Clinical Trials

Nilotinib hydrochloride has been evaluated in numerous clinical trials spanning Early Phase 1 through Phase 4, primarily targeting hematologic malignancies such as Philadelphia chromosome-positive chronic myeloid leukemia and acute lymphoblastic leukemia, alongside solid tumors and graft-versus-host disease. Supported by academic institutions and sponsors including Bristol-Myers Squibb, Mayo Clinic, and Massachusetts General Hospital, these studies encompass completed, recruiting, and terminated statuses. Notable protocols include combination regimens for TKI-resistant disease and optimization trials in elderly populations.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07493408 NOT_YET_RECRUITING
Ph+ Acute Lymphoblastic Leukemia (Ph+ALL); Blastic Transformation of Chronic Myeloid Leukemia; Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia (Ph+ B-ALL); Haematopoietic Stem Cell Transplant, Allogeneic
The University of Hong Kong
 2026-03-30 PHASE2
NCT01746836 RECRUITING
Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Philadelphia Chromosome Positive, BCR-ABL1 Positive Chronic Myelogenous Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive
M.D. Anderson Cancer Center
 2013-01-17 PHASE2
NCT04877522 RECRUITING
Chronic Myelogenous Leukemia; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Novartis Pharmaceuticals
 2022-08-30 PHASE4
NCT05564377 RECRUITING
Advanced Malignant Solid Neoplasm; Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IV Breast Cancer AJCC v8; Locally Advanced Malignant Solid Neoplasm; Malignant Female Reproductive System Neoplasm; Metastatic HER2-Negative Breast Carcinoma; Metastatic Malignant Solid Neoplasm; Recurrent Endometrial Carcinoma; Recurrent Fallopian Tube Carcinoma; Recurrent Malignant Female Reproductive System Neoplasm; Recurrent Malignant Solid Neoplasm; Recurrent Ovarian Carcinoma; Recurrent Primary Peritoneal Carcinoma; Unresectable HER2-Negative Breast Carcinoma; Unresectable Malignant Solid Neoplasm
National Cancer Institute (NCI)
 2023-04-07 PHASE2
NCT06409936 RECRUITING
CML, Chronic Phase; Chronic Myeloid Leukemia, Chronic Phase; Chronic Myeloid Leukemia, BCR/ABL-Positive; Chronic Myeloid Leukemia
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2025-06-26 PHASE2
NCT04626024 ACTIVE_NOT_RECRUITING
Chronic Myeloid Leukemia; Chronic Myeloid Leukemia, BCR/ABL-Positive, in Remission; Chronic Myeloid Leukemia in Remission
Baylor College of Medicine
 2020-12-22 PHASE2
NCT03516279 ACTIVE_NOT_RECRUITING
Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Minimal Residual Disease
ECOG-ACRIN Cancer Research Group
 2019-06-26 PHASE2
NCT04903119 RECRUITING
Metastatic Melanoma; BRAF Gene Mutation
Rina Plattner
 2022-06-01 PHASE1
NCT04838041 RECRUITING
Chronic Phase Chronic Myelogenous Leukemia
Medical College of Wisconsin
 2021-11-11 PHASE2
NCT02029001 RECRUITING
Malignant Solid Neoplasms
Centre Leon Berard
 2014-03 PHASE2
NCT02925234 RECRUITING
Cancer; Tumors; Neoplasm; Neoplasia
The Netherlands Cancer Institute
 2016-08 PHASE2
NCT05554341 ACTIVE_NOT_RECRUITING
Metastatic Malignant Solid Neoplasm; Refractory Malignant Solid Neoplasm
National Cancer Institute (NCI)
 2023-07-14 PHASE2
NCT04449549 RECRUITING
Neoplasms
National Cancer Institute (NCI)
 2020-08-24 PHASE2
NCT03906292 ACTIVE_NOT_RECRUITING
Chronic Myeloid Leukemia
University of Jena
 2019-08-19 PHASE2
NCT02917720 ACTIVE_NOT_RECRUITING
Chronic Myeloid Leukemia
European LeukemiaNet
 2016-09 PHASE2
NCT04147533 ACTIVE_NOT_RECRUITING
Chronic Myeloid Leukemia, Chronic Phase; Withdrawal;Drug
Masaryk University
 2020-06-16 PHASE2
NCT05143528 UNKNOWN
Alzheimer Disease
KeifeRx, LLC
 2022-02-01 PHASE3
NCT03874858 TERMINATED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2019-03-22 PHASE2
NCT01316250 COMPLETED
Chronic Myelogenous Leukemia
American University of Beirut Medical Center
 2010-08
NCT05456191 ACTIVE_NOT_RECRUITING
Philadelphia Chromosome-Positive Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2022-11-21 PHASE3
NCT02379416 COMPLETED
Solid Tumors
National Cancer Institute (NCI)
 2015-04-13 PHASE1
NCT04002674 COMPLETED
Dementia With Lewy Bodies
Georgetown University
 2019-07-01 PHASE2
NCT05185947 COMPLETED
Gynecologic Cancer; Gynecologic Neoplasms; Peritoneal Carcinomatosis; Peritoneal Neoplasms; Ovarian Cancer; Ovarian Neoplasms; Colorectal Cancer; Colorectal Neoplasms; Appendiceal Cancer; Appendiceal Neoplasms; Cholangiocarcinoma; Breast Cancer
National Cancer Institute (NCI)
 2022-10-13 PHASE2
NCT04578847 UNKNOWN
Chronic Myeloid Leukemia; Chronic Myeloid Leukemia in Remission; BCR-ABL Positive Chronic Myelogenous Leukemia
National Research Center for Hematology, Russia
 2020-01-15 PHASE2
NCT07138352 COMPLETED
Cancer
Xspray Pharma AB
 2024-10-03 PHASE1
NCT04835584 UNKNOWN
Chronic Myeloid Leukemia
Kartos Therapeutics, Inc.
 2021-05-07 PHASE1; PHASE2
NCT00756509 COMPLETED
Gastrointestinal Stromal Tumors
Novartis Pharmaceuticals
 2008-08-29 PHASE4
NCT07138378 COMPLETED
Cancer
Xspray Pharma AB
 2024-11-15 PHASE1
NCT04126681 COMPLETED
Chronic Myeloid Leukemia, Chronic Phase
Ascentage Pharma Group Inc.
 2019-10-21 PHASE2
NCT03654768 ACTIVE_NOT_RECRUITING
Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
SWOG Cancer Research Network
 2018-10-24 PHASE2
NCT04205903 COMPLETED
Anatomic Stage I Breast Cancer AJCC v8; Anatomic Stage IA Breast Cancer AJCC v8; Anatomic Stage IB Breast Cancer AJCC v8; Anatomic Stage II Breast Cancer AJCC v8; Anatomic Stage IIA Breast Cancer AJCC v8; Anatomic Stage IIB Breast Cancer AJCC v8; Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IIIA Breast Cancer AJCC v8; Anatomic Stage IIIB Breast Cancer AJCC v8; Anatomic Stage IIIC Breast Cancer AJCC v8; Prognostic Stage I Breast Cancer AJCC v8; Prognostic Stage IA Breast Cancer AJCC v8; Prognostic Stage IB Breast Cancer AJCC v8; Prognostic Stage II Breast Cancer AJCC v8; Prognostic Stage IIA Breast Cancer AJCC v8; Prognostic Stage IIB Breast Cancer AJCC v8; Prognostic Stage III Breast Cancer AJCC v8; Prognostic Stage IIIA Breast Cancer AJCC v8; Prognostic Stage IIIB Breast Cancer AJCC v8; Prognostic Stage IIIC Breast Cancer AJCC v8
Ohio State University Comprehensive Cancer Center
 2020-12-11 PHASE1
NCT02326311 COMPLETED
Leukemia, Chronic Myeloid
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
 2015-06-10 PHASE3
NCT03784014 COMPLETED
Soft Tissue Sarcoma
Institut National de la Santé Et de la Recherche Médicale, France
 2019-10-19 PHASE3
NCT04971226 ACTIVE_NOT_RECRUITING
Chronic Myeloid Leukemia (CML) Philadelphia Chromosome Positive
Novartis Pharmaceuticals
 2021-10-06 PHASE3
NCT01863745 COMPLETED
Gastrointestinal Stromal Tumors
Novartis Pharmaceuticals
 2013-06-25 PHASE2
NCT01735955 COMPLETED
Chronic Myelogenous Leukemia (CML); Metastatic Gastrointestinal Stromal Tumors (GIST); Acute Lymphoblastic Leukemia (ALL); Receptor Tyrosine Kinase (KIT) Mutated Melanoma
Novartis Pharmaceuticals
 2013-03-29 PHASE4
NCT03942094 UNKNOWN
Chronic Myeloid Leukemia, Chronic Phase; Nilotinib
Shenzhen Second People's Hospital
 2019-06-01 PHASE3
NCT02602314 UNKNOWN
Chronyc Myeloid Leukemia
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2016-11-11 PHASE4
NCT02201459 COMPLETED
Chronic Myeloid Leukemia
Hospices Civils de Lyon
 2014-08 PHASE3
NCT01657604 COMPLETED
Chronic Myeloid Leukemia
University of Jena
 2012-08-24 PHASE3
NCT02001818 COMPLETED
Chronic Myeloid Leukaemia
Australasian Leukaemia and Lymphoma Group
 2014-04-11 PHASE2
NCT03578367 COMPLETED
CML; Chronic Myelogenous Leukemia; Leukemia, Myeloid Chronic; Hematologic Diseases
Novartis Pharmaceuticals
 2018-11-22 PHASE2
NCT02973711 WITHDRAWN
Leukemia, Chronic Myeloid
University of Michigan Rogel Cancer Center
 2018-01 PHASE1; PHASE2
NCT02081378 COMPLETED
Chronic Myelogenous Leukemia; Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia
Novartis Pharmaceuticals
 2014-04-24 PHASE1
NCT02269267 COMPLETED
Leukemia, Myeloid, Chronic
Medical College of Wisconsin
 2014-12-18 PHASE2
NCT02546674 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2016-02-18 PHASE4
NCT02353728 COMPLETED
Chronic Myeloid Leukemia
Weill Medical College of Cornell University
 2015-01 PHASE2
NCT01774630 COMPLETED
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
University Hospital, Bordeaux
 2013-04-10 PHASE2
NCT02947893 COMPLETED
Alzheimer's Disease
Georgetown University
 2017-01 PHASE2
NCT02611492 UNKNOWN
Philadelphia Chromosome Positive Adult Acute Lymphoblastic Leukemia
Assistance Publique - Hôpitaux de Paris
 2016-04 PHASE3
NCT03764215 UNKNOWN
Huntington Disease
Georgetown University
 2018-11-15 PHASE1
NCT02627677 TERMINATED
Chronic Phase Chronic Myeloid Leukemia
Ariad Pharmaceuticals
 2015-12-31 PHASE3
NCT04448821 COMPLETED
Healthy
Hyewon Chung
 2020-07-23 PHASE1
NCT03932669 COMPLETED
Ataxia, Cerebellar; Ataxia, Progressive
Seoul National University Hospital
 2018-11-19 PHASE2
NCT03228303 UNKNOWN
Chronic Myeloid Leukemia
Assiut University
 2017-12-01 EARLY_PHASE1
NCT01743989 COMPLETED
Philadelphia Chromosome Positive (PH+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP)
Novartis Pharmaceuticals
 2013-04-15 PHASE3
NCT01856283 COMPLETED
Leukemia, Myeloid, Chronic-Phase
Niguarda Hospital
 2013-03 PHASE2
NCT02954978 COMPLETED
Parkinson Disease; Parkinsons Disease With Dementia
Georgetown University
 2017-01 PHASE2
NCT01528085 COMPLETED
Philadelphia Chromsome Positive Acute Lymphoblastic Leukemia
Goethe University
 2012-01 PHASE2
NCT03205267 UNKNOWN
Chronic Myelogenous Leukaemia
University of Bonn
 2016-03 PHASE2
NCT01751425 TERMINATED
Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission; Minimal Residual Disease; Philadelphia Chromosome Positive, BCR-ABL1 Positive Chronic Myelogenous Leukemia
M.D. Anderson Cancer Center
 2013-07-24 PHASE1
NCT01690065 UNKNOWN
Chronic Myeloid Leukemia in Myeloid Blast Crisis; Untreated Adult Acute Myeloid Leukemia
Ulsan University Hospital
 2012-09 PHASE2
NCT03205488 COMPLETED
Parkinson Disease
Northwestern University
 2017-10-16 PHASE2
NCT03214718 UNKNOWN
Chronic Myeloid Leukemia Patients
Assiut University
 2017-08-05
NCT01274351 COMPLETED
Chronic Myelogenous Leukemia
Novartis Pharmaceuticals
 2011-01-25 PHASE2
NCT01887522 TERMINATED
Refractory Low-grade Gliomas; Recurrent Low-grade Gliomas
Gustave Roussy, Cancer Campus, Grand Paris
 2016-07-06 PHASE2
NCT01914484 COMPLETED
Chronic Phase Chronic Myeloid Leukemia; Accelerated Phase Chronic Myeloid Leukemia; Blastic Phase Chronic Myeloid Leukemia; Philadelphia Positive Acute Lymphoblastic Leukemia; Resistant to Tyrosine Kinase Inhibitor Therapy
University Health Network, Toronto
 2013-08-01 PHASE1; PHASE2
NCT02174445 TERMINATED
Chronic Myeloid Leukemia
Prof. Dr. Nikolas von Bubnoff
 2014-03 PHASE3
NCT01744665 COMPLETED
CML
Novartis Pharmaceuticals
 2013-08-12 PHASE2
NCT03079505 TERMINATED
Chronic Myeloid Leukemia - Chronic Phase
Hamad Medical Corporation
 2017-08-03 PHASE3
NCT01667133 COMPLETED
Chronic Myeloid Leukemia (CML); Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)
Ariad Pharmaceuticals
 2012-08-31 PHASE1; PHASE2
NCT00129740 COMPLETED
Leukemia, Myelogenous, Chronic
M.D. Anderson Cancer Center
 2005-06-27 PHASE2
NCT02709083 TERMINATED
Chronic Myelogenous Leukemia; Chronic Myeloid Leukemia; Leukemia
Emory University
 2016-10 PHASE2
NCT01804985 COMPLETED
Chronic Myeloid Leukaemia
University of Liverpool
 2013-12 PHASE2
NCT00481052 COMPLETED
Chronic Myeloid Leukemia
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2007-06-23 PHASE2
NCT02253277 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2015-02-18 PHASE1
NCT02389920 UNKNOWN
Leukemia, Chronic Myeloid
Samsung Medical Center
 2015-04 PHASE4
NCT02774512 WITHDRAWN
Colon Cancer
Institut Bergonié
 2016-05 EARLY_PHASE1
NCT01806571 COMPLETED
Untreated Adult Acute Myeloid Leukemia
Mayo Clinic
 2015-03-12 PHASE2
NCT02891395 COMPLETED
Graft Versus Host Disease
University Hospital, Lille
 2012-12-24 PHASE2
NCT02225574 TERMINATED
Leukemia
M.D. Anderson Cancer Center
 2015-03-11 PHASE1
NCT02272777 COMPLETED
Leukemia
Novartis Pharmaceuticals
 2014-07-17 PHASE3
NCT01605981 WITHDRAWN
Chronic Myeloid Leukemia (CML)
Novartis Pharmaceuticals
 2014-02 PHASE4
NCT01395121 COMPLETED
Mucosal Lentiginous Melanoma; Acral Lentiginous Malignant Melanoma
Institute of Cancer Research, United Kingdom
 2009-12 PHASE2
NCT01871311 TERMINATED
Colorectal Cancer; Head and Neck Cancer
Georgetown University
 2014-05 PHASE1
NCT02115386 TERMINATED
Philadelphia Positive (Ph+) Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2015-12-17 PHASE3
NCT03332511 COMPLETED
Chronic Myeloid Leukemia, Chronic Phase
Seoul National University Hospital
 2013-05-06 PHASE4
NCT01702064 COMPLETED
Chronic Phase Chronic Myeloid Leukemia
H. Lee Moffitt Cancer Center and Research Institute
 2013-02-21 PHASE1
NCT01140568 COMPLETED
Glioma
David Piccioni, M.D., Ph.D
 2010-04-21 PHASE2
NCT02108951 TERMINATED
Philidelphia Positive Chronic Myeloid Leukaemia
Novartis Pharmaceuticals
 2014-07-07 PHASE3
NCT02086487 TERMINATED
Myeloid Leukemia, Chronic
King Abdullah International Medical Research Center
 2013-03 PHASE4
NCT01275586 COMPLETED
Neurofibromatosis; NF1; Neurofibromas
Indiana University
 2011-01 EARLY_PHASE1
NCT01844765 COMPLETED
Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia
Novartis Pharmaceuticals
 2013-08-20 PHASE2
NCT01784068 COMPLETED
Chronic Myelogenous Leukemia
Novartis Pharmaceuticals
 2013-03-19 PHASE2
NCT01866553 TERMINATED
Chronic Myeloid Leukemia
Amsterdam UMC, location VUmc
 2013-04 PHASE2
NCT00750659 COMPLETED
Chronic Myeloid Leukemia; Acute Lymphoblastic Leukemia; Stem Cell Transplantation
Sheba Medical Center
 2009-07 PHASE2
NCT01407198 COMPLETED
Chordoma
Massachusetts General Hospital
 2011-08 PHASE1
NCT01025505 COMPLETED
Treatment; Stem Cell Transplantation
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2012-06 PHASE2
NCT02587169 UNKNOWN
Retroperitoneal Liposarcoma; Retroperitoneal Leiomyosarcoma; Chondrosarcoma
Broto, Javier Martín, M.D.
 2012-01 PHASE1; PHASE2
NCT01698905 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2012-12-20 PHASE2
NCT01884922 COMPLETED
Refractory Low-grade Gliomas; Recurrent Low-grade Gliomas
Gustave Roussy, Cancer Campus, Grand Paris
 2013-05-29 PHASE1
NCT01670084 WITHDRAWN
B-cell Adult Acute Lymphoblastic Leukemia; Blastic Phase Chronic Myelogenous Leukemia; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Philadelphia Chromosome Positive Adult Precursor Acute Lymphoblastic Leukemia; Untreated Adult Acute Lymphoblastic Leukemia
Mayo Clinic
 2012-12 PHASE2
NCT01077544 COMPLETED
Chronic Myeloid Leukemia; Acute Lymphoblastic Leukemia
Novartis Pharmaceuticals
 2011-04-14 PHASE1
NCT00760877 COMPLETED
CHRONIC MYELOGENOUS LEUKEMIA
Novartis Pharmaceuticals
 2009-06 PHASE3
NCT02281474 COMPLETED
Parkinson's Disease; Parkinson's Disease Dementia; Diffuse Lewy Body Disease
Georgetown University
 2014-11 PHASE1
NCT01222143 TERMINATED
Acute Myeloid Leukemia
University Health Network, Toronto
 2010-10 PHASE1; PHASE2
NCT01490775 TERMINATED
Chronic Phase CML
Rex Cancer Center, Raleigh, NC
 2009-04
NCT01810718 COMPLETED
Chronic Graft Versus Host Disease
Gruppo Italiano Trapianto di Midollo Osseo
 2011-11 PHASE1
NCT01028222 COMPLETED
Melanoma
Novartis Pharmaceuticals
 2010-06 PHASE2
NCT01254188 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2011-04 PHASE3
NCT01535391 COMPLETED
Chronic Myeloid Leukemia
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2012-01 PHASE3
NCT01227577 COMPLETED
Chronic Myelogenous Leukemia in Chronic Phase
Novartis Pharmaceuticals
 2010-11 PHASE4
NCT01275196 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2011-04 PHASE3
NCT00769327 COMPLETED
Leukemia
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2009-02-09 PHASE2
NCT01490983 TERMINATED
Chronic Myeloid Leukemia
Rex Cancer Center, Raleigh, NC
 2009-04
NCT00785785 COMPLETED
Gastrointestinal Stromal Tumor (GIST)
Novartis Pharmaceuticals
 2009-03 PHASE3
NCT00036738 COMPLETED
Adult Acute Lymphoblastic Leukemia in Remission; Adult B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1; Blastic Phase; Childhood Acute Lymphoblastic Leukemia in Remission; Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Chronic Phase of Disease; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Childhood Acute Lymphoblastic Leukemia; Recurrent Disease
Fred Hutchinson Cancer Center
 2001-07-13 PHASE2
NCT01166139 COMPLETED
Systemic Sclerosis
Hospital for Special Surgery, New York
 2010-07 PHASE2
NCT01061177 COMPLETED
CML in Chronic Phase
Novartis Pharmaceuticals
 2010-05 PHASE4
NCT00802841 COMPLETED
Chronic Myelogenous Leukemia
Novartis Pharmaceuticals
 2009-05 PHASE3
NCT01289028 COMPLETED
Gastrointestinal Stromal Tumors
Novartis Pharmaceuticals
 2008-11 PHASE3
NCT01400074 UNKNOWN
Chronic Myeloid Leukemia
Seoul St. Mary's Hospital
 2009-01 PHASE3
NCT01207492 COMPLETED
Pigmented Villonodular Synovitis; Diffuse-type Giant Cell Tumor; Tenosynovial Giant Cell Tumor
Andrew J. Wagner, MD, PhD
 2010-09 PHASE2
NCT01456676 COMPLETED
Philadelphia Chromosome Positive Chronic Myelogenous Leukemia
Novartis Pharmaceuticals
 2012-01 PHASE1
NCT01220648 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2012-04 PHASE1
NCT01099514 COMPLETED
Metastatic Melanoma With KIT Aberration
Samsung Medical Center
 2009-08 PHASE2
NCT00702403 COMPLETED
Accelerated Phase Chronic Myelogenous Leukemia; Adult Acute Lymphoblastic Leukemia in Remission; Blastic Phase Chronic Myelogenous Leukemia; Childhood Acute Lymphoblastic Leukemia in Remission; Childhood Chronic Myelogenous Leukemia; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Chronic Phase Chronic Myelogenous Leukemia; Philadelphia Positive Adult Acute Lymphoblastic Leukemia; Philadelphia Positive Childhood Acute Lymphoblastic Leukemia; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Childhood Acute Lymphoblastic Leukemia; Relapsing Chronic Myelogenous Leukemia; Untreated Adult Acute Lymphoblastic Leukemia; Untreated Childhood Acute Lymphoblastic Leukemia
Fred Hutchinson Cancer Center
 2008-08-14 PHASE1; PHASE2
NCT01168050 UNKNOWN
Malignant Skin Melanoma T0; Stage III Melanoma; Stage IV Melanoma; Amplification
Assistance Publique - Hôpitaux de Paris
 2010-07 PHASE2
NCT01043874 COMPLETED
Philadelphia Chromosome Positive; Chronic Myelogenous Leukemia in Chronic Phase
Novartis Pharmaceuticals
 2009-12 PHASE4
NCT01155817 COMPLETED
Bone Marrow Transplant Failure; Lymphoma, Non-Hodgkin; Lymphoma, T-Cell, Peripheral
Stanford University
 2010-08 PHASE1
NCT01223898 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2010-06 PHASE1
NCT01252017 COMPLETED
Patients Who Have Received Allo-HSCT
National Taiwan University Hospital
 2010-11 PHASE2
NCT01819389 COMPLETED
Leukemia, Myeloid, Chronic, BCR-ABL Positive
Hospital Universitario Dr. Jose E. Gonzalez
 2012-10 PHASE3
NCT00718562 COMPLETED
Gastrointestinal Stromal Tumors
Novartis Pharmaceuticals
 2008-09 PHASE2
NCT01261429 COMPLETED
Pigmented Villonodular Synovitis
Centre Leon Berard
 2010-12 PHASE2
NCT01169753 TERMINATED
Chronic Myeloid Leukemia
M.D. Anderson Cancer Center
 2011-05
NCT01179737 TERMINATED
Pulmonary Arterial Hypertension
Novartis Pharmaceuticals
 2010-07 PHASE2
NCT00980018 COMPLETED
Chronic Myelogenous Leukemia
Novartis Pharmaceuticals
 2009-12 PHASE4
NCT01602952 COMPLETED
Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Philadelphia Chromosome; Hematologic Diseases
Il-Yang Pharm. Co., Ltd.
 2008-07 PHASE1; PHASE2
NCT01206088 COMPLETED
CML; Nilotinib; Imatinib Resistant; Imatinib Intolerant
Novartis Pharmaceuticals
 2009-02 PHASE4
NCT00109707 COMPLETED
Chronic Myelogenous Leukemia; Acute Lymphoblastic Leukemia (Philadelphia Chromosome Positive); Hypereosinophilic Syndrome; Systemic Mastocytosis
Novartis Pharmaceuticals
 2005-04 PHASE1; PHASE2
NCT00751036 TERMINATED
Gastrointestinal Stromal Tumors
Novartis Pharmaceuticals
 2009-06 PHASE3
NCT00905398 TERMINATED
Precursor B-Cell Lymphoblastic Leukemia-Lymphoma; Acute Lymphoblastic Leukemia
Rony Schaffel
 2009-05 PHASE1; PHASE2
NCT00844298 COMPLETED
Leukemia
Asan Medical Center
 2009-01 PHASE2
NCT00762632 COMPLETED
Acute Myeloid Leukemia
Technical University of Munich
 2007-12 PHASE1; PHASE2
NCT00644878 TERMINATED
Chronic Myelogenous Leukemia - Chronic Phase
Novartis Pharmaceuticals
 2008-10 PHASE2
NCT00786812 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2008-08 PHASE4
NCT01089595 TERMINATED
GIST; Metastatic Disease
Fox Chase Cancer Center
 2009-02 PHASE2
NCT01201538 TERMINATED
Growing Vestibular Schwannomas
University Health Network, Toronto
 2010-10 PHASE2
NCT01110668 COMPLETED
Gastrointestinal Stromal Tumor
Novartis Pharmaceuticals
 2008-09 PHASE2
NCT00732888 COMPLETED
Healthy
Jan Beumer
 2008-11 PHASE1
NCT01131325 TERMINATED
CML; Philadelphia Chromosome Positive (Ph+); Chronic Myelogenous Leukemia Chronic Phase(CML-CP) Patients With Low Imatinib Trough Levels
Novartis Pharmaceuticals
 2010-10-21 PHASE4
NCT00809211 COMPLETED
Leukemia
Cancer Trials Ireland
 2008-10 PHASE2
NCT00788775 COMPLETED
Mucosal Lentiginous Melanoma; Acral Melanoma; Melanoma
Dana-Farber Cancer Institute
 2009-01-23 PHASE2
NCT01126892 COMPLETED
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
 2009-01 PHASE3
NCT01294618 COMPLETED
Chronic Myelogenous Leukemia
Hospices Civils de Lyon
 2011-03 PHASE2
NCT00441155 COMPLETED
Gastrointestinal Stromal Tumors
Novartis Pharmaceuticals
 2006-11 PHASE1
NCT00573378 TERMINATED
Chronic Myeloid Leukemia
University of Michigan Rogel Cancer Center
 2007-09 PHASE2
NCT00633295 COMPLETED
Gastrointestinal Stromal Tumors (GIST)
Novartis Pharmaceuticals
 2008-06 PHASE2
NCT01368523 COMPLETED
Chronic Myelogenous Leukemia
Novartis Pharmaceuticals
 2008-12 PHASE4
NCT01279473 COMPLETED
Chronic Myelogenous Leukemia; Acute Lymphoblastic Leukemia
Novartis Pharmaceuticals
 2005-08 PHASE1; PHASE2
NCT00471497 COMPLETED
Myelogenous Leukemia, Chronic
Novartis Pharmaceuticals
 2007-07-31 PHASE3
NCT00519090 TERMINATED
Myelogenous Leukemia
Novartis Pharmaceuticals
 2007-10 PHASE3
NCT00471328 COMPLETED
Gastrointestinal Stromal Tumors
Novartis Pharmaceuticals
 2007-03 PHASE3
NCT00782834 TERMINATED
Gastrointestinal Stromal Tumors
Fox Chase Cancer Center
 2008-07 PHASE2
NCT00418626 COMPLETED
Healthy
Novartis Pharmaceuticals
 2006-11 PHASE1
NCT00384228 COMPLETED
Chronic Myelogenous Leukemia; Acute Lymphoblastic Leukemia (Philadelphia Chromosome Positive)
Novartis Pharmaceuticals
 2005-05 PHASE1; PHASE2
NCT00418756 COMPLETED
Healthy
Novartis Pharmaceuticals
 2006-10 PHASE1
NCT00418769 COMPLETED
Healthy
Novartis Pharmaceuticals
 2006-06 PHASE1
NCT01270893 WITHDRAWN
Gastrointestinal Cancer
M.D. Anderson Cancer Center
 2011-01 PHASE2

(data from https://clinicaltrials.gov, updated on 2026-03-25)

Check the Nilotinib hydrochloride product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Nilotinib hydrochloride selectively binds to the ATP-binding site of the active BCR-ABL tyrosine kinase, inhibiting enzyme autophosphorylation and blocking downstream oncogenic signaling cascades. This molecular blockade arrests cell cycle progression and triggers apoptosis in BCR-ABL-expressing cells, suppressing aberrant leukemic proliferation in clinical indications such as chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.