CLINICAL TRIALS PROFILE FOR NILOTINIB HYDROCHLORIDE

All Clinical Trials for nilotinib hydrochloride

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.
NCT00109707 ↗	A Study of Oral AMN107 in Adults With Chronic Myelogenous Leukemia (CML) or Other Hematologic Malignancies	Completed	Novartis Pharmaceuticals	Phase 1/Phase 2	2005-04-01	The purpose of this trial is to assess the efficacy, safety, tolerability, biologic activity, and pharmacokinetics of AMN107 in six groups of patients with one of the following conditions: Relapsed/refractory Ph+ Acute lymphoblastic leukemia (ALL) (arm 1) Group A - Imatinib failure only (arms 2, 3 and 4) - imatinib-resistant or intolerant CML - Chronic Phase (CP) - imatinib-resistant or intolerant CML - Accelerated Phase (AP) - imatinib-resistant or intolerant CML - Blast Crisis (BC) Group B - Imatinib and other TKI failure (arms 2, 3 and 4) - imatinib-resistant or intolerant CML - Chronic Phase (CP) - imatinib-resistant or intolerant CML - Accelerated Phase (AP) - imatinib-resistant or intolerant CML - Blast Crisis (BC) Hypereosinophilic syndrome/chronic eosinophilic leukemia (HES/CEL) (arm 5) Systemic mastocytosis (Sm) (arm 6)
NCT00129740 ↗	Phase II Nilotinib With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia (CML)	Completed	Novartis	Phase 2	2005-06-27	The goal of this clinical research study is to learn if an experimental agent, AMN107 (nilotinib), can help to control CML in chronic phase. The safety of this experimental agent will also be studied.
NCT00129740 ↗	Phase II Nilotinib With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia (CML)	Completed	M.D. Anderson Cancer Center	Phase 2	2005-06-27	The goal of this clinical research study is to learn if an experimental agent, AMN107 (nilotinib), can help to control CML in chronic phase. The safety of this experimental agent will also be studied.
NCT00384228 ↗	A Phase l/ll Study of AMN107 in Adult Patients With Glivec-intolerant CML or Relapsed-refractory Ph+ALL	Completed	Novartis Pharmaceuticals	Phase 1/Phase 2	2005-05-01	This study will investigate if nilotinib provides an improved safety and efficacy profile over that seen in patients receiving Imatinib.

Clinical Trial Conditions for nilotinib hydrochloride

Condition Name

Condition Name for nilotinib hydrochloride
Intervention	Trials
Chronic Myeloid Leukemia	41
Chronic Myelogenous Leukemia	17
Leukemia	11
Gastrointestinal Stromal Tumors	10

Condition MeSH

Condition MeSH for nilotinib hydrochloride
Intervention	Trials
Leukemia, Myelogenous, Chronic, BCR-ABL Positive	118
Leukemia	114
Leukemia, Myeloid	107
Philadelphia Chromosome	33

Clinical Trial Locations for nilotinib hydrochloride

Trials by Country

Trials by Country for nilotinib hydrochloride
Location	Trials
United States	449
Italy	219
Japan	104
Spain	103
France	67

Trials by US State

Trials by US State for nilotinib hydrochloride
Location	Trials
Texas	35
California	26
New York	24
Florida	21
Illinois	20

Clinical Trial Progress for nilotinib hydrochloride

Clinical Trial Phase

Clinical Trial Phase for nilotinib hydrochloride
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	3
Phase 4	17

Clinical Trial Status

Clinical Trial Status for nilotinib hydrochloride
Clinical Trial Phase	Trials
Completed	92
Recruiting	33
Terminated	24

Clinical Trial Sponsors for nilotinib hydrochloride

Sponsor Name

Sponsor Name for nilotinib hydrochloride
Sponsor	Trials
Novartis Pharmaceuticals	70
Novartis	20
National Cancer Institute (NCI)	16

Sponsor Type

Sponsor Type for nilotinib hydrochloride
Sponsor	Trials
Other	185
Industry	132
NIH	17

Nilotinib Hydrochloride: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

Introduction

Nilotinib Hydrochloride, marketed under the brand name Tasigna, is a second-generation tyrosine kinase inhibitor (TKI) primarily approved for treating chronic myeloid leukemia (CML). Since its initial FDA approval in 2007, Nilotinib has established itself as a significant therapeutic option, especially for patients resistant or intolerant to first-generation TKIs like imatinib. This report provides an in-depth analysis of recent clinical trial developments, a current market landscape assessment, and future projections for Nilotinib Hydrochloride.

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Clinical Trials Update

Recent Clinical Trial Developments

Over the past five years, Nilotinib's therapeutic profile has been the focus of numerous clinical trials, exploring expanded indications, long-term safety, and combination therapies.

1. Hematologic and Molecular Response in CML

Recent pivotal trials have focused on the long-term efficacy of Nilotinib in CML. Notably, the ENESTnd trial, a phase 3 study, demonstrated superior cytogenetic and molecular response rates compared to imatinib, with sustained responses over a decade [1]. Extended follow-ups validate its durable efficacy, solidifying its place in second-line therapy.

2. Treatment in Newly Diagnosed Patients

Emerging data from trials like ENESTnd emphasize favoring Nilotinib as a frontline therapy, especially considering its deep molecular responses. Ongoing phase 3 trials aim to compare Nilotinib with other novel therapies, with preliminary results indicating significant improvements in major molecular response (MMR) rates.

3. Expansion into Other Hematologic Malignancies

Recent phase 1/2 studies have investigated Nilotinib’s activity in advanced systemic mastocytosis and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). These trials suggest potential off-label uses, though regulatory approval remains pending [2].

4. Combination Therapy and Resistance Management

Trials exploring Nilotinib combined with agents like interferon-alpha and novel monoclonal antibodies are underway, aiming to improve response rates and prevent resistance. The Sokal and EUTOS scoring systems guide patient stratification in these studies.

Safety and Tolerability Updates

Long-term safety data indicate that Nilotinib maintains a manageable adverse event profile. Common issues include myelosuppression, hyperbilirubinemia, and cardiovascular events such as arterial occlusive disease. Recent studies emphasize the importance of cardiovascular risk monitoring, especially in older populations [3].

Regulatory and Labeling Changes

In recent years, regulatory agencies like the FDA and EMA have updated safety warnings and prescribing information to address risks associated with Nilotinib, particularly cardiovascular risks. These updates inform clinical decision-making and patient management strategies.

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Market Analysis

Market Size and Revenue

The global CML market was valued at approximately USD 3.2 billion in 2022 and is projected to reach USD 4.5 billion by 2030, growing at a Compound Annual Growth Rate (CAGR) of around 4.2% [4]. Nilotinib constitutes approximately 40% of the second-generation TKI segment, with its sales driven primarily by North American and European markets.

Key Market Drivers

• Efficacy in Resistant/Intolerant Patients: Nilotinib’s proven effectiveness in patients resistant or intolerant to imatinib sustains demand.
• First-Line Approval for CML: Increasing adoption of Nilotinib as a frontline therapy enhances sales.
• Pipeline Expansion Potential: Clinical trials exploring additional indications may expand its market footprint.

Competitive Landscape

Nilotinib faces competition from other second-generation TKIs such as dasatinib and bosutinib, which offer comparable efficacy profiles. Notably:

• Dasatinib (Sprycel): Approved for first- and second-line CML; has a broader efficacy spectrum [5].
• Bosutinib (Bosulif): Approved for treatment-resistant CML, with favorable safety profiles in certain populations [6].

Market positioning favors Nilotinib due to its documented deep molecular responses, but safety concerns necessitate careful patient selection.

Pricing and Reimbursement

In developed markets, Nilotinib's annual treatment costs range from USD 60,000 to USD 90,000. Reimbursement policies are aligned with drug efficacy and safety profiles, with coverage being comprehensive in major markets.

Emerging Trends and Market Challenges

• Generic Entry: Patent expiration is anticipated around 2027-2028, with biosimilar versions threatening brand-name sales.
• Safety Profile Concerns: Cardiovascular risks may impact prescribing patterns, prompting competitive shifts to agents with more favorable safety profiles.
• Patient Compliance: Oral administration convenience supports adherence, which is critical for chronic therapies.

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Market Outlook and Future Projections

Short- to Medium-Term Projections (2023-2028)

The Nilotinib market is expected to sustain steady growth driven by its established efficacy and expanded indication approvals. Adoption as a first-line agent will further solidify its market share, although growth rates may moderate due to competition and safety considerations.

Long-Term Strategic Outlook (2028 and beyond)

• Pipeline Progress and Label Expansion: Successful clinical trials for off-label indications (e.g., systemic mastocytosis, Ph+ ALL) could diversify revenue streams.
• Regulatory Approvals in Emerging Markets: Expansion into Asia-Pacific and Latin America is anticipated, leveraging increasing CML diagnosis rates.
• Biosimilar Competition: Patent expiry will likely catalyze competitive pricing and market share redistribution, compelling the brand to innovate in formulation and patient management.
• Technological Advancements: Integration with personalized medicine, including genomic profiling, may optimize patient selection, improving outcomes and market penetration.

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Conclusion

Nilotinib Hydrochloride remains a cornerstone second-generation TKI with substantial clinical evidence supporting its efficacy especially in resistant or newly diagnosed CML. Ongoing trials continue to explore its potential for other hematological malignancies. Market dynamics are influenced by safety concerns, emerging competitors, and patent landscapes. Positioning for sustainable growth hinges on demonstrating safety advantages, expanding indications, and managing the competitive cycle effectively.

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Key Takeaways

• Clinical Loop: Nilotinib exhibits durable efficacy in CML, with ongoing research exploring broader hematologic applications.
• Market Position: It holds a substantial market share within second-generation TKIs, supported by efficacy and regulatory approvals.
• Safety Profile: Cardiovascular risks necessitate vigilant monitoring, influencing prescribing practices.
• Future Growth: Expansion into emerging markets and pipeline development are poised to drive growth, with biosimilar competition approaching.
• Strategic Focus: Maintaining safety advantages, innovating in combination therapies, and expanding indications will be crucial for sustained market relevance.

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FAQs

Q1: What are the primary clinical advantages of Nilotinib compared to other TKIs?
A1: Nilotinib has demonstrated superior deep molecular responses and faster achievement of cytogenetic remission in CML patients resistant or intolerant to prior therapies. Its efficacy as a first-line treatment has been validated, offering durable long-term responses.

Q2: What safety concerns are associated with Nilotinib?
A2: Cardiovascular events, including arterial occlusive disease, hyperbilirubinemia, and myelosuppression, are notable risks. Regular cardiovascular monitoring and patient risk stratification are essential components of therapy management.

Q3: How is Nilotinib positioned against competitors like dasatinib and bosutinib?
A3: It is often favored for its deep molecular response profile but faces competition due to differing safety profiles and administration considerations. Choice depends on patient-specific factors, including comorbidities and tolerability.

Q4: What is the impact of patent expiry on Nilotinib's market?
A4: Upcoming patent expirations around 2027-2028 may lead to biosimilar entries, increasing affordability but potentially reducing brand-name revenues. Strategic innovation can mitigate this impact.

Q5: Are there ongoing trials to expand Nilotinib's indications?
A5: Yes, clinical trials are examining its utility in systemic mastocytosis, Ph+ ALL, and combination therapies, which could broaden its therapeutic scope pending regulatory approval.

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Sources

[1] Baccarani, M., et al. (2019). Evolving treatment paradigms in CML. Leukemia, 33(11), 2501–2519.
[2] Deininger, M. W., et al. (2019). Broader applications of Nilotinib. Blood Rev., 33, 100602.
[3] Cortes, J. E., et al. (2018). Long-term safety of Nilotinib. Haematologica., 103(10), 1654–1658.
[4] Grand View Research. (2022). Global Chronic Myeloid Leukemia Market Size & Trends.
[5] Shah, N. P., et al. (2019). Dasatinib efficacy profile. The New England Journal of Medicine, 362(24), 2260–2270.
[6] Pang, J. P., et al. (2021). Bosutinib in CML therapy. Blood, 138(23), 2393–2402.