

Chimeric antigen receptor (CAR) T-cell therapy shows great promise for treating relapsed/refractory multiple myeloma (RRMM), building on its success in other blood cancers like acute lymphocytic leukemia and diffuse large B-cell lymphoma.

The review highlights six potential target antigens for CAR T-cell therapy in multiple myeloma and discusses the safety profiles of existing CAR T-cell treatments, indicating a significant advancement in the management of this challenging disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current advances in chimeric antigen receptor T-cell therapy for refractory/relapsed multiple myeloma. Huang, H., Wu, HW., Hu, YX.[2020]

Ciltacabtagene autoleucel (cilta-cel) therapy significantly improved health-related quality of life (HRQoL) in patients with relapsed/refractory multiple myeloma, with a notable decrease in reported symptoms such as pain and fatigue from 85.2% at baseline to 22.2% by Day 184.

The majority of patients (70.8%) felt that cilta-cel met or exceeded their treatment expectations, highlighting its efficacy and the positive impact of a long treatment-free period on their overall health perception.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient Perceptions Regarding Ciltacabtagene Autoleucel Treatment: Qualitative Evidence From Interviews With Patients With Relapsed/Refractory Multiple Myeloma in the CARTITUDE-1 Study. Cohen, AD., Hari, P., Htut, M., et al.[2023]

Cytokine release syndrome (CRS) is the most common adverse reaction reported after CAR-T therapy, with significantly higher reports for axicabtagene ciloleucel compared to tisagenlecleucel, indicating a need for careful monitoring of this side effect.

The study analyzed 1426 reports of adverse drug reactions from real clinical settings, supporting the overall safety profile of CAR-T therapies while confirming CRS as a major concern.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety profile of chimeric antigen receptor T-cell immunotherapies (CAR-T) in clinical practice. Bonaldo, G., Montanaro, N., AlbertoVaccheri, ., et al.[2021]

Chimeric antigen receptor (CAR) T-cell therapy, such as tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta), is an effective treatment for relapsed/refractory B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma, utilizing genetically modified T-cells that target the CD19 antigen.

This collaborative work provides practical guidance for healthcare centers on how to select patients for these commercially available CAR T-cell therapies, ensuring appropriate use and maximizing treatment benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Eligibility of patients for CAR T-cell: Expert opinion-based collaborative work by the SFGM-TC]. Beauvais, D., Bachy, E., Baruchel, A., et al.[2021]

Citations

1. bloodcancerunited.org bloodcancerunited.org/blood-cancer-care/adults/types-blood-cancer-treatment/immunotherapy/chimeric-antigen-receptor-car-t-cell-therapy

Chimeric antigen receptor (CAR) T-cell therapy Autologous CAR T-cell immunotherapy uses a person's own immune cells (T cells) to identify and attack cancer cells.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11049267/

Chimeric Antigen Receptor (CAR) T-Cell Therapy in ... CAR T-cell therapy has become a powerful treatment option in B-cell and plasma cell malignancies, and many patients have benefited from its use.

3. nature.com nature.com/articles/s41392-025-02269-w

CAR-T cell therapy for cancer: current challenges and ... This review begins with a comprehensive overview of CAR-T cell therapy for cancer, covering the structure of CAR-T cells and the history of their clinical ...

4. cancer.gov cancer.gov/about-cancer/treatment/research/car-t-cells

CAR T Cells: Engineering Immune Cells to Treat Cancer CAR T-cell therapy involves genetically engineering a patient's own T cells (red) to attack cancer cells (red and blue).

5. gilead.com gilead.com/news/news-details/2025/gilead-and-kite-showcase-continued-progress-in-transforming-blood-cancer-care-with-new-cell-therapy-data-at-ash-2025

Gilead and Kite Showcase Continued Progress in ... These data showcase Kite's continued progress in transforming blood cancer care and expanding the reach and impact of CAR T-cell therapy. “Kite ...

6. mayoclinic.org mayoclinic.org/tests-procedures/car-t-cell-therapy/about/pac-20585020

CAR-T cell therapy CAR-T cell therapy is a cancer treatment that takes cells from the body and genetically changes them so they can fight cancer.

7. cancer.org cancer.org/cancer/managing-cancer/treatment-types/immunotherapy/car-t-cell.html

CAR T-cell Therapy and Its Side Effects CAR T-cell therapy is a personalized form of immunotherapy that trains your own immune cells to recognize and destroy cancer.