Myeloid Leukemia, Chronic, Chronic PhasePhase 2OPTICNCT02467270
Ponatinib in Participants With Resistant Chronic Phase Chronic Myeloid Leukemia (CP-CML) to Characterize the Efficacy and Safety of a Range of Doses
A Phase 2 study testing Ponatinib in people with Myeloid Leukemia, Chronic, Chronic Phase, run by Takeda at 86 sites in 21 countries.
Trial summary
The purpose of this study is to characterize the efficacy of ponatinib administered in 3 starting doses (45 mg, 30 mg, and 15 mg daily) in participants with CP-CML who are resistant to prior tyrosine-kinase inhibitor (TKI) therapy or have T315I mutation, as measured by <=1 % Breakpoint Cluster Region-Abelson… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myeloid Leukemia, Chronic, Chronic Phase
- What is being tested?
- Ponatinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 283 participants
- Where
- 86 sitesin 21 countries
- Study dates
- 13 Jul 2015to 23 Apr 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 >= 10010^9/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
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
- Have adequate renal function as defined by the following criterion:
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- o] Serum creatinine <=1.5ULN for institution o] Estimated creatinine clearance >=30 milliliter per minute (mL/min) (Cockcroft-Gault formula)
- Have adequate hepatic function as defined by the following criteria:
- o] Total serum bilirubin <=1.5ULN, unless due to Gilbert's syndrome o] Alanine transaminase (ALT) <=2.5ULN, or <=5ULN if leukemic infiltration of the liver is present o] Aspartate transaminase (AST) <=2.5ULN, or <=5ULN if leukemic infiltration of the liver is present
- Have normal pancreatic status as defined by the following criterion:
- o] Serum lipase and amylase <=1.5ULN
- 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.
- Have a negative pregnancy test documented prior to enrollment (for females of childbearing potential).
- 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).
- Provide written informed consent.
- Be willing and able to comply with scheduled visits and study procedures.
- 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.
Who cannot join
- 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.
- 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.
- 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.
- 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).
- Are taking medications with a known risk of Torsades de Pointes.
- Have previously been treated with ponatinib.
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- 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.
- 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
- 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.
- Have poorly controlled diabetes defined as HbA1c values of >7.5%. Participants with preexisting, well-controlled diabetes are not excluded.
- Have a significant bleeding disorder unrelated to CML.
- Have a history of alcohol abuse.
- Have a history of either acute pancreatitis within 1 year of study enrollment or of chronic pancreatitis.
- Have malabsorption syndrome or other gastrointestinal illness that could affect oral absorption of study drug.
- 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.
- Are pregnant or lactating.
- 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.
- Have an active infection which requires intravenous antibiotics.
- Have a known history of human immunodeficiency virus infection; testing is not required in the absence of prior documentation or known history.
- Have any condition or illness that, in the opinion of the investigator, would compromise participant safety or interfere with the evaluation of the drug.
- Have hypersensitivity to the ponatinib active substance or to any of its inactive ingredients.
Trial locations and contacts
86 sites in 21 countries.
86 sites in 21 countries
United States16 sites
Michigan Medicine
Ann Arbor, Michigan, 48109
Emory University Winship Cancer Institute
Atlanta, Georgia, 30322
University of Maryland Medical Center
Baltimore, Maryland, 21201
Cleveland Clinic Taussig Cancer Institute Main Campus
Cleveland, Ohio, 44195
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, 48201
Duke University Medical Center
Durham, North Carolina, 27710
Hackensack University Medical Center
Hackensack, New Jersey, 07601
MD Anderson Cancer Center
Houston, Texas, 77030
Indiana Blood & Marrow Transplantation
Indianapolis, Indiana, 46237
University of Minnesota Medical School
Minneapolis, Minnesota, 55455
Memorial Sloan-Kettering Cancer Center - New York
New York, New York, 10065
NewYork-Presbyterian Weill Cornell Medical Center
New York, New York, 10065
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Abramson Cancer Center
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
University of Utah Huntsman Cancer Institute
Salt Lake City, Utah, 84112
Russia9 sites
Chelyabinsk Regional Clinical Hospital
Chelyabinsk, 454076
Kemerovo Regional Clinical Hospital
Kemerovo, 650066
GBUZ Moscow Clinical Scientific Center DZM
Moscow, 111123
Russian Academy of Medical Science
Moscow, 125167
Rostov State Medical University
Rostov-on-Don, Rostov Oblast, 344022
FGU Russian Scientific Research Institute of Hematology and Transfusiology
Saint Petersburg, 191024
Almazov Federal North-West Medical Research Centre of Department of Health of Russian Federation
Saint Petersburg, 197341
Samara State Medical University
Samara, 443099
Saratov State Medical University
Saratov, 355018
France8 sites
Center Hospitalier Universitaire d'Angers
Angers, Pays de la Loire Region, 49933
Centre de Lutte Contre le Cancer - Institut Bergonie
Bordeaux, Aquitaine, 33076
Centre Hospitalier Regional Universitaire de Lille
Lille, Hauts-de-France, 59037
Centre Hospitalier Universitaire de Nantes Hotel Dieu
Nantes, Pays de la Loire Region, 44093
Centre Hospitalier Universitaire de Nice Hopital l'Archet
Nice, Provence-Alpes-Côte d'Azur Region, 06202
Centre Hospitalier Universitaire de Poitiers
Poitiers, Poitou-charentes, 86021
Institut Universitaire du Cancer de Toulouse Oncopole
Toulouse, Midi-pyrenees, 31059
Centre Hospitalier Universitaire de Nancy Hopital de Brabois
Vandœuvre-lès-Nancy, Lorraine, 54511
Germany8 sites
Uniklinik RWTH Aachen
Aachen, North Rhine-Westphalia, 52074
Charite Universitatsmedizin Berlin
Berlin, 13353
Universitaetsklinikum Essen
Essen, North Rhine-Westphalia, 45147
Universitatsklinikum Hamburg-Eppendorf
Hamburg, 20246
Universitatsklinikum Jena
Jena, Thuringia, 07747
Universitaetsklinikum Heidelberg
Mannheim, Baden-Wurttemberg, 68169
Universitatsmedizin Rostock
Rostock, Mecklenburg-Vorpommern, 18057
Universitatsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081
Italy7 sites
Azienda Ospedaliero - Universitaria Policlinico - Vittorio Emanuele
Catania, 95124
Azienda Ospedaliera Universitaria San Martino
Genova, 16132
Azienda Ospedaliera San Gerardo di Monza
Monza, Monza E Brianza, 20090
Azienda Ospedaliera Ospedali Riuniti Marche Nord
Pesaro, Pesaro E Urbino, 61100
Azienda Sanitaria Locale di Pescara Ospedale Civile Dello Spirito Santo
Pescara, 65124
Sapienza Universita Di Roma
Roma, 00161
AOUI - Ospedale Policlinico "Giambattista Rossi" di Borgo Roma
Verona, 37134
Spain6 sites
Hospital Clinic i Provincial de Barcelona
Barcelona, 08036
Hospital Universitario de Gran Canaria Doctor Nergrin
Las Palmas de Gran Canaria, LAS Palmas, 35010
Hospital Universitario de La Princesa
Madrid, 28006
Hospital Universitario Ramon Y Cajal
Madrid, 28034
Hospital Regional Universitario Carlos Haya
Málaga, Andalusia, 29010
Hospital Clinico Universitario de Valencia
Valencia, 46010
United Kingdom6 sites
Beatson West of Scotland Cancer Centre
Glasgow, Scotland, G12 0YN
Royal Liverpool University Hospital NHS Trust
Liverpool, England, L7 8XP
King's College Hospital NHS Foundation Trust
London, England, SE5 9RS
Imperial College Healthcare NHS Trust
London, England, W12 0NN
Nottingham City Hospital NHS Trust
Nottingham, England, NG5 1PB
Churchill Hospital
Oxford, England, OX3 7LE
Poland5 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, Pomeranian Voivodeship, 80-952
Malopolskie Centrum Medyczne
Krakow, Lesser Poland Voivodeship, 30-510
Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi
Lodz, Łódź Voivodeship, 93-510
Instytut Hematologii i Transfuzjologii
Warsaw, Masovian Voivodeship, 02-776
Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wroclawiu
Wroclaw, Lower Silesian Voivodeship, 50-367
Argentina3 sites
Fundaleu
Ciudad Autonoma de Buenos Aires, Buenos Aires, C1114AAN
Hospital General de Agudo Jose Maria Ramos Mejia
Ciudad Autonoma de Buenos Aires, Buenos Aires, C1221ADC
Hospital Italiano La Plata
La Plata, Buenos Aires, B1900AXI
Canada3 sites
Jewish General Hospital
Montreal, Quebec, H3T 1E2
Saskatchewan Cancer Agency
Regina, Saskatchewan, S4T 7T1
Princess Margaret Hospital - Toronto
Toronto, Ontario, M5G 2M9
Australia2 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Royal North Shore Hospital
Saint Leonards, New South Wales, 2065
Chile2 sites
Hospital del Salvador
Providencia, Santiago Metropolitan, 7500922
Centro de Investigaciones Clinicas Vina del Mar
Viña del Mar, Valparaiso, 2540364
Czechia2 sites
Fakultni Nemocnice Olomouc
Olomouc, 772 00
Ustav Hematologie a Krevni Transfuze Praha
Prague, Prague, 12820
Portugal2 sites
Instituto Portugues de Oncologia de Lisboa Francisco Gentil
Lisbon, 1090-023
Centro Hospitalar Sao Joao
Porto, 4200-319
South Korea1 site
The Catholic University of Korea, Seoul St. Mary's Hospital
Seoul, 6591
About this study
Official title: A Randomized, Open-label, Phase 2 Trial of Ponatinib in Patients With Resistant Chronic Phase Chronic Myeloid Leukemia to Characterize the Efficacy and Safety of a Range of Doses
The purpose of this study is to characterize the efficacy of ponatinib administered in 3 starting doses (45 mg, 30 mg, and 15 mg daily) in participants with CP-CML who are resistant to prior tyrosine-kinase inhibitor (TKI) therapy or have T315I mutation, as measured by <=1 % Breakpoint Cluster Region-Abelson Transcript Level using International Scale (BCR-ABL1IS) at 12 months.
Read the detailed description
The drug being tested in this study is ponatinib. This study will characterize the safety and efficacy of ponatinib over a range of 3 starting doses.
The study will enroll 276 participants in 3 cohorts and each cohort will have 92 participants. All the participants will be randomized to receive once-daily oral administration of 1 of 3 starting doses of ponatinib:
- Cohort A: 45 mg ponatinib tablet
- Cohort B: 30 mg ponatinib tablet
- Cohort C: 15 mg ponatinib tablet
The study is designed to consist of 2 periods: 24-cycle Main treatment period and optional treatment continuation period. Participants will be treated with their randomized dose of study drug in the Main Treatment Period until the occurrence of at least one of the following: absence of CHR by 3 months, absence of MCyR at 12 months, absence of <=1% BCR-ABL1IS at 12 months, loss of <=1% BCR-ABL1IS development of intolerance, or completion of all 24 cycles of treatment (whichever occurs first). Following completion of approximately 5 years or following early withdrawal, participants may enter into an optional treatment continuation period.
This multi-center trial will be conducted in the United States, United Kingdom, Republic of Korea, Spain, France, Taiwan, Australia, Canada, Italy, Chile, Japan, Germany, Argentina, Poland, Czech Republic, Denmark, Hong Kong, Portugal, Russia, Singapore, Switzerland, and Sweden. The overall time to participate in this study is approximately 96 months. Participants will make a final visit to the clinic approximately 30 days after the last dose of study treatment.
- Conditions
- Myeloid Leukemia, Chronic, Chronic Phase
- Being tested
- Ponatinib
- Keywords
- Chronic Phase Chronic Myeloid Leukemia, Molecular Response, CML, Leukemia, Leukemia, Myeloid, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Neoplasms by Histologic Type, Neoplasms, Myeloproliferative Disorders, Bone Marrow Diseases, Hematologic Diseases, CP-CML
- Medical subject headings
- Leukemia, Myeloid, Chronic-Phase, Leukemia, Leukemia, Myeloid, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Neoplasms by Histologic Type, Neoplasms, Myeloproliferative Disorders, Bone Marrow Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PonatinibDrug
Also called Iclusig, AP24534
Tablet, taken orally once daily.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Period: Cohort A: Ponatinib 45 milligrams (mg) | Experimental | Participants received ponatinib 45 mg orally once daily in each 28-day cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily. Receives: Drug: Ponatinib |
| Treatment Period: Cohort B: Ponatinib 30 mg | Experimental | Participants received ponatinib 30 mg orally once daily in each 28 day Cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily. Receives: Drug: Ponatinib |
| Treatment Period: Cohort C: Ponatinib 15 mg | Experimental | Participants received ponatinib 15 mg orally once daily in each 28 day Cycle. Receives: Drug: Ponatinib |
| Close-out Period: Cohort A: Ponatinib 45 mg | Experimental | Participants received ponatinib 45 mg orally once daily in each 28-day cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily. Receives: Drug: Ponatinib |
| Close-out Period: Cohort B: Ponatinib 30 mg | Experimental | Participants received ponatinib 30 mg orally once daily in each 28 day Cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily. Receives: Drug: Ponatinib |
| Close-out Period: Cohort C: Ponatinib 15 mg | Experimental | Participants received ponatinib 15 mg orally once daily in each 28 day Cycle. Receives: Drug: Ponatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Molecular Response (MR2: <=1% Breakpoint Cluster Region-Abelson Transcript Level) as Measured by the International Scale (BCR-ABL1IS) at Month 12
MR2 was defined as a ratio of reverse transcribed transcript of BCR-ABL to ABL <=1% Breakpoint Cluster Region-Abelson Transcript Level as measured by the International Scale (BCR-ABL1IS), equivalent to a 2-log reduction in transcript.
Measured: 12 months after the first dose of study treatment
Secondary outcomes (16)
Percentage of Participants With Major Molecular Response (MMR/MR3)
MMR/MR3 was defined as a ratio of reverse transcribed transcript of BCR-ABL to ABL ≤0.1% on the international scale (equivalent to a 3-log reduction in transcript).
Measured: 12, 24 and 60 months after the first dose of study treatment
Percentage of Participants With Major Cytogenetic Response (MCyR)
MCyR was defined as percentage of participants with complete cytogenetic response (CCyR) or partial cytogenetic response (PCyR). Cytogenetic response is the percentage of Philadelphia chromosome positive (Ph+) metaphases in bone marrow (BM). Response is further defined as MCyR: CCyR or PCyR, where CCyR: 0% Ph + metaphases; PCyR: >0 to 35% Ph + metaphases.
Measured: 12 months after the first dose of study treatment
Duration of Major Molecular Response (MMR/MR3)
Duration of MMR/MR3 was defined as the interval between the first assessment at which the criteria for <=0.1% MMR were met until the earliest date at which loss of <=0.1% MMR occurred, or the criteria for progression were met. Progression to accelerated phase (AP) was defined as: >= 15% and <30% blasts in peripheral blood or bone marrow or >=20% basophils in peripheral blood or bone marrow or >=30% blasts + promyelocytes in peripheral blood or bone marrow (but <30% blasts) or <100*109 platelets per liter (/L) in peripheral blood unrelated to therapy or cytogenetic, genetic evidence of clonal evolution, and no extramedullary disease. Progression to blast phase (BP) was defined as: >=30% blasts in peripheral blood or bone marrow or extramedullary disease other than hepatosplenomegaly.
Measured: Baseline up to approximately 8.9 years
Percentage of Participants With Adjusted Incidence Rates for Treatment Emergent Arterial Occlusive Events (AOEs), Venous Thrombotic Events (VTEs), Adverse Events (AEs), and Serious AEs (SAEs)
Percentage of participants with adjusted incidence rates who developed AOEs and VTEs were categorized according to arterial occlusive events and venous thrombotic events. AE is any untoward medical occurrence in participant administered medicinal investigational drug. Untoward medical occurrence does not necessarily have to have causal relationship with treatment. SAE is any untoward medical occurrence that results in death; is life-threatening; requires inpatient hospitalization or prolongation of present hospitalization; results in persistent or significant disability/incapacity; is congenital anomaly/birth defect/is medically important event that may not be immediately life-threatening/result in death or hospitalization, but may jeopardize participant/may require intervention to prevent one of other outcomes listed in definition above, or involves suspected transmission via a medicinal product of an infectious agent.
Measured: From first dose up to 30 days after last dose of the study drug (up to approximately 9.8 years)
Percentage of Participants With Complete Cytogenetic Response (CCyR)
Cytogenetic response was defined as the percentage of Ph+ metaphases in bone marrow (peripheral blood may not be used), with a review of a minimum of 20 metaphases. CCyR was defined as 0% Ph+ metaphases.
Measured: Month 12
Percentage of Participants With Molecular Response 4 (MR4) and Molecular Response 4.5 (MR4.5)
MR4 was defined as <=0.01% BCR-ABL1IS. MR 4.5was defined as <=0.0032% BCR-ALB1IS. MR4 and MR4.5 by each time point means the best outcome up to each time point after randomization.
Measured: Up to approximately 9.5 years
Percentage of Participants With Molecular Response 1 (MR1)
MR1 was defined as percentage of participants achieving a ratio of <=10% Breakpoint Cluster Region-Abelson (BCR-ABL1) transcripts on the international scale. MR1 is molecular response with 1-log reduction in transcript.
Measured: 3 months after the first dose of study treatment
Percentage of Participants With Complete Hematologic Response (CHR)
CHR was defined as achieving all of the following measurements: white blood cells (WBC) <= institutional upper limit of normal (ULN), platelets less than (<)450,000 per cubic millimeter (/mm^3), no blasts or promyelocytes in peripheral blood, <5% myelocytes plus metamyelocytes in peripheral blood, basophils in peripheral blood <5%, and no extramedullary involvement (including no hepatomegaly or splenomegaly).
Measured: 3 months after the first dose of study treatment
Percentage of Participants With Treatment Emergent AEs Leading to Treatment Discontinuation, Dose Reduction and Dose Interruption
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug.
Measured: Up to approximately 9.8 years
Kaplan-Meier Estimate of Duration of Response (DOR) of <=1% BCR-ABL1 IS (MR2) at Months 12 and 24
DOR (≤1% BCR-ABL1IS) is defined as interval between first assessment at which criteria for ≤1% BCR-ABL1IS are met until earliest date at which loss of ≤1% BCR-ABL1IS occurs, or criteria for progression accelerated phase (AP) or blast Phase (BP) of chronic myeloid leukemia (CML) are met. Loss of ≤1% BCR-ABL1IS is an increase to >1% of BCR-ABL1IS. Progression to AP: ≥15% and <30% blasts in peripheral blood or bone marrow or ≥20% basophils in peripheral blood or bone marrow or ≥30% blasts + promyelocytes in peripheral blood or bone marrow (but <30% blasts) or <100*109 platelets per liter in peripheral blood unrelated to therapy or cytogenetic, genetic evidence of clonal evolution, and no extramedullary disease. Progression to BP: ≥30% blasts in peripheral blood or bone marrow or extramedullary disease other than hepatosplenomegaly. Kaplan-Meier method was used for analysis of percentage of participants who achieved DOR of 12 and 24 months.
Measured: 12 and 24 months after the first dose of study treatment
Kaplan-Meier Estimate of Duration of Response (DOR) of Major Molecular Response (MMR/MR3)
Duration of MMR/MR3 is defined as interval between first assessment at which criteria for MMR are met until earliest date at which loss of MMR occurs, or criteria for progression (progression to AP or BP of CML) are met. Participants remaining in MMR will be censored at last date at which criteria for MMR are met. Loss of MMR is an increase to >0.1% of BCR-ABL1IS. Progression to AP: >= 15% and <30% blasts in peripheral blood or bone marrow or >=20% basophils in peripheral blood or bone marrow or >=30% blasts+promyelocytes in peripheral blood or bone marrow (but <30% blasts) or <100*109 platelets/L in peripheral blood unrelated to therapy or cytogenetic, genetic evidence of clonal evolution, and no extramedullary disease. Progression to BP: >=30% blasts in peripheral blood or bone marrow or extramedullary disease other than hepatosplenomegaly. Kaplan-Meier method was used for analysis of percentage of participants who achieved DOR of 12 and 24 months.
Measured: 12 and 24 months after the first dose of study treatment
Duration of Response in MR2 Responders
Duration of response in "responders" was defined as any participants who achieved ≤1% BCR-ABL1IS (MR2) at any time during the study. Responders were defined as those participants who met all of the following: were randomized and treated, responded at 12 months after the initiation of study treatment, and underwent baseline polymerase chain reaction (PCR) assessment.
Measured: Baseline up to approximately 8.9 years
Time to Response (MR2)
Time to response was defined as the time interval from the date of the first dose of the study drug until the initial observation of CR or PR for participants with confirmed CR/PR. Response was defined as any participants who achieved ≤1% BCR-ABL1IS (MR2) at any time during the study. CR for target lesion: disappearance of all extranodal lesions and all pathological lymph nodes must have decreased to <10 mm in short axis. CR for non-target lesion: Disappearance of all extranodal non-target lesions, all lymph nodes must be non-pathological in size (<10mm short axis) and normalization of tumor marker level. PR: at least a 30% decrease in the SLD of target lesions, taking as reference the Baseline sum diameters.
Measured: Baseline up to approximately 5 years
Percentage of Participants With Progression to Accelerated Phase (AP)-Chronic Myeloid Leukemia (CML) or Blast Phase (BP)-CML
Progression to AP is defined as: >=15% and <30% blasts in peripheral blood or bone marrow or >=20% basophils in peripheral blood or bone marrow or >=30% blasts + promyelocytes in peripheral blood or bone marrow (but <30% blasts) or <100*109 platelets/L in peripheral blood unrelated to therapy or cytogenetic, genetic evidence of clonal evolution, and no extramedullary disease. Progression to BP is defined as: >=30% blasts in peripheral blood or bone marrow or extramedullary disease other than hepatosplenomegaly.
Measured: From first dose date of study treatment up to approximately 8.9 years
Progression-free Survival (PFS)
PFS was defined as the interval between the first dose date of study treatment and the first date at which the criteria for progression were met (progression to the AP or BP of CML), or death due to any cause, censored at the last response assessment. Progression to AP was defined as: >=15% and <30% blasts in peripheral blood or bone marrow or >=20% basophils in peripheral blood or bone marrow or >=30% blasts + promyelocytes in peripheral blood or bone marrow (but <30% blasts) or <100*109 platelets/L in peripheral blood unrelated to therapy or cytogenetic, genetic evidence of clonal evolution, and no extramedullary disease. Progression to BP was defined as: >=30% blasts in peripheral blood or bone marrow or extramedullary disease other than hepatosplenomegaly.
Measured: Up to approximately 8.9 years
Overall Survival (OS)
OS was defined as the interval between the first dose of study treatment and death due to any cause, censored at the last contact date when the participant was alive.
Measured: Up to approximately 8.9 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2015 (estimated)
- Study start13 Jul 2015
- Primary completion29 Apr 2020
- Study completion23 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Myeloid Leukemia, Chronic, Chronic PhaseStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 283 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02467270
- Sponsor's protocol ID
- AP24534-14-203
- Other IDs
- 2014-001617-12, 15/LO/1192, U1111-1238-0007, 2024-514516-27-00, 2014
- Acronym
- OPTIC
- Status last verified
- Mar 2026
- First submitted
- 2 Jun 2015
- Last updated
- 17 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Publications
Kantarjian HM, Jabbour E, Deininger M, Abruzzese E, Apperley J, Cortes J, Chuah C, DeAngelo DJ, DiPersio J, Hochhaus A, Lipton J, Nicolini FE, Pinilla-Ibarz J, Rea D, Rosti G, Rousselot P, Shah NP, Talpaz M, Srivastava S, Ren X, Mauro M. Ponatinib after failure of second-generation tyrosine kinase inhibitor in resistant chronic-phase chronic myeloid leukemia. Am J Hematol. 2022 Nov;97(11):1419-1426. doi: 10.1002/ajh.26686. Epub 2022 Aug 30.
PubMed 36054756Cortes J, Apperley J, Lomaia E, Moiraghi B, Undurraga Sutton M, Pavlovsky C, Chuah C, Sacha T, Lipton JH, Schiffer CA, McCloskey J, Hochhaus A, Rousselot P, Rosti G, de Lavallade H, Turkina A, Rojas C, Arthur CK, Maness L, Talpaz M, Mauro M, Hall T, Lu V, Srivastava S, Deininger M. Ponatinib dose-ranging study in chronic-phase chronic myeloid leukemia: a randomized, open-label phase 2 clinical trial. Blood. 2021 Nov 25;138(21):2042-2050. doi: 10.1182/blood.2021012082.
PubMed 34407543
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-label, Phase 2 Trial of Ponatinib in Patients With Resistant Chronic Phase Chronic Myeloid Leukemia to Characterize the Efficacy and Safety of a Range of Doses
- ClinicalTrials.gov ID
- NCT02467270
- Acronym / sponsor ID
- OPTIC / AP24534-14-203
- Conditions
- Myeloid Leukemia, Chronic, Chronic Phase
- Keywords
- Chronic Phase Chronic Myeloid Leukemia, Molecular Response, CML, Leukemia, Leukemia, Myeloid, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Neoplasms by Histologic Type, Neoplasms
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 283 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 13 Jul 2015 (actual)
- Primary completion
- 29 Apr 2020 (actual)
- Study completion
- 23 Apr 2025 (actual)
- First posted
- 10 Jun 2015 (estimated)
- Results
- Posted 14 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to characterize the efficacy of ponatinib administered in 3 starting doses (45 mg, 30 mg, and 15 mg daily) in participants with CP-CML who are resistant to prior tyrosine-kinase inhibitor (TKI) therapy or have T315I mutation, as measured by <=1 % Breakpoint Cluster Region-Abelson Transcript Level using International Scale (BCR-ABL1IS) at 12 months.
Detailed description
The drug being tested in this study is ponatinib. This study will characterize the safety and efficacy of ponatinib over a range of 3 starting doses.
The study will enroll 276 participants in 3 cohorts and each cohort will have 92 participants. All the participants will be randomized to receive once-daily oral administration of 1 of 3 starting doses of ponatinib:
- Cohort A: 45 mg ponatinib tablet
- Cohort B: 30 mg ponatinib tablet
- Cohort C: 15 mg ponatinib tablet
The study is designed to consist of 2 periods: 24-cycle Main treatment period and optional treatment continuation period. Participants will be treated with their randomized dose of study drug in the Main Treatment Period until the occurrence of at least one of the following: absence of CHR by 3 months, absence of MCyR at 12 months, absence of <=1% BCR-ABL1IS at 12 months, loss of <=1% BCR-ABL1IS development of intolerance, or completion of all 24 cycles of treatment (whichever occurs first). Following completion of approximately 5 years or following early withdrawal, participants may enter into an optional treatment continuation period.
This multi-center trial will be conducted in the United States, United Kingdom, Republic of Korea, Spain, France, Taiwan, Australia, Canada, Italy, Chile, Japan, Germany, Argentina, Poland, Czech Republic, Denmark, Hong Kong, Portugal, Russia, Singapore, Switzerland, and Sweden. The overall time to participate in this study is approximately 96 months. Participants will make a final visit to the clinic approximately 30 days after the last dose of study treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Treatment Period: Cohort A: Ponatinib 45 milligrams (mg) | Experimental | Drug: PonatinibParticipants received ponatinib 45 mg orally once daily in each 28-day cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily. |
| Treatment Period: Cohort B: Ponatinib 30 mg | Experimental | Drug: PonatinibParticipants received ponatinib 30 mg orally once daily in each 28 day Cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily. |
| Treatment Period: Cohort C: Ponatinib 15 mg | Experimental | Drug: PonatinibParticipants received ponatinib 15 mg orally once daily in each 28 day Cycle. |
| Close-out Period: Cohort A: Ponatinib 45 mg | Experimental | Drug: PonatinibParticipants received ponatinib 45 mg orally once daily in each 28-day cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily. |
| Close-out Period: Cohort B: Ponatinib 30 mg | Experimental | Drug: PonatinibParticipants received ponatinib 30 mg orally once daily in each 28 day Cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily. |
| Close-out Period: Cohort C: Ponatinib 15 mg | Experimental | Drug: PonatinibParticipants received ponatinib 15 mg orally once daily in each 28 day Cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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 >= 10010^9/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
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
Key exclusion criteria (22 listed)
- 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.
- 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.
- 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.
- 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).
- Are taking medications with a known risk of Torsades de Pointes.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PonatinibAlso: Iclusig, AP24534Tablet, taken orally once daily. | Drug | Close-out Period: Cohort A: Ponatinib 45 mg, Close-out Period: Cohort B: Ponatinib 30 mg, Close-out Period: Cohort C: Ponatinib 15 mg, Treatment Period: Cohort A: Ponatinib 45 milligrams (mg), Treatment Period: Cohort B: Ponatinib 30 mg, Treatment Period: Cohort C: Ponatinib 15 mg |
Primary outcome
Percentage of Participants With Molecular Response (MR2: <=1% Breakpoint Cluster Region-Abelson Transcript Level) as Measured by the International Scale (BCR-ABL1IS) at Month 12
Time frame: 12 months after the first dose of study treatment
MR2 was defined as a ratio of reverse transcribed transcript of BCR-ABL to ABL <=1% Breakpoint Cluster Region-Abelson Transcript Level as measured by the International Scale (BCR-ABL1IS), equivalent to a 2-log reduction in transcript.
16 secondary outcomes
Percentage of Participants With Major Molecular Response (MMR/MR3)
Time frame: 12, 24 and 60 months after the first dose of study treatment
Percentage of Participants With Major Cytogenetic Response (MCyR)
Time frame: 12 months after the first dose of study treatment
Duration of Major Molecular Response (MMR/MR3)
Time frame: Baseline up to approximately 8.9 years
Percentage of Participants With Adjusted Incidence Rates for Treatment Emergent Arterial Occlusive Events (AOEs), Venous Thrombotic Events (VTEs), Adverse Events (AEs), and Serious AEs (SAEs)
Time frame: From first dose up to 30 days after last dose of the study drug (up to approximately 9.8 years)
Percentage of Participants With Complete Cytogenetic Response (CCyR)
Time frame: Month 12
Percentage of Participants With Molecular Response 4 (MR4) and Molecular Response 4.5 (MR4.5)
Time frame: Up to approximately 9.5 years
Percentage of Participants With Molecular Response 1 (MR1)
Time frame: 3 months after the first dose of study treatment
Percentage of Participants With Complete Hematologic Response (CHR)
Time frame: 3 months after the first dose of study treatment
Percentage of Participants With Treatment Emergent AEs Leading to Treatment Discontinuation, Dose Reduction and Dose Interruption
Time frame: Up to approximately 9.8 years
Kaplan-Meier Estimate of Duration of Response (DOR) of <=1% BCR-ABL1 IS (MR2) at Months 12 and 24
Time frame: 12 and 24 months after the first dose of study treatment
Kaplan-Meier Estimate of Duration of Response (DOR) of Major Molecular Response (MMR/MR3)
Time frame: 12 and 24 months after the first dose of study treatment
Duration of Response in MR2 Responders
Time frame: Baseline up to approximately 8.9 years
Time to Response (MR2)
Time frame: Baseline up to approximately 5 years
Percentage of Participants With Progression to Accelerated Phase (AP)-Chronic Myeloid Leukemia (CML) or Blast Phase (BP)-CML
Time frame: From first dose date of study treatment up to approximately 8.9 years
Progression-free Survival (PFS)
Time frame: Up to approximately 8.9 years
Overall Survival (OS)
Time frame: Up to approximately 8.9 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36054756Kantarjian HM, Jabbour E, Deininger M, Abruzzese E, Apperley J, Cortes J, Chuah C, DeAngelo DJ, DiPersio J, Hochhaus A, Lipton J, Nicolini FE, Pinilla-Ibarz J, Rea D, Rosti G, Rousselot P, Shah NP, Talpaz M, Srivastava S, Ren X, Mauro M. Ponatinib after failure of second-generation tyrosine kinase inhibitor in resistant chronic-phase chronic myeloid leukemia. Am J Hematol. 2022 Nov;97(11):1419-1426. doi: 10.1002/ajh.26686. Epub 2022 Aug 30.
- Derived from this study: PubMed 34407543Cortes J, Apperley J, Lomaia E, Moiraghi B, Undurraga Sutton M, Pavlovsky C, Chuah C, Sacha T, Lipton JH, Schiffer CA, McCloskey J, Hochhaus A, Rousselot P, Rosti G, de Lavallade H, Turkina A, Rojas C, Arthur CK, Maness L, Talpaz M, Mauro M, Hall T, Lu V, Srivastava S, Deininger M. Ponatinib dose-ranging study in chronic-phase chronic myeloid leukemia: a randomized, open-label phase 2 clinical trial. Blood. 2021 Nov 25;138(21):2042-2050. doi: 10.1182/blood.2021012082.
- Click here for more information about this trial in easy-to-understand language, including a Plain Language Summary of the results if the trial has been completed.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Takeda
86 sites in 21 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ponatinib in people with Myeloid Leukemia, Chronic, Chronic Phase, run by Takeda at 86 sites in 21 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 86 sites in United States, Russia, France, Germany, Italy and 16 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Molecular Response (MR2: <=1% Breakpoint Cluster Region-Abelson Transcript Level) as Measured by the International Scale (BCR-ABL1IS) at Month 12 (time frame: 12 months after the first dose of study treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02467270, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2026.