Clinical Trials

Numerous clinical trials spanning Phase 1 through Phase 3 evaluate ponatinib across hematologic malignancies—such as Philadelphia chromosome-positive acute lymphoblastic leukemia, chronic myeloid leukemia, and acute myeloid leukemia—and solid tumors including non-small cell lung cancer, glioblastoma, and gastrointestinal stromal tumors. Sponsored by major academic institutions and cooperative research groups like the M.D. Anderson Cancer Center, Dana-Farber Cancer Institute, and GIMEMA, these active, recruiting, and completed protocols investigate sequential therapies, combination regimens, and targeted interventions.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06860269 RECRUITING
Acute Lymphoblastic Leukemia
Assistance Publique - Hôpitaux de Paris
 2025-05-06 PHASE2; PHASE3
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
NCT07614022 NOT_YET_RECRUITING
Philadelphia Positive Acute Lymphoblastic Leukemia
Wake Forest University Health Sciences
 2026-10 PHASE1
NCT06061094 RECRUITING
Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
Goethe University
 2023-07-14 PHASE2
NCT04070443 ACTIVE_NOT_RECRUITING
Philadelphia Chromosome Positive CML; BCR-ABL Positive Chronic Myelogenous Leukemia
Centre Leon Berard
 2019-11-13 PHASE2
NCT06514534 RECRUITING
Chronic Myeloid Leukemia (CML)
Novartis Pharmaceuticals
 2025-02-18 PHASE2
NCT07224100 RECRUITING
B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1; B Lymphoblastic Leukemia/Lymphoma With t(9;22)(q34.1;q11.2); BCR-ABL1; Lymphoblastic Lymphoma
University of Washington
 2026-03-30 PHASE2
NCT04530565 ACTIVE_NOT_RECRUITING
B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1
National Cancer Institute (NCI)
 2021-01-25 PHASE3
NCT04747912 RECRUITING
Lymphoblastic Leukemia; Acute Lymphoblastic Leukemia; ph+ Acute Lymphoblastic Leukemia
University of Chicago
 2021-03-02 PHASE2
NCT06813079 RECRUITING
Pancreatic Ductal Carcinoma; Advanced Cancer; Epithelial Tumor
University Health Network, Toronto
 2025-04-07 PHASE2
NCT03934372 RECRUITING
Acute Myeloid Leukemia; Accelerated Phase Chronic Myeloid Leukemia; Blast Phase Chronic Myeloid Leukemia; Chronic Phase Chronic Myeloid Leukemia; Acute Lymphoblastic Leukemia; Acute Lymphocytic Leukemia; Leukemia; Lymphoma; Solid Tumors
Incyte Biosciences International Sàrl
 2020-01-29 PHASE1; PHASE2
NCT07301424 NOT_YET_RECRUITING
Acute Lymphoblastic Leukemia (ALL) Philadelphia Chromosome-positive (Ph+); BCR-ABL Positive Acute Lymphoblastic Leukemia
University of Alberta
 2027-01-02 PHASE2
NCT03263572 RECRUITING
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Acute Lymphoblastic Leukemia; BCR-ABL1 Fusion Protein Expression; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Philadelphia Chromosome Positive; Recurrent Acute Lymphoblastic Leukemia; Refractory Acute Lymphoblastic Leukemia; t(9;22)
M.D. Anderson Cancer Center
 2017-11-29 PHASE2
NCT01424982 ACTIVE_NOT_RECRUITING
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Acute Lymphoblastic Leukemia; Adult Acute Lymphoblastic Leukemia in Complete Remission; B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Philadelphia Chromosome Positive; Untreated Adult Acute Lymphoblastic Leukemia
M.D. Anderson Cancer Center
 2011-10-05 PHASE2
NCT04722848 ACTIVE_NOT_RECRUITING
Acute Lymphoblastic Leukemia (Philadelphia Chromosome Positive); ALL, Adult; Philadelphia-Positive ALL
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2021-09-08 PHASE3
NCT03147612 ACTIVE_NOT_RECRUITING
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia With BCR-ABL1; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Recurrent Acute Lymphoblastic Leukemia; Refractory Acute Lymphoblastic Leukemia
M.D. Anderson Cancer Center
 2018-02-08 PHASE2
NCT03739814 RECRUITING
B Acute Lymphoblastic Leukemia, Philadelphia Chromosome Negative; Recurrent B Acute Lymphoblastic Leukemia; Refractory B Acute Lymphoblastic Leukemia
National Cancer Institute (NCI)
 2019-05-08 PHASE2
NCT06207123 RECRUITING
Acute Leukemia; Lymphoblastic Leukemia; Lymphoblastic Lymphoma
University of Chicago
 2024-09-13 PHASE1; PHASE2
NCT04188405 COMPLETED
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Acute Myeloid Leukemia; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Recurrent Acute Myeloid Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Refractory Acute Myeloid Leukemia; Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive
M.D. Anderson Cancer Center
 2020-05-17 PHASE2
NCT05268003 TERMINATED
Acute Lymphoblastic Leukemia; Leukemia
M.D. Anderson Cancer Center
 2022-06-07 PHASE2
NCT05306301 ACTIVE_NOT_RECRUITING
Chemotherapy; Leukemia, Acute Lymphoblastic
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2022-10-05 PHASE2
NCT04845035 WITHDRAWN
Acute Lymphoblastic Leukemia
University of Michigan Rogel Cancer Center
 2024-01 PHASE2
NCT04160546 ACTIVE_NOT_RECRUITING
Chronic Myeloid Leukemia, Chronic Phase
Fundacion CRIS de Investigación para Vencer el Cáncer
 2020-01-17 PHASE2
NCT03895671 COMPLETED
CHRONIC MYELOGENOUS LEUKAEMIA IN ACCELERATED PHASE; CHRONIC MYELOGENOUS LEUKAEMIA IN MYELOID BLAST CRISIS
Versailles Hospital
 2019-06-19 PHASE2
NCT04591431 UNKNOWN
Breast Cancer; Gastrointestinal Cancer; Non Small Cell Lung Cancer; Other Cancer
Fondazione per la Medicina Personalizzata
 2020-10-07 PHASE2
NCT04475731 ACTIVE_NOT_RECRUITING
Philadelphia-Positive ALL; Acute Lymphoblastic Leukemia, in Relapse
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2021-05-04 PHASE2
NCT04709731 UNKNOWN
Chronic Myeloid Leukemia (CML)
Associazione Italiana Pazienti Leucemia Mieloide Cronica
 2021-02-01 PHASE2
NCT03576547 TERMINATED
Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Philadelphia Chromosome Positive; Recurrent Acute Lymphoblastic Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Refractory Acute Lymphoblastic Leukemia; Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive; t(9;22)
M.D. Anderson Cancer Center
 2018-06-26 PHASE1; PHASE2
NCT04688983 UNKNOWN
Acute Lymphoblastic Leukemia, Adult
Cardiff University
 2021-01 PHASE2
NCT04501614 TERMINATED
Pediatric Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia (Ph+ALL); Ph+ Mixed Phenotype Acute Leukemia (MPAL); Philadelphia Chromosome-Like ALL (Ph-like ALL)
Takeda
 2021-02-24 PHASE1; PHASE2
NCT03690115 COMPLETED
Leukemia, Myeloid, Acute
Versailles Hospital
 2019-12-02 PHASE2
NCT04554459 TERMINATED
Acute Lymphoblastic Leukemia, Adult; Ph+ ALL; Newly Diagnosed
Institute of Hematology and Blood Transfusion, Czech Republic
 2021-02-16 PHASE2
NCT03807479 TERMINATED
Leukemia, Myeloid, Chronic-Phase
GWT-TUD GmbH
 2018-12-11 PHASE2
NCT02272998 COMPLETED
Malignant Neoplasm
Sameek Roychowdhury
 2015-02-24 PHASE2
NCT05306301 Recruiting
Chemotherapy|Leukemia Acute Lymphoblastic
Gruppo Italiano Malattie EMatologiche dell''Adulto
 2022-10-05 Phase 2
NCT03589326 ACTIVE_NOT_RECRUITING
Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ALL)
Takeda
 2018-10-04 PHASE3
NCT04233346 ACTIVE_NOT_RECRUITING
Chronic Myeloid Leukemia; Acute Lymphoblastic Leukemia
Otsuka Beijing Research Institute
 2020-07-09 PHASE2
NCT05268003 Recruiting
Acute Lymphoblastic Leukemia|Leukemia
M.D. Anderson Cancer Center
 2022-06-07 Phase 2
NCT04043676 UNKNOWN
Chronic Myeloid Leukemia
Fundación Teófilo Hernando, Spain
 2019-04-30 PHASE2
NCT03704688 COMPLETED
Non Small Cell Lung Cancer; KRAS Gene Mutation
Memorial Sloan Kettering Cancer Center
 2018-10-09 PHASE1; PHASE2
NCT02428543 COMPLETED
Acute Myeloid Lukemia
Versailles Hospital
 2013-07 PHASE1; PHASE2
NCT02829840 WITHDRAWN
Leukemia; FLT3-Mutated Acute Myeloid Leukemia; FLT3-Mutated High-Risk Myelodysplastic Syndrome
M.D. Anderson Cancer Center
 2016-09 PHASE1; PHASE2
NCT03838692 WITHDRAWN
Medullary Thyroid Cancer
Antonio Fojo
 2019-07-26 PHASE2
NCT03878524 TERMINATED
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Anatomic Stage IV Breast Cancer AJCC v8; Anemia; Ann Arbor Stage III Hodgkin Lymphoma; Ann Arbor Stage III Non-Hodgkin Lymphoma; Ann Arbor Stage IV Hodgkin Lymphoma; Ann Arbor Stage IV Non-Hodgkin Lymphoma; Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Castration-Resistant Prostate Carcinoma; Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Hematopoietic and Lymphoid System Neoplasm; Locally Advanced Pancreatic Adenocarcinoma; Metastatic Breast Carcinoma; Metastatic Malignant Solid Neoplasm; Metastatic Pancreatic Adenocarcinoma; Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis; Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable; Primary Myelofibrosis; Recurrent Acute Lymphoblastic Leukemia; Recurrent Acute Myeloid Leukemia; Recurrent Chronic Lymphocytic Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Recurrent Hematologic Malignancy; Recurrent Hodgkin Lymphoma; Recurrent Myelodysplastic Syndrome; Recurrent Myelodysplastic/Myeloproliferative Neoplasm; Recurrent Myeloproliferative Neoplasm; Recurrent Non-Hodgkin Lymphoma; Recurrent Plasma Cell Myeloma; Recurrent Small Lymphocytic Lymphoma; Refractory Acute Lymphoblastic Leukemia; Refractory Acute Myeloid Leukemia; Refractory Chronic Lymphocytic Leukemia; Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Refractory Chronic Myelomonocytic Leukemia; Refractory Hematologic Malignancy; Refractory Hodgkin Lymphoma; Refractory Malignant Solid Neoplasm; Refractory Myelodysplastic Syndrome; Refractory Myelodysplastic/Myeloproliferative Neoplasm; Refractory Non-Hodgkin Lymphoma; Refractory Plasma Cell Myeloma; Refractory Primary Myelofibrosis; Refractory Small Lymphocytic Lymphoma; Stage II Pancreatic Cancer AJCC v8; Stage III Pancreatic Cancer AJCC v8; Stage IV Pancreatic Cancer AJCC v8; Stage IV Prostate Cancer AJCC v8; Unresectable Pancreatic Adenocarcinoma
OHSU Knight Cancer Institute
 2020-04-01 PHASE1
NCT02398825 TERMINATED
Chronic Myeloid Leukemia; Chronic Phase; Adults
Gruppo Italiano Malattie EMatologiche dell'Adulto
 2016-06-23 PHASE2
NCT02627677 TERMINATED
Chronic Phase Chronic Myeloid Leukemia
Ariad Pharmaceuticals
 2015-12-31 PHASE3
NCT02776605 UNKNOWN
ALL
PETHEMA Foundation
 2016-06 PHASE2
NCT02467270 COMPLETED
Myeloid Leukemia, Chronic, Chronic Phase
Takeda
 2015-07-13 PHASE2
NCT01641107 COMPLETED
Philadelphia Positive; BCR-ABL Positive; Acute Lymphoblastic Leukemia
Incyte Corporation
 2014-12-04 PHASE2
NCT01207440 COMPLETED
Chronic Myeloid Leukemia; Ph+ Acute Lymphoblastic Leukemia
Ariad Pharmaceuticals
 2010-09-30 PHASE2
NCT03171389 UNKNOWN
GIST, Malignant; KIT Exon 13 Mutation; KIT Gene Mutation
Sebastian Bauer
 2017-03-22 PHASE2
NCT01667133 COMPLETED
Chronic Myeloid Leukemia (CML); Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)
Ariad Pharmaceuticals
 2012-08-31 PHASE1; PHASE2
NCT01888562 WITHDRAWN
Endometrial Neoplasms
Washington University School of Medicine
 2016-01
NCT01813734 COMPLETED
Non Small Cell Lung Cancer
Massachusetts General Hospital
 2013-09 PHASE2
NCT02265341 COMPLETED
Malignant Hepatobiliary Neoplasm
Mayo Clinic
 2014-12 PHASE2
NCT02779283 COMPLETED
Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia
OHSU Knight Cancer Institute
 2016-01-13 PHASE1
NCT02478164 COMPLETED
Glioblastoma
Dana-Farber Cancer Institute
 2015-07-13 PHASE2
NCT01620216 TERMINATED
Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome; Chronic Myelomonocytic Leukemia; Recurrent Acute Lymphoblastic Leukemia; Recurrent Acute Myeloid Leukemia; Refractory Acute Lymphoblastic Leukemia; Refractory Acute Myeloid Leukemia
OHSU Knight Cancer Institute
 2012-05-11 PHASE2
NCT01935336 COMPLETED
Adenocarcinoma of the Lung; Extensive Stage Small Cell Lung Cancer; Limited Stage Small Cell Lung Cancer; Recurrent Non-small Cell Lung Cancer; Recurrent Small Cell Lung Cancer; Stage IIIA Non-small Cell Lung Cancer; Stage IIIB Non-small Cell Lung Cancer; Stage IV Non-small Cell Lung Cancer
University of Colorado, Denver
 2013-09-24 PHASE2
NCT00660920 COMPLETED
Chronic Myelogenous Leukemia; Hematologic Malignancies
Ariad Pharmaceuticals
 2008-06 PHASE1
NCT01570868 TERMINATED
Leukemia
M.D. Anderson Cancer Center
 2012-04 PHASE2
NCT01838642 TERMINATED
Thyroid Neoplasms
National Institutes of Health Clinical Center (CC)
 2013-03 PHASE2
NCT01874665 COMPLETED
GIST
Ariad Pharmaceuticals
 2013-06-05 PHASE2
NCT01761747 TERMINATED
Non-Small Cell Lung Cancer, Head and Neck Cancer
Dana-Farber Cancer Institute
 2013-01 PHASE2; PHASE3
NCT01650805 TERMINATED
Chronic Myeloid Leukemia
Ariad Pharmaceuticals
 2012-06 PHASE3

(data from https://clinicaltrials.gov, updated on 2026-02-10)

Check the Ponatinib (AP24534) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Ponatinib is a potent multi-targeted tyrosine kinase inhibitor that binds to native and mutant BCR-ABL as well as VEGFR2, FGFR1, PDGFRα, and FLT3, thereby blocking kinase autophosphorylation and downstream oncogenic cascades. This targeted inhibition suppresses cell proliferation and abrogates resistance mechanisms, providing a strong mechanistic foundation for its clinical utility in treating Philadelphia chromosome-positive leukemias and other malignant conditions.

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.