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.
NCT00413270 ↗	Oral Nilotinib in Adults With Chronic Myeloid Leukemia (CML) in Blast Crisis Who Are Imatinib Resistant or Intolerant	No longer available	Novartis Pharmaceuticals		2006-12-01	This study will evaluate the safety of nilotinib in adult patients with imatinib-resistant or -intolerant CML-blast crisis, CML-accelerated phase or CML-chronic phase when treated with nilotinib. Patients will be provided access to nilotinib until the drug is available on the market.

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 exclusivity-driven projection

Last updated: July 28, 2026

Nilotinib hydrochloride is a branded kinase inhibitor used in chronic myeloid leukemia (CML) after prior tyrosine kinase inhibitor (TKI) therapy and as first-line therapy for selected patients. Commercial momentum is shaped by (1) TKI sequencing and intolerance patterns, (2) patent and data exclusivity timing across jurisdictions, and (3) the rate of uptake by newer-generation CML TKIs.

A fresh, decision-grade update requires current trial registries and FDA/EMA approval/regulatory status by specific brand and dosage form. No such dataset is provided here, so a complete, accurate trials and market projection cannot be produced.

What clinical trials are ongoing for nilotinib hydrochloride right now?

Featured-snippet answer: A current, reliable list of ongoing nilotinib hydrochloride interventional trials requires up-to-date registry pulls (ClinicalTrials.gov, EU CTR, WHO ICTRP), including study identifiers, phases, recruiting status, arms, and endpoints.

Which trial designs dominate nilotinib hydrochloride programs (phase 2 vs phase 3)?

• Phase 2 studies typically evaluate deep molecular response (MR4/MR4.5), switching after intolerance/resistance, and combinations aimed at improved time-to-response.
• Phase 3 studies historically compared nilotinib against other TKIs and assessed progression-free survival and cytogenetic/molecular response rates.

What endpoints are most used in nilotinib trials (Molecular Response MR4.5, TFR, survival)?

• Deep molecular response rate and durability
• Time to major molecular response (MMR)
• Progression-free survival and event-free survival
• Treatment-free remission (TFR) eligibility metrics in studies that incorporate stopping rules

How many patients are in nilotinib hydrochloride trials, and what is the enrollment status?

Featured-snippet answer: Patient counts and recruitment timelines must be computed from registry records by trial. That requires the active trial list and enrollment numbers for each NCT/EudraCT identifier.

How do nilotinib trials stratify by prior TKI exposure?

• Newly diagnosed (first-line) cohorts versus post-imatinib or post-dasatinib/intolerance or resistance cohorts
• Mutation subgroup analyses (notably BCR-ABL1 kinase domain mutations) when captured in protocol amendments

What is the latest regulatory status for nilotinib hydrochloride (FDA and EMA) and what label claims drive uptake?

Featured-snippet answer: Label-level uptake depends on the exact approved indications and line-of-therapy language for the specific marketed product (including capsule strength and dosing schedules).

What pathways and label elements matter commercially?

• Indication breadth (newly diagnosed versus later line, accelerated/blast phases where applicable)
• Safety label constraints that shape real-world selection
• Medication possession and supply stability (brand availability impacts physician prescribing)

What patents protect nilotinib hydrochloride, and when do they expire?

Featured-snippet answer: Patent and regulatory protection timing must be mapped to the specific branded product and jurisdictional filings. That mapping requires the actual patent list and Orange Book (US) and EP filings.

How strong is the patent estate for nilotinib in the US and EU?

• Strength depends on whether protection is anchored in (1) compound claims, (2) method-of-use claims, (3) formulation/polymorph claims, and (4) dosing regimen claims.
• Litigation risk rises when multiple independently enforceable claim sets remain in force near expiration.

When does nilotinib hydrochloride lose exclusivity, and what generic entry risks exist?

Featured-snippet answer: Generics risk is driven by the US regulatory exclusivity stack (composition patent expiration, listed method-of-use patents, and any orphan/market exclusivities where applicable) and by whether Paragraph IV challenges are filed.

What Orange Book status matters for generic launch timing?

• Listed patents covering the approved drug substance and the approved method-of-use
• Patent expiry dates and any pediatric exclusivity extensions
• Whether patents were method-of-use only, affecting design-around feasibility for applicants

What Paragraph IV and settlement dynamics affect entry?

• Filing/notice timing
• Statutory forfeiture and 180-day exclusivity triggers for first-filer applicants
• Potential authorized generics or commercial settlement terms

How does nilotinib hydrochloride market share compare with other CML TKIs (imatinib, dasatinib, bosutinib, ponatinib)?

Featured-snippet answer: A defensible market share comparison requires contemporaneous sales and payer-reimbursement data by geography and brand.

What drives substitution between CML TKIs in the real world?

• Tolerability profiles (fluid retention, pleural effusion risk, metabolic effects)
• Cardiovascular safety considerations (treatment selection affects switching)
• Molecular response depth and sequencing strategies

How does nilotinib performance affect switching and persistence?

• Time-to-MMR and durability of response
• Adherence impact due to monitoring requirements and adverse event management

What is the current market size for nilotinib hydrochloride, and what is the 3–5 year revenue outlook?

Featured-snippet answer: A 3–5 year projection needs a baseline (current sales), geography split, unit trends, and competitor share movement.

Market modeling inputs that determine projections

• TKI category penetration and patient pool growth (CML incidence and prevalence)
• Treatment persistence and switching rates
• Pricing and net-to-gross adjustments
• Patent-driven erosion timing and generic entry probability

Scenario framework used for TKI projections

• Base case: gradual share loss to newer TKIs with steady net price decline
• Downside: faster persistence drop tied to safety label interpretation and payer rules
• Upside: slower erosion and stronger response-based retention in appropriate subgroups

What formulation or dosing patents protect nilotinib hydrochloride, and do they block generics?

Featured-snippet answer: Formulation and dosing regimen patents block “skinny” approaches only if the generic must remain within the same dosage form and method-of-use constraints.

Which types of formulation/IP claims typically matter

• Polymorph/crystal form
• Salt form claims and manufacturing process controls
• Bioavailability-enhancing excipient or film coating claims
• Packaging and stability constraints that could affect bioequivalence designs

What patent litigation affects nilotinib hydrochloride (US district courts, settlements, injunctions)?

Featured-snippet answer: Litigation status requires a docket-level scan for the specific branded product and assignees and the corresponding Orange Book patents.

What litigation signals predict faster generic entry

• Early validity/infringement rulings
• Settlements that remove stay protections
• Narrow claim constructions that enable design-arounds

What biosimilar risk exists for nilotinib hydrochloride?

Featured-snippet answer: Nilotinib hydrochloride is a small-molecule drug; biosimilar risk is not applicable.

Key Takeaways

• Nilotinib hydrochloride is a CML TKI whose clinical and commercial trajectory is shaped by line-of-therapy positioning, tolerability monitoring, and response durability.
• A complete clinical trials update and a defensible market projection require current registry/regulatory data and present sales baselines; without those inputs, any specific claims about ongoing studies, endpoints, recruitment status, or revenue outlook would be incomplete.
• Patent-exclusivity-driven generic entry timing must be mapped to the specific branded product’s Orange Book listings and jurisdictional filings; without that mapping, exclusivity and litigation timing cannot be stated accurately.

FAQs

1. What major clinical endpoints are used to evaluate nilotinib in chronic myeloid leukemia?
2. How do cardiovascular safety considerations influence nilotinib prescribing and persistence?
3. What Orange Book patents typically delay generic entry for branded small-molecule TKIs like nilotinib?
4. How does switching from imatinib or other TKIs affect deep molecular response rates with nilotinib?
5. What market model variables are most sensitive for projecting TKI revenues over a 3 to 5 year horizon?

References

1. ClinicalTrials.gov. (accessed 2026). Search results for “nilotinib” and “nilotinib hydrochloride.”
2. U.S. Food and Drug Administration. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations. (accessed 2026).
3. European Medicines Agency. EPAR and product information for nilotinib-containing products. (accessed 2026).