Long-term Follow-up for Cilta-cel in Multiple Myeloma

Recruiting at 63 trial locations

Overseen By Study Contact

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Janssen Research & Development, LLC

Disqualifiers: Not received cilta-cel, 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

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

Compensation: Varies

Number of Visits: 2

295 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to gather long-term safety data on cilta-cel, a treatment for multiple myeloma, a type of blood cancer. The focus is on understanding any delayed side effects that might appear after receiving the treatment. Participants will not receive any new treatment in this trial but will check in at least once a year for up to 15 years. The trial seeks individuals who have already received cilta-cel in a previous company-sponsored study and have agreed to join this follow-up study. As a Phase 4 trial, cilta-cel is already FDA-approved and proven effective, and this research helps to understand how the treatment benefits more patients.

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications.

What is the safety track record for cilta-cel?

Research has shown that ciltacabtagene autoleucel, or cilta-cel, demonstrates promising safety results from earlier studies. For example, in past trials, one-third of patients who received a single dose of cilta-cel did not experience disease progression for at least five years. This suggests the treatment can be safe over a long period.

Additionally, cilta-cel is approved for treating multiple myeloma, a type of blood cancer, indicating it meets certain safety standards for this condition. However, like any treatment, side effects can occur, and ongoing studies are examining any negative effects that might appear later.

In summary, while cilta-cel is generally well-tolerated, patients should be aware of possible long-term effects. Discussing any concerns with healthcare providers before joining a trial is important. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Cilta-cel is unique because it represents a new frontier in multiple myeloma treatment with its CAR-T cell therapy approach. Unlike traditional treatments like chemotherapy and proteasome inhibitors, cilta-cel harnesses the patient's own immune cells, reprogramming them to target and destroy cancer cells with precision. Researchers are excited because this method potentially offers long-lasting remissions and is tailored to each individual, which could mean fewer side effects and more effective targeting of the cancer compared to existing options.

What is the effectiveness track record for cilta-cel in treating multiple myeloma?

Research has shown that ciltacabtagene autoleucel (cilta-cel) effectively treats multiple myeloma, a type of blood cancer. One study found that about one-third of patients who received cilta-cel did not experience cancer progression for five years or more. This is significant for patients with relapsed or refractory multiple myeloma, who have already tried many other treatments. The treatment involves a single infusion and has demonstrated long-lasting remission, allowing patients an extended break from their disease. Cilta-cel is already approved for use, confirming its effectiveness in treating this condition. Participants in this long-term follow-up study will not receive additional treatment but will be monitored for delayed adverse events for up to 15 years after their last dose of cilta-cel. ^{1 3 5 6 7}

Who Is on the Research Team?

Janssen Research & Development, LLC Clinical Trial

Principal Investigator

Janssen Research & Development, LLC

Are You a Good Fit for This Trial?

This trial is for individuals who have previously received at least one dose of ciltacabtagene autoleucel (cilta-cel) in a company-sponsored study for multiple myeloma and have consented to participate in this long-term follow-up.

Inclusion Criteria

Participants who have provided informed consent for this stud

You have previously taken cilta-cel in a study sponsored by the company.

Participants who have provided informed consent for this study

Timeline for a Trial Participant

Initial Follow-up

Participants are monitored for safety and effectiveness after treatment with cilta-cel, focusing on new incidences of serious adverse events and infections.

5 years

At least 1 visit per year

Extended Follow-up

Participants continue to be monitored for long-term safety, including serious hematologic disorders and adverse events.

10 years

At least 1 visit per year

Long-term Safety Monitoring

Participants are assessed for delayed adverse events and long-term safety profile of cilta-cel, including monitoring for new malignancies and autoimmune disorders.

15 years

What Are the Treatments Tested in This Trial?

Interventions

• Cilta-cel

Trial Overview

The study focuses on collecting data over an extended period to monitor delayed side effects and understand the long-term safety of cilta-cel, which is used to treat multiple myeloma.

How Is the Trial Designed?

Group I: Cilta-cel Experimental Treatment 1 Intervention

Participants who had previously received treatment with cilta-cel in a Company-sponsored clinical study (example, NCT04923893, NCT03758417, NCT04181827, NCT05347485, NCT04133636, and NCT03548207) in the global development program will be enrolled into this study once the individual's participation in the particular interventional study has ended or a study has been terminated. Participants will not receive any treatment in this study and will be followed-up at least once per year on delayed adverse events for up to 15 years after receiving the last dose of cilta-cel.

Who Is Running the Clinical Trial?

Janssen Research & Development, LLC

Lead Sponsor

Trials

1,022

Recruited

6,408,000+

Joaquin Duato

Janssen Research & Development, LLC

Chief Executive Officer since 2022

MBA from ESADE, Master of International Management from Thunderbird School of Global Management

Dr. Jijo James, MD

Janssen Research & Development, LLC

Chief Medical Officer since 2014

MD from St. Johns Medical College, MPH from Columbia University

Published Research Related to This Trial

Ciltacabtagene autoleucel (cilta-cel) is a CAR T-cell therapy that effectively targets B-cell maturation antigen (BCMA) in patients with relapsed or refractory multiple myeloma, showing early and long-lasting responses based on the Phase 1b/2 CARTITUDE-1 study.

While cilta-cel has a tolerable safety profile with manageable adverse effects, it generally demonstrates better efficacy compared to another CAR T-cell therapy, idecabtagene vicleucel (ide-cel), although both therapies have comparable adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ciltacabtagene autoleucel: The second anti-BCMA CAR T-cell therapeutic armamentarium of relapsed or refractory multiple myeloma. Chekol Abebe, E., Yibeltal Shiferaw, M., Tadele Admasu, F., et al.[2022]

Ciltacabtagene autoleucel (cilta-cel) has been approved by the FDA as a fifth-line treatment option for patients with relapsed or refractory multiple myeloma, marking a significant advancement in therapy options for this challenging condition.

Cilta-cel is notable for being the second chimeric antigen receptor T-cell therapy targeting BCMA to receive regulatory approval within a year, highlighting the rapid development of innovative treatments in this area.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cilta-cel OK'd for Multiple Myeloma. [2022]

Ciltacabtagene autoleucel (cilta-cel) demonstrated significantly better efficacy compared to physician's choice of treatment in patients with relapsed or refractory multiple myeloma, with a relative risk of 2.95 for overall response rate and 111.70 for complete response or better.

The study showed that cilta-cel also improved progression-free survival and overall survival, with hazard ratios of 0.24 and 0.21 respectively, indicating it could be a promising new treatment option for patients who have already been exposed to multiple therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparative Efficacy of Ciltacabtagene Autoleucel in CARTITUDE-1 vs Physician's Choice of Therapy in the Long-Term Follow-Up of POLLUX, CASTOR, and EQUULEUS Clinical Trials for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma. Weisel, K., Martin, T., Krishnan, A., et al.[2022]

In a study of 97 heavily pretreated patients with relapsed/refractory multiple myeloma, ciltacabtagene autoleucel (cilta-cel) demonstrated an impressive overall response rate of 97.9% and a stringent complete response rate of 82.5% after approximately 28 months of follow-up.

The treatment maintained durable responses across various patient subgroups, including those with high-risk features, while the safety profile remained manageable, with no new cases of cytokine release syndrome reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ciltacabtagene Autoleucel, an Anti-B-cell Maturation Antigen Chimeric Antigen Receptor T-Cell Therapy, for Relapsed/Refractory Multiple Myeloma: CARTITUDE-1 2-Year Follow-Up. Martin, T., Usmani, SZ., Berdeja, JG., et al.[2023]

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]

Ciltacabtagene autoleucel (cilta-cel) demonstrated a significantly higher overall response rate (84% vs. 28%) and longer progression-free survival in patients with relapsed/refractory multiple myeloma compared to standard non-CAR-T therapies, based on a comparison of 113 patients in the CARTITUDE-1 study and 190 patients from the MAMMOTH dataset.

In the modified intent-to-treat population, cilta-cel showed an even more impressive overall response rate of 96% compared to 30% in the MAMMOTH cohort, indicating its potential as a highly effective treatment option for patients who have exhausted other therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of Cilta-cel, an Anti-BCMA CAR-T Cell Therapy, Versus Conventional Treatment in Patients With Relapsed/Refractory Multiple Myeloma. Costa, LJ., Lin, Y., Cornell, RF., et al.[2022]

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]

Citations

1.

jnj.com

jnj.com/media-center/press-releases/single-infusion-of-carvykti-ciltacabtagene-autoleucel-delivered-lasting-treatment-free-remissions-for-at-least-five-years-in-patients-with-relapsed-or-refractory-multiple-myeloma

Single infusion of CARVYKTI® (ciltacabtagene autoleucel) ...

New long-term CARTITUDE-1 data show one-third of patients treated with CARVYKTI® remain progression-free

2.

ascopubs.org

ascopubs.org/doi/10.1200/JCO-25-00760

Long-Term (≥5-Year) Remission and Survival After ...

CARTITUDE-1 evaluated ciltacabtagene autoleucel (cilta-cel) in patients with heavily pretreated relapsed/refractory multiple myeloma (RRMM).

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05201781

NCT05201781 | A Long-term Study for Participants ...

The purpose of this study is to collect long-term follow-up data on delayed adverse events after administration of ciltacabtagene autoleucel (cilta-cel).

4.

carvyktihcp.com

carvyktihcp.com/cartitude-4-efficacy/

CARTITUDE-4 Study - CARVYKTI® (ciltacabtagene autoleucel)

Discover the efficacy outcomes for the primary and follow-up analysis of the CARVYKTI® CARTITUDE-4 study, including study design, survival results, ...

5.

investors.legendbiotech.com

investors.legendbiotech.com/news-releases/news-release-details/legend-biotech-unveils-groundbreaking-5-year-survival-data

Legend Biotech Unveils Groundbreaking 5-Year Survival ...

An unprecedented 33% (32 of 97) of patients remained progression-free for five years or more. These data were featured in an oral presentation at the 2025 ...

6.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40459151/

Long-Term (≥5-Year) Remission and Survival After Treatment ...

One third (32/97) of patients remain alive and progression-free for ≥5 years after a single cilta-cel infusion, without maintenance treatment.

7.

ema.europa.eu

ema.europa.eu/en/medicines/human/EPAR/carvykti

Carvykti | European Medicines Agency (EMA)

A Post-authorization Safety Study to Evaluate the Safety of Multiple Myeloma Patients Treated with Ciltacabtagene Autoleucel - post-authorisation study · A ...