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New dosing study aims to tame Drug-Resistant leukemia

NCT ID NCT02467270

Disease control Sponsor: Takeda Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested three different starting doses (45 mg, 30 mg, and 15 mg daily) of the drug ponatinib in 283 adults with chronic phase chronic myeloid leukemia (CML) that had stopped responding to other treatments or had a specific genetic mutation (T315I). The main goal was to see how well each dose reduced leukemia cells to very low levels after 12 months. Ponatinib is taken daily and is not a cure, but aims to control the disease long-term.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

283 people

The number who actually took part.

Started

Jul 2015

Finished

Apr 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Have chronic phase-chronic myelogenous leukemia/chronic myeloid leukemia (CP-CML) and have received at least two prior tyrosine kinase inhibitor (TKI) therapies and have demonstrated resistance to treatment OR have documented history of presence of T315I mutation after receiving any number of prior TKI. o\] The diagnosis of chronic myeloid leukemia (CML) will be made using standard hematopathologic and cytogenetic criteria; CP-CML will be defined by all of the following: i \<15% blasts in bone marrow ii \<30% blasts plus promyelocytes in bone marrow iii \<20% basophils in peripheral blood. iv \>= 100\*10\^9/liter (L) platelets (\>=100,000/mm\^3). v No evidence of extramedullary disease except hepatosplenomegaly vi No prior diagnosis of AP-CML, and BP-CML o\] Cytogenetic assessment at screening must demonstrate the BCR-ABL1 fusion by presence of the t(9;22) Philadelphia chromosome. i Variant translocations are only allowed provided they meet inclusion criterion 1d. o\] Resistance to prior TKI therapy is defined as follows (participants must meet at least 1 criterion): i Three months after the initiation of prior TKI therapy: No cytogenetic response (\>95% Ph+) or failure to achieve CHR or new mutation ii Six months after the initiation of prior TKI therapy: BCR-ABL1IS \>10% and/or Ph+ \>65% or new mutation iii Twelve months after the initiation of prior TKI therapy: BCR ABL1IS \>10% and/or Ph+ \>35% or new mutation iv At any time after the initiation of prior TKI therapy, the development of a new BCR-ABL1 kinase domain mutation(s) v At any time after the initiation of prior TKI therapy, the development of new clonal evolution vi At any time after the initiation of prior TKI therapy, the loss of CHR, or CCyR, or the confirmed loss of MMR in 2 consecutive tests, one of which has a BCR-ABL1IS transcript level of \>=1% or new mutation o\] \>1% of BCR-ABL1IS as shown by real-time polymerase chain reaction 2. Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2. 3. Have adequate renal function as defined by the following criterion: o\] Serum creatinine \<=1.5\*ULN for institution o\] Estimated creatinine clearance \>=30 milliliter per minute (mL/min) (Cockcroft-Gault formula) 4. Have adequate hepatic function as defined by the following criteria: o\] Total serum bilirubin \<=1.5\*ULN, unless due to Gilbert's syndrome o\] Alanine transaminase (ALT) \<=2.5\*ULN, or \<=5\*ULN if leukemic infiltration of the liver is present o\] Aspartate transaminase (AST) \<=2.5\*ULN, or \<=5\*ULN if leukemic infiltration of the liver is present 5. Have normal pancreatic status as defined by the following criterion: o\] Serum lipase and amylase \<=1.5\*ULN 6. Have normal QT interval corrected (Frederica) (QTcF) interval on screening electrocardiogram (ECG) evaluation, defined as QTcF of \<=450 milliseconds (ms) in males or \<=470 ms in females. 7. Have a negative pregnancy test documented prior to enrollment (for females of childbearing potential). 8. Agree to use a highly effective form of contraception with sexual partners from randomization through at least 4 months after the end of treatment (for female and male participants who are fertile). 9. Provide written informed consent. 10. Be willing and able to comply with scheduled visits and study procedures. 11. Have recovered from toxicities related to prior anticancer therapy to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v 4.0 grade \<=1. Exclusion Criteria: 1. Have used any approved TKIs or investigational agents within 2 weeks or 6 half-lives of the agent, whichever is longer, prior to receiving study drug. 2. Received interferon, cytarabine, or immunotherapy within 14 days, or any other cytotoxic chemotherapy, radiotherapy, or investigational therapy within 28 days prior to receiving the first dose of ponatinib, or have not recovered (\>grade 1 by NCI CTCAE, version 4.0) from AEs (except alopecia), due to agents previously administered. 3. Have undergone autologous or allogeneic stem cell transplant \<60 days prior to receiving the first dose of ponatinib; have any evidence of ongoing graft-versus-host disease (GVHD) or GVHD requiring immunosuppressive therapy. 4. Are being considered for hematopoietic stem cell transplant (HSCT) within 6-12 months of enrollment (note: ponatinib is not to be used as a bridge to HSCT in this trial). 5. Are taking medications with a known risk of Torsades de Pointes. 6. Have previously been treated with ponatinib. 7. Have active CNS disease as evidenced by cytology or pathology; in the absence of clinical CNS disease, lumbar puncture is not required. History itself of CNS involvement is not exclusionary if CNS has been cleared with a documented negative lumbar puncture. 8. Have clinically significant, uncontrolled, or active cardiovascular disease, specifically including, but not restricted to: o\] Any history of myocardial infarction (MI), unstable angina, cerebrovascular accident, or Transient Ischemic Attack (TIA) o\] Any history of peripheral vascular infarction, including visceral infarction o\] Any revascularization procedure, including the placement of a stent o\] Congestive heart failure (CHF) (New York Heart Association \[NYHA\] class III or IV) within 6 months prior to enrollment, or left ventricular ejection fraction (LVEF) less than lower limit of normal, per local institutional standards, within 6 months prior to enrollment o\] History of clinically significant (as determined by the treating physician) atrial arrhythmia or any history of ventricular arrhythmia o\] Venous thromboembolism, including deep venous thrombosis or pulmonary embolism, within 6 months prior to enrollment 9. Have uncontrolled hypertension (that is, \>150 and \>90 for systolic blood pressure (SBP) and diastolic blood pressure (DBP) respectively). Participants with hypertension should be under treatment at study entry to ensure blood pressure control. Those requiring 3 or more antihypertensive medications should be discussed with the medical monitor. 10. Have poorly controlled diabetes defined as HbA1c values of \>7.5%. Participants with preexisting, well-controlled diabetes are not excluded. 11. Have a significant bleeding disorder unrelated to CML. 12. Have a history of alcohol abuse. 13. Have a history of either acute pancreatitis within 1 year of study enrollment or of chronic pancreatitis. 14. Have malabsorption syndrome or other gastrointestinal illness that could affect oral absorption of study drug. 15. Have a history of another malignancy, other than cervical cancer in situ or basal cell or squamous cell carcinoma of the skin; the exception is if participants have been disease-free for at least 5 years, and are deemed by the investigator to be at low risk for recurrence of that malignancy. 16. Are pregnant or lactating. 17. Have undergone major surgery (with the exception of minor surgical procedures, such as catheter placement or BM biopsy) within 14 days prior to first dose of ponatinib. 18. Have an active infection which requires intravenous antibiotics. 19. Have a known history of human immunodeficiency virus infection; testing is not required in the absence of prior documentation or known history. 20. Have any condition or illness that, in the opinion of the investigator, would compromise participant safety or interfere with the evaluation of the drug. 21. Have hypersensitivity to the ponatinib active substance or to any of its inactive ingredients.

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Conditions

The condition(s) this trial relates to.

bone marrow disorder chronic myelogenous leukemia, BCR-ABL1 positive hematologic disorder leukemia Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid, Chronic-Phase myeloid leukemia Myeloproliferative Disorders neoplasm Neoplasms by Histologic Type

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Myeloid leukemia, chronic, chronic phase

Contacts and locations

Locations

  • AOUI - Ospedale Policlinico "Giambattista Rossi" di Borgo Roma

    Verona, 37134, Italy

  • Aarhus University Hospital

    Aarhus C, \Aarhus, DK-8000, Denmark

  • Abramson Cancer Center

    Philadelphia, Pennsylvania, 19104, United States

  • Akademiska Sjukhuset

    Uppsala, 751 85, Sweden

  • Almazov Federal North-West Medical Research Centre of Department of Health of Russian Federation

    Saint Petersburg, 197341, Russia

  • Azienda Ospedaliera Ospedali Riuniti Marche Nord

    Pesaro, Pesaro E Urbino, 61100, Italy

  • Azienda Ospedaliera San Gerardo di Monza

    Monza, Monza E Brianza, 20090, Italy

  • Azienda Ospedaliera Universitaria San Martino

    Genova, 16132, Italy

  • Azienda Ospedaliero - Universitaria Policlinico - Vittorio Emanuele

    Catania, 95124, Italy

  • Azienda Sanitaria Locale di Pescara Ospedale Civile Dello Spirito Santo

    Pescara, 65124, Italy

  • Barbara Ann Karmanos Cancer Institute

    Detroit, Michigan, 48201, United States

  • Beatson West of Scotland Cancer Centre

    Glasgow, Scotland, G12 0YN, United Kingdom

  • Center Hospitalier Universitaire d'Angers

    Angers, Pays de la Loire Region, 49933, France

  • Centre Hospitalier Regional Universitaire de Lille

    Lille, Hauts-de-France, 59037, France

  • Centre Hospitalier Universitaire de Nancy Hopital de Brabois

    Vandœuvre-lès-Nancy, Lorraine, 54511, France

  • Centre Hospitalier Universitaire de Nantes Hotel Dieu

    Nantes, Pays de la Loire Region, 44093, France

  • Centre Hospitalier Universitaire de Nice Hopital l'Archet

    Nice, Provence-Alpes-Côte d'Azur Region, 06202, France

  • Centre Hospitalier Universitaire de Poitiers

    Poitiers, Poitou-charentes, 86021, France

  • Centre de Lutte Contre le Cancer - Institut Bergonie

    Bordeaux, Aquitaine, 33076, France

  • Centro Hospitalar Sao Joao

    Porto, 4200-319, Portugal

  • Centro de Investigaciones Clinicas Vina del Mar

    Viña del Mar, Valparaiso, 2540364, Chile

  • Charite Universitatsmedizin Berlin

    Berlin, 13353, Germany

  • Chelyabinsk Regional Clinical Hospital

    Chelyabinsk, 454076, Russia

  • Churchill Hospital

    Oxford, England, OX3 7LE, United Kingdom

  • Cleveland Clinic Taussig Cancer Institute Main Campus

    Cleveland, Ohio, 44195, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Emory University Winship Cancer Institute

    Atlanta, Georgia, 30322, United States

  • FGU Russian Scientific Research Institute of Hematology and Transfusiology

    Saint Petersburg, 191024, Russia

  • Fakultni Nemocnice Olomouc

    Olomouc, 772 00, Czechia

  • Fundaleu

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1114AAN, Argentina

  • GBUZ Moscow Clinical Scientific Center DZM

    Moscow, 111123, Russia

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Hospital Clinic i Provincial de Barcelona

    Barcelona, 08036, Spain

  • Hospital Clinico Universitario de Valencia

    Valencia, 46010, Spain

  • Hospital General de Agudo Jose Maria Ramos Mejia

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1221ADC, Argentina

  • Hospital Italiano La Plata

    La Plata, Buenos Aires, B1900AXI, Argentina

  • Hospital Regional Universitario Carlos Haya

    Málaga, Andalusia, 29010, Spain

  • Hospital Universitario Ramon Y Cajal

    Madrid, 28034, Spain

  • Hospital Universitario de Gran Canaria Doctor Nergrin

    Las Palmas de Gran Canaria, LAS Palmas, 35010, Spain

  • Hospital Universitario de La Princesa

    Madrid, 28006, Spain

  • Hospital del Salvador

    Providencia, Santiago Metropolitan, 7500922, Chile

  • Imperial College Healthcare NHS Trust

    London, England, W12 0NN, United Kingdom

  • Indiana Blood & Marrow Transplantation

    Indianapolis, Indiana, 46237, United States

  • Institut Universitaire du Cancer de Toulouse Oncopole

    Toulouse, Midi-pyrenees, 31059, France

  • Instituto Portugues de Oncologia de Lisboa Francisco Gentil

    Lisbon, 1090-023, Portugal

  • Instytut Hematologii i Transfuzjologii

    Warsaw, Masovian Voivodeship, 02-776, Poland

  • Jewish General Hospital

    Montreal, Quebec, H3T 1E2, Canada

  • Kemerovo Regional Clinical Hospital

    Kemerovo, 650066, Russia

  • King's College Hospital NHS Foundation Trust

    London, England, SE5 9RS, United Kingdom

  • MD Anderson Cancer Center

    Houston, Texas, 77030, United States

  • Malopolskie Centrum Medyczne

    Krakow, Lesser Poland Voivodeship, 30-510, Poland

  • Memorial Sloan-Kettering Cancer Center - New York

    New York, New York, 10065, United States

  • Michigan Medicine

    Ann Arbor, Michigan, 48109, United States

  • National Taiwan University Hospital

    Taipei, 100, Taiwan

  • NewYork-Presbyterian Weill Cornell Medical Center

    New York, New York, 10065, United States

  • Nottingham City Hospital NHS Trust

    Nottingham, England, NG5 1PB, United Kingdom

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • Princess Margaret Hospital - Toronto

    Toronto, Ontario, M5G 2M9, Canada

  • Queen Mary Hospital

    Hong Kong, 852, Hong Kong

  • Rostov State Medical University

    Rostov-on-Don, Rostov Oblast, 344022, Russia

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Royal Liverpool University Hospital NHS Trust

    Liverpool, England, L7 8XP, United Kingdom

  • Royal North Shore Hospital

    Saint Leonards, New South Wales, 2065, Australia

  • Russian Academy of Medical Science

    Moscow, 125167, Russia

  • Samara State Medical University

    Samara, 443099, Russia

  • Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wroclawiu

    Wroclaw, Lower Silesian Voivodeship, 50-367, Poland

  • Sapienza Universita Di Roma

    Roma, 00161, Italy

  • Saratov State Medical University

    Saratov, 355018, Russia

  • Saskatchewan Cancer Agency

    Regina, Saskatchewan, S4T 7T1, Canada

  • Singapore General Hospital

    Singapore, 169856, Singapore

  • The Catholic University of Korea, Seoul St. Mary's Hospital

    Seoul, 6591, South Korea

  • Uniklinik RWTH Aachen

    Aachen, North Rhine-Westphalia, 52074, Germany

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia, 45147, Germany

  • Universitaetsklinikum Heidelberg

    Mannheim, Baden-Wurttemberg, 68169, Germany

  • Universitatsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • Universitatsklinikum Jena

    Jena, Thuringia, 07747, Germany

  • Universitatsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081, Germany

  • Universitatsmedizin Rostock

    Rostock, Mecklenburg-Vorpommern, 18057, Germany

  • University Hospital Zurich

    Zurich, 8091, Switzerland

  • University of Maryland Medical Center

    Baltimore, Maryland, 21201, United States

  • University of Minnesota Medical School

    Minneapolis, Minnesota, 55455, United States

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198, United States

  • University of Utah Huntsman Cancer Institute

    Salt Lake City, Utah, 84112, United States

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, Pomeranian Voivodeship, 80-952, Poland

  • Ustav Hematologie a Krevni Transfuze Praha

    Prague, Prague, 12820, Czechia

  • Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi

    Lodz, Łódź Voivodeship, 93-510, Poland