CLINICAL TRIALS PROFILE FOR TISAGENLECLEUCEL

All Clinical Trials for tisagenlecleucel

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
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.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	M.D. Anderson Cancer Center	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.
NCT03570892 ↗	Tisagenlecleucel in Adult Patients With Aggressive B-cell Non-Hodgkin Lymphoma	Active, not recruiting	Novartis Pharmaceuticals	Phase 3	2019-05-07	This is a randomized, open label, multicenter phase III trial comparing the efficacy, safety, and tolerability of tisagenlecleucel to Standard Of Care in adult patients with aggressive B-cell Non-Hodgkin Lymphoma after failure of rituximab and anthracycline containing frontline immunochemotherapy.

Clinical Trial Conditions for tisagenlecleucel

Condition Name

Condition Name for tisagenlecleucel
Intervention	Trials
Acute Lymphoblastic Leukemia	4
Refractory Diffuse Large B-cell Lymphoma	3
Diffuse Large B-Cell Lymphoma	3
Non-Hodgkin Lymphoma	3

Condition MeSH

Condition MeSH for tisagenlecleucel
Intervention	Trials
Lymphoma	12
Lymphoma, B-Cell	10
Lymphoma, Large B-Cell, Diffuse	8
Precursor Cell Lymphoblastic Leukemia-Lymphoma	5

Clinical Trial Locations for tisagenlecleucel

Trials by Country

Trials by Country for tisagenlecleucel
Location	Trials
United States	54
Japan	8
Australia	6
Germany	6
Spain	5

Trials by US State

Trials by US State for tisagenlecleucel
Location	Trials
California	5
Texas	4
Pennsylvania	4
Illinois	4
Georgia	4

Clinical Trial Progress for tisagenlecleucel

Clinical Trial Phase

Clinical Trial Phase for tisagenlecleucel
Clinical Trial Phase	Trials
PHASE3	1
PHASE1	2
Phase 3	3

Clinical Trial Status

Clinical Trial Status for tisagenlecleucel
Clinical Trial Phase	Trials
Recruiting	7
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
Other	14
Industry	13
NIH	3

Tisagenlecleucel: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: November 1, 2025

Introduction

Tisagenlecleucel, commercially known as Kymriah, represents a groundbreaking advance in immunotherapy—specifically chimeric antigen receptor T-cell (CAR-T) therapy—targeting hematologic malignancies. Approved by the FDA in 2017 for certain relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) and later for diffuse large B-cell lymphoma (DLBCL), it signifies a milestone in personalized cancer treatment. This comprehensive review synthesizes recent clinical trials, analyzes the market landscape, and projects future growth trajectories for tisagenlecleucel over the coming years.

Clinical Trials: Updates and Developments

Recent and Ongoing Clinical Trials

Tisagenlecleucel continues to be a focal point of innovative clinical studies aimed at expanding its indications and optimizing safety and efficacy. As of 2023, several pivotal and exploratory trials are underway:

• Pivotal Trials for New Indications:
  The JULIET trial (NCT02445248) assessed its efficacy in R/R DLBCL, leading to FDA approval for DLBCL in 2018. Ongoing follow-up studies aim to assess durability and long-term remission rates.

• Investigational Pediatric and Adult Indications:
  The ELIANA trial remains a foundational study for pediatric and young adult ALL, with long-term follow-up data demonstrating sustained remissions in a subset of patients. Additional trials are investigating its use in other B-cell malignancies, including mantle cell lymphoma and multiple myeloma, though these are in early stages.

• Combination and Sequencing Strategies:
  Current research explores combining tisagenlecleucel with checkpoint inhibitors or other immunomodulatory agents to mitigate relapse. Trials like the CALM study (NCT04504709) explore combination therapies to enhance CAR-T cell persistence.

• Safety and Toxicity Management:
  Studies such as the JULIET follow-up focus on cytokine release syndrome (CRS) and neurotoxicity management. Innovative approaches, including in vivo cytokine blockade, are under clinical validation.

Clinical Efficacy and Outcomes

Data from pivotal trials highlight robust response rates:

• In pediatric ALL, complete remission (CR) rates exceed 80%, with durable remissions observed up to five years post-treatment [1].
• R/R DLBCL responds with high initial remission rates (~50-60%), though durability varies, emphasizing ongoing research into resistance mechanisms [2].

Safety Profile and Adverse Events

While effective, tisagenlecleucel's toxicity profile warrants caution:

• CRS, neurotoxicity, and cytopenias are common. Advances in management protocols, including corticosteroids and tocilizumab, have improved safety outcomes [3].
• Ongoing trials aim to refine patient selection and preconditioning regimens to mitigate adverse events.

Market Analysis

Current Market Landscape

The global CAR-T therapy market, valued at approximately USD 3.2 billion in 2022, is expected to grow at a compound annual growth rate (CAGR) of around 25% through 2030 [4]. Tisagenlecleucel holds a significant share, driven by:

• Its first-mover advantage post-FDA approval.
• Approval for pediatric ALL and DLBCL, two high-prevalence blood cancers.
• Expansion into new indications, fueling increasing sales.

Key Competitors and Competitive Dynamics

Tisagenlecleucel's main competitors include:

• Lisocabtagene maraleucel (Breyanzi): Approved for DLBCL and related disorders.
• Axicabtagene ciloleucel (Yescarta): Another leading CAR-T therapy targeting similar indications.
• Cilta-cel (Carvykti): Approved in late 2022 for multiple myeloma, represent competition in broader hematologic malignancies.

Despite competitive pressures, tisagenlecleucel benefits from a well-established manufacturing process and clinical data, cementing its market position.

Market Challenges

• High Treatment Costs: The therapy's price exceeds USD 400,000 per treatment, restricting access in some markets.
• Manufacturing Complexities: Personalized cell manufacturing delays and logistical hurdles affect supply and scalability.
• Safety Concerns: Adverse events impose additional treatment burdens and influence payer decisions.

Market Opportunities

• Expanded indications in other B-cell malignancies and autoimmune disorders.
• Adoption in outpatient settings as safety profiles improve.
• Innovations in manufacturing, such as 'off-the-shelf' allogeneic CAR-T cells, may complement or challenge tisagenlecleucel's market dominance.

Future Projections

Market Growth Forecast

The CAR-T therapy market, with tisagenlecleucel as a leading product, is poised for exponential growth:

• 2023-2030: Estimated to reach USD 12-15 billion globally, driven by increased adoption, new indications, and improved manufacturing processes.
• Regional Dynamics: North America continues to lead due to established healthcare infrastructure and reimbursement policies, with Asia-Pacific experiencing rapid growth owing to increasing cancer prevalence and evolving regulatory approval pathways.

Regulatory and Scientific Outlook

• Regulatory agencies worldwide are increasingly supportive of CAR-T therapies, including conditional approvals and accelerated pathways.
• Ongoing trials aim to broaden indications to solid tumors—a frontier that, if successful, could radically alter market dynamics.
• Advances in gene-editing and scalable manufacturing may substantially reduce costs and improve access.

Innovation and Next-Generation CAR-Ts

Research into allogeneic, off-the-shelf CAR-T products, such as AlloCAR and others, proposes solutions to current limitations, including manufacturing delays. These could either complement or compete with tisagenlecleucel.

Conclusion

Tisagenlecleucel remains a cornerstone of CAR-T therapy, with ongoing clinical trials advocating for expanded use and improved safety. The evolving competitive landscape, combined with manufacturing innovations and regulatory support, portends robust market growth. Its current and projected performance underscores the importance for industry stakeholders to strategically navigate clinical development, regulatory pathways, and reimbursement frameworks to maximize impact and profitability.

---

Key Takeaways

• Clinical efficacy: Tisagenlecleucel delivers high remission rates in pediatric ALL and R/R DLBCL, with durability promising longer-term disease control.
• Evolving landscape: Multiple ongoing trials aim to expand indications and improve safety profiles, particularly in combination therapies.
• Market strength: As a first-mover in CAR-T, tisagenlecleucel holds a significant share, maintained by efficacy, safety, and ongoing developments.
• Growth drivers: Increasing adoption, new markets, and manufacturing advancements are key to sustaining and accelerating growth.
• Challenges: Cost, manufacturing complexities, and safety management restent hurdles that require innovative solutions.

FAQs

1. What are the primary indications for tisagenlecleucel today?
Tisagenlecleucel is approved for pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) and for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

2. How does tisagenlecleucel compare with other CAR-T therapies?
While similar in mechanism, tisagenlecleucel differs in manufacturing process, cost, safety profile, and approved indications. Its early market entry gives it an advantage, but competitors like Yescarta and Breyanzi have expanded options for clinicians.

3. What are the main safety concerns associated with tisagenlecleucel?
Cytokine release syndrome (CRS) and neurotoxicity are notable adverse events. Advances in management protocols and patient selection are reducing these risks.

4. What future indications might extend the use of tisagenlecleucel?
Potential expansion includes other B-cell malignancies such as mantle cell lymphoma, multiple myeloma, and possibly autoimmune diseases, pending successful trials.

5. How will manufacturing innovations impact the market for tisagenlecleucel?
Progress in scalable, off-the-shelf CAR-T products may challenge autologous therapies like tisagenlecleucel but could also create new market segments through improved accessibility and cost-effectiveness.

---

References

[1] Maude, S. L., et al. (2018). Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. The New England Journal of Medicine, 378(5), 439–448.
[2] Schuster, S. J., et al. (2019). Tisagenlecleucel in Adult Relapsed or Refractory Diffuse Large B-Cell Lymphoma. The New England Journal of Medicine, 380(1), 45–56.
[3] Lee, D. W., et al. (2019). Current Management of Cytokine Release Syndrome after T-cell–Engaging Immunotherapies. Nature Reviews Clinical Oncology, 16(8), 471–487.
[4] Grand View Research. (2023). CAR-T Cell Therapy Market Size, Share & Trends Analysis Report.