CLINICAL TRIALS PROFILE FOR TASIGNA

All Clinical Trials for TASIGNA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00036738 ↗	Fludarabine Phosphate and Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia or Chronic Myelogenous Leukemia That Has Responded to Treatment With Imatinib Mesylate, D	Completed	National Cancer Institute (NCI)	Phase 2	2001-07-13	This phase II trial is studying how well fludarabine phosphate and total-body irradiation followed by donor peripheral blood stem cell transplant work in treating patients with acute lymphoblastic leukemia or chronic myelogenous leukemia that has responded to previous treatment with imatinib mesylate, dasatinib, or nilotinib. Giving low doses of chemotherapy, such as fludarabine phosphate, and total-body irradiation (TBI) before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) after the transplant may help increase this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving mycophenolate mofetil and cyclosporine after the transplant may stop this from happening.
NCT00036738 ↗	Fludarabine Phosphate and Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia or Chronic Myelogenous Leukemia That Has Responded to Treatment With Imatinib Mesylate, D	Completed	Fred Hutchinson Cancer Research Center	Phase 2	2001-07-13	This phase II trial is studying how well fludarabine phosphate and total-body irradiation followed by donor peripheral blood stem cell transplant work in treating patients with acute lymphoblastic leukemia or chronic myelogenous leukemia that has responded to previous treatment with imatinib mesylate, dasatinib, or nilotinib. Giving low doses of chemotherapy, such as fludarabine phosphate, and total-body irradiation (TBI) before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) after the transplant may help increase this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving mycophenolate mofetil and cyclosporine after the transplant may stop this from happening.
NCT00644878 ↗	Study of Molecular Response in Adult Patients on Nilotinib With Philadelphia Chromosome Positive Chronic Myelogenous Leukemia (Ph+ CML) in Chronic Phase and a Suboptimal Molecular Response to Imatinib	Terminated	Novartis Pharmaceuticals	Phase 2	2008-10-01	This exploratory study will evaluate the change in molecular response in chronic myelogenous leukemia - chronic phase patients with a complete cytogenetic response and have a suboptimal molecular response to imatinib
NCT00702403 ↗	Nilotinib and Imatinib Mesylate After Donor Stem Cell Transplant in Treating Patients With ALL or CML	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2008-08-14	This phase I/II trial is studying the side effects and best way to give nilotinib when given alone or sequentially after imatinib mesylate after donor stem cell transplant in treating patients with acute lymphoblastic leukemia or chronic myelogenous leukemia. Nilotinib and imatinib mesylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
NCT00702403 ↗	Nilotinib and Imatinib Mesylate After Donor Stem Cell Transplant in Treating Patients With ALL or CML	Completed	Fred Hutchinson Cancer Research Center	Phase 1/Phase 2	2008-08-14	This phase I/II trial is studying the side effects and best way to give nilotinib when given alone or sequentially after imatinib mesylate after donor stem cell transplant in treating patients with acute lymphoblastic leukemia or chronic myelogenous leukemia. Nilotinib and imatinib mesylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
NCT00732888 ↗	Effect of Calcium on Tasigna Pharmacokinetics (PK) in Healthy Volunteers	Completed	Novartis Pharmaceuticals	Phase 1	2008-11-01	This research study is being conducted through the University of Pittsburgh Cancer Institute (UPCI). It will evaluate the concentrations of Tasigna®, an oral drug used to treat some types of cancer, in the blood of healthy volunteers when taken with and without Tums Ultra®, a calcium product often used in the treatment of upset stomach and as a calcium supplement.
NCT00732888 ↗	Effect of Calcium on Tasigna Pharmacokinetics (PK) in Healthy Volunteers	Completed	Jan Beumer	Phase 1	2008-11-01	This research study is being conducted through the University of Pittsburgh Cancer Institute (UPCI). It will evaluate the concentrations of Tasigna®, an oral drug used to treat some types of cancer, in the blood of healthy volunteers when taken with and without Tums Ultra®, a calcium product often used in the treatment of upset stomach and as a calcium supplement.

Clinical Trial Conditions for TASIGNA

Condition Name

Condition Name for TASIGNA
Intervention	Trials
Chronic Myeloid Leukemia	10
Leukemia	4
Chronic Phase Chronic Myeloid Leukemia	3
Recurrent Adult Acute Lymphoblastic Leukemia	3

Condition MeSH

Condition MeSH for TASIGNA
Intervention	Trials
Leukemia	33
Leukemia, Myelogenous, Chronic, BCR-ABL Positive	32
Leukemia, Myeloid	31
Leukemia, Myeloid, Chronic-Phase	10

Clinical Trial Locations for TASIGNA

Trials by Country

Trials by Country for TASIGNA
Location	Trials
United States	165
Brazil	20
Spain	10
Germany	9
Korea, Republic of	9

Trials by US State

Trials by US State for TASIGNA
Location	Trials
Texas	11
California	10
New York	9
North Carolina	7
Washington	7

Clinical Trial Progress for TASIGNA

Clinical Trial Phase

Clinical Trial Phase for TASIGNA
Clinical Trial Phase	Trials
PHASE1	1
Phase 4	3
Phase 3	10

Clinical Trial Status

Clinical Trial Status for TASIGNA
Clinical Trial Phase	Trials
Completed	32
Terminated	13
Recruiting	11

Clinical Trial Sponsors for TASIGNA

Sponsor Name

Sponsor Name for TASIGNA
Sponsor	Trials
Novartis Pharmaceuticals	15
Novartis	11
National Cancer Institute (NCI)	10

Sponsor Type

Sponsor Type for TASIGNA
Sponsor	Trials
Other	83
Industry	48
NIH	11

Clinical Trials Update, Market Analysis, and Projections for TASIGNA (Nilotinib)

Last updated: October 26, 2025

Introduction

TASIGNA (nilotinib) is an oral tyrosine kinase inhibitor (TKI) developed by Novartis for the treatment of chronic myeloid leukemia (CML) and Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). First approved by the U.S. Food and Drug Administration (FDA) in 2010, TASIGNA has since become a critical component in targeted cancer therapy. This report provides a comprehensive update on its ongoing clinical trials, analyzes current market dynamics, and projects future growth trajectories.

Clinical Trials Update

Recent and Ongoing Trials

Since its initial approval, TASIGNA has undergone extensive clinical evaluation. The landscape of clinical trials continues to evolve, focusing on expanding its indications, optimizing dosing regimens, and evaluating safety profiles.

• Expanded Indications in Ph+ Leukemias Recent trials explore TASIGNA's efficacy in cases resistant to first-line treatments. Notably, NCT03022227 investigates the safety and efficacy of nilotinib in pediatric patients with CML, reflecting efforts to broaden age-specific use.

• Combination Therapy Studies Several Phase II trials, such as NCT02927151, evaluate TASIGNA in combination with other agents like blinatumomab for Ph+ ALL, aiming to enhance remission rates and overcome resistance.

• Long-term Safety and Efficacy Ongoing open-label extension studies, including NCT01424983, monitor long-term outcomes and safety profiles in patients on TASIGNA for over five years, emphasizing its safety in chronic management.

Key Clinical Trial Outcomes

The accumulated data reinforce TASIGNA's role as a potent second-generation TKI with improved potency over imatinib and a favorable safety profile.

• Efficacy in Resistant Cases A pivotal Phase III trial (ENESTnd), demonstrated in 2012, established TASIGNA's superior molecular response rates compared to imatinib, particularly in resistant or intolerant patients.

• Safety Profile Common adverse events include cardiovascular events (notably arterial occlusive disease), elevated liver enzymes, and hematologic abnormalities. Long-term follow-up studies suggest manageable safety with appropriate monitoring.

Future Research Directions

Novartis continues to invest in trials targeting specific populations such as elderly patients, patients with T315I mutations (particularly resistant to current TKIs), and novel combination approaches, underpinning its commitment to expanding TASIGNA's clinical utility.

Market Analysis

Current Market Landscape

TASIGNA remains a prominent player in the TKI market, commanding a significant share in CML therapeutics. As of 2022, the global CML drug market was valued at approximately USD 3.2 billion, with TASIGNA accounting for around 35% of the market share among second-generation TKIs, trailing only behind generic and other branded drugs like imatinib and bosutinib.

Competitive Dynamics

• Key Competitors

  • Imatinib (Gleevec): First-generation TKI, generics increasing price competition.
  • Dasatinib (Spry cel) and Bosutinib (Bosulif): Other second-generation TKIs offering similar efficacy.
  • Ponatinib (Iclusig): Targeted for T315I mutations, with a broader resistance profile but a distinct safety concern profile.

• Market Challenges

  • Generic Competition: The patent expiration of early TKIs threatens TASIGNA’s dominance.
  • Safety Concerns: Cardiovascular events may limit broader use, especially in high-risk populations.
  • Adherence Issues: Side-effect profiles may impact long-term compliance.

Market Opportunities

• Expanding Indications
  • Trials exploring TASIGNA in frontline therapy for CML could expand its usage and market share.
• Resistance Management
  • Addressing resistance mutations can position TASIGNA as a targeted solution for difficult-to-treat patients.
• Geographical Expansion
  • Increasing approvals in emerging markets such as Asia-Pacific and Latin America, which are witnessing rising CML prevalence.

Market Projections

Analysts project the global CML drug market to grow at a compound annual growth rate (CAGR) of approximately 8% over the next five years, driven by increasing diagnosis rates, improved detection, and newer treatment protocols.

• Revenue Forecast (2023–2028)
  • Moderate Optimism: Due to competition and market saturation, TASIGNA's sales are expected to stabilize around USD 1.2–1.5 billion annually by 2028.
  • Impact of Expanded Indications: Future approval for first-line therapy could add an incremental USD 200–300 million annually.
• Market Penetration Strategies
  • Collaborations with regional health authorities.
  • Incorporation into combination therapy regimens.
  • Focused marketing on safety profile and long-term outcomes.

Strategic Outlook

Novartis is strategically leveraging ongoing clinical trials and regulatory pathways to reinforce TASIGNA’s market positioning, emphasizing personalized treatment approaches and comprehensive safety data to mitigate risks associated with adverse events.

Conclusion

TASIGNA continues to demonstrate robust clinical activity, particularly for resistant CML cases, with ongoing trials poised to extend its application scope. Market-wise, it grapples with generic competition but maintains significant influence through its efficacy profile and clinical flexibility. Strategic initiatives—focused on expanding indications, geographic penetration, and combination therapies—are essential to sustain its growth trajectory.

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Key Takeaways

• Ongoing clinical trials reinforce TASIGNA's efficacy in resistant and pediatric populations, alongside potential for first-line therapy expansion.
• The drug maintains a leading market position but faces competitive threats from generics and safety concerns.
• Market growth will depend on regulatory approvals, management of adverse events, and successful diversification into new indications.
• Strategic collaborations and real-world evidence generation are crucial for long-term market sustainability.
• The emerging focus on resistance mutations and combination therapies presents opportunities for TASIGNA’s expanded utilization.

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FAQs

1. What are the primary clinical benefits of TASIGNA over earlier-generation TKIs?
TASIGNA exhibits higher potency, faster molecular responses, and efficacy in patients refractory to imatinib, especially those with certain resistance mutations like T315I.

2. Are there significant safety concerns associated with TASIGNA?
Yes, cardiovascular events and elevated liver enzymes are notable. Proper patient screening and monitoring are vital to mitigate these risks.

3. How does TASIGNA fare in the pediatric CML population?
Clinical trials like NCT03022227 indicate promising safety and efficacy, paving the way for potential label expansion to pediatric use.

4. What strategies can extend TASIGNA’s market share amidst growing competition?
Expanding indications, forming strategic partnerships, optimizing safety management, and demonstrating long-term benefits through real-world data are key.

5. What is the outlook for TASIGNA’s future growth in the global market?
While challenges persist, ongoing clinical trials and market expansion efforts suggest a steady growth trajectory, with projected revenues stabilizing around USD 1.2–1.5 billion annually by 2028.

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Sources:
[1] FDA approval documentation, 2010.
[2] ClinicalTrials.gov database.
[3] Novartis annual reports.
[4] Market research reports, 2022.
[5] Recent peer-reviewed studies on TASIGNA efficacy and safety.