CLINICAL TRIALS PROFILE FOR TISAGENLECLEUCEL

All Clinical Trials for tisagenlecleucel

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	Intrexon Corporation	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	Ziopharm	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	Ziopharm Oncology	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT02445248 ↗	Study of Efficacy and Safety of CTL019 in Adult DLBCL Patients	Active, not recruiting	Novartis Pharmaceuticals	Phase 2	2015-07-29	This is a multi-center, phase II study to determine the efficacy and safety of CTL019 in adult patients with relapsed or refractory DLBCL.

Clinical Trial Conditions for tisagenlecleucel

Condition Name

Condition Name for tisagenlecleucel
Intervention	Trials
Diffuse Large B-Cell Lymphoma	3
Non-Hodgkin Lymphoma	3
Recurrent Diffuse Large B-Cell Lymphoma	3
Acute Lymphoblastic Leukemia	3

Condition MeSH

Condition MeSH for tisagenlecleucel
Intervention	Trials
Lymphoma	12
Lymphoma, B-Cell	10
Lymphoma, Large B-Cell, Diffuse	7
Leukemia, Lymphoid	4

Clinical Trial Locations for tisagenlecleucel

Trials by Country

+

−

Trials by Country for tisagenlecleucel
Location	Trials
United States	51
Japan	8
Germany	6
Australia	6
Spain	5

Trials by US State

+

−

Trials by US State for tisagenlecleucel
Location	Trials
Texas	4
Pennsylvania	4
Illinois	4
Georgia	4
California	4

Clinical Trial Progress for tisagenlecleucel

Clinical Trial Phase

Clinical Trial Phase for tisagenlecleucel
Clinical Trial Phase	Trials
Phase 3	3
Phase 2	4
Phase 1/Phase 2	2

Clinical Trial Status

Clinical Trial Status for tisagenlecleucel
Clinical Trial Phase	Trials
Recruiting	5
Active, not recruiting	5
Not yet recruiting	4

Clinical Trial Sponsors for tisagenlecleucel

Sponsor Name

Sponsor Name for tisagenlecleucel
Sponsor	Trials
Novartis Pharmaceuticals	7
National Cancer Institute (NCI)	3
Masonic Cancer Center, University of Minnesota	2

Sponsor Type

Sponsor Type for tisagenlecleucel
Sponsor	Trials
Industry	13
Other	10
NIH	3

Tisagenlecleucel: A Comprehensive Overview of Clinical Trials, Market Analysis, and Projections

Introduction

Tisagenlecleucel, marketed as Kymriah, is a groundbreaking chimeric antigen receptor (CAR) T-cell therapy that has revolutionized the treatment of certain types of blood cancers. This article delves into the latest updates from clinical trials, market analysis, and future projections for this innovative drug.

Clinical Trial Updates

Efficacy in Pediatric and Young Adult Patients

In the global phase II ELIANA trial, tisagenlecleucel demonstrated an overall remission rate of 82% in pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). With a median follow-up of 38.8 months, the median event-free survival was 24 months, and the median overall survival was not reached. Notably, 44% of patients remained event-free, and 63% were alive at 3 years, indicating favorable long-term outcomes[1].

Durable Responses in Adults

The ELARA trial, focusing on adults with relapsed/refractory follicular lymphoma (r/r FL), reported high response rates and a favorable safety profile. After a median follow-up of 29 months, the trial showed a complete response rate of 68.1% and an overall response rate of 86.2%. No new safety signals or treatment-related deaths were observed, highlighting the durable efficacy and safety of tisagenlecleucel in this patient population[5].

Safety Profile

Long-term follow-up data from clinical trials indicate that tisagenlecleucel has a favorable safety profile. In pediatric and young adult patients with R/R B-ALL, no new or unexpected long-term adverse events were reported, and grade 3/4 adverse events were limited to 29% of patients more than a year after infusion[1]. Similarly, in adults with r/r FL, the incidence of grade ≥3 cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome was ≤1%[5].

Market Analysis

Current Market Size and Growth Projections

The global CAR T-cell therapy market, led by tisagenlecleucel, is experiencing rapid growth. As of 2023, the market was valued at USD 9.11 billion and is projected to reach USD 127.53 billion by 2033. Tisagenlecleucel is anticipated to exhibit the highest growth rate within this market due to its pioneering status, clinical efficacy, and ongoing research initiatives aimed at expanding its therapeutic applications[2].

Competitive Landscape

Tisagenlecleucel, as the first FDA-approved CAR T-cell therapy, holds a significant position in the market. Its approval for adults with certain types of B-cell non-Hodgkin lymphoma and young individuals with B-cell acute lymphoblastic leukemia has set a precedent for other CAR T-cell therapies. The drug's continued success in clinical trials and its potential application in other cancer types further solidify its market dominance[2].

Cost-Effectiveness Analysis

Economic Value Assessment

A cost-utility analysis comparing tisagenlecleucel with standard care for pediatric patients with acute lymphoblastic leukemia highlighted the drug's economic value. The study showed that tisagenlecleucel's cost-effectiveness would be significantly improved with a long-term cure rate of over 40% or a price discount of 49%. At a willingness-to-pay threshold of $150,000 per quality-adjusted life-year (QALY), tisagenlecleucel had a 32% likelihood of being cost-effective[3].

Long-Term Survival and Quality of Life

The analysis also considered long-term survival and quality of life. Patients treated with tisagenlecleucel reported improvements in quality of life up to 36 months after infusion. This, combined with the drug's ability to provide long-term survival benefits, underscores its value in terms of both clinical efficacy and patient quality of life[1][3].

Future Projections and Research Initiatives

Expanding Therapeutic Footprint

Tisagenlecleucel is being studied for its potential application in various malignancies, including acute myeloid leukemia, chronic lymphocytic leukemia, Hodgkin lymphoma, and solid tumors. Although its effectiveness for these indications has not yet been established, ongoing research initiatives are aimed at expanding its therapeutic footprint[4].

Addressing Unmet Needs

The drug's ability to induce durable responses in patients with high-risk baseline disease characteristics, such as those with bulky disease or high tumor burden, positions it as a critical treatment option for patients with limited therapeutic alternatives. Future trials will continue to explore its efficacy in these challenging patient populations[5].

Key Takeaways

• Clinical Efficacy: Tisagenlecleucel has demonstrated high remission rates and durable responses in both pediatric and adult patients with various types of blood cancers.
• Safety Profile: The drug has a favorable long-term safety profile with minimal new or unexpected adverse events.
• Market Growth: The global CAR T-cell therapy market, led by tisagenlecleucel, is projected to grow significantly, reaching USD 127.53 billion by 2033.
• Cost-Effectiveness: Tisagenlecleucel's economic value is enhanced by its potential for long-term cure rates and improvements in patient quality of life.
• Future Research: Ongoing studies aim to expand the drug's therapeutic applications to other types of cancers.

FAQs

What is tisagenlecleucel, and how does it work?

Tisagenlecleucel is a CAR T-cell therapy that involves genetically modifying a patient's T cells to recognize and attack cancer cells. It is approved for the treatment of certain types of B-cell non-Hodgkin lymphoma and B-cell acute lymphoblastic leukemia.

What are the key clinical trial findings for tisagenlecleucel?

Clinical trials have shown high remission rates and durable responses in both pediatric and adult patients. For example, the ELIANA trial reported an overall remission rate of 82% in pediatric and young adult patients with R/R B-ALL, while the ELARA trial showed a complete response rate of 68.1% in adults with r/r FL.

How is the cost-effectiveness of tisagenlecleucel assessed?

The cost-effectiveness of tisagenlecleucel is assessed through cost-utility analyses that compare it with standard care. The drug's cost-effectiveness improves with higher long-term cure rates or significant price discounts.

What are the potential future applications of tisagenlecleucel?

Tisagenlecleucel is being studied for its potential use in various other malignancies, including acute myeloid leukemia, chronic lymphocytic leukemia, and solid tumors, although its effectiveness for these indications has not yet been established.

What is the current market size and growth projection for the CAR T-cell therapy market?

The global CAR T-cell therapy market was valued at USD 9.11 billion in 2023 and is projected to reach USD 127.53 billion by 2033, with tisagenlecleucel expected to exhibit the highest growth rate within this market.

What are the common adverse events associated with tisagenlecleucel?

Common adverse events include cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome, although these are generally mild and occur in a small percentage of patients.

Sources

1. Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients with Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia. PubMed.
2. CAR T-Cell Therapy Market Size to Hit USD 127.53 Billion by 2033. Biospace.
3. Cost-effectiveness of Tisagenlecleucel vs Standard Care in Acute Lymphoblastic Leukemia. JAMA Oncology.
4. Tisagenlecleucel (Kymriah) - Medical Clinical Policy Bulletins. Aetna.
5. Durable Response After Tisagenlecleucel in Adults with Relapsed/Refractory Follicular Lymphoma: ELARA Trial Update. Blood.