Ponatinib in Newly Diagnosed Chronic Myeloid Leukemia (CML) (EPIC)

NCT ID: NCT01650805

Last Updated: 2014-11-17

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

307 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-06-30

Study Completion Date

2013-10-31

Brief Summary

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The purpose of this study is to compare the efficacy of ponatinib and imatinib in patients with newly diagnosed chronic myeloid leukemia (CML) in the chronic phase.

Detailed Description

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This multicenter, international, phase 3 trial will test the hypothesis that ponatinib is an effective treatment for newly diagnosed CP-CML patients when compared with standard imatinib.

Patients will be randomized in a 1:1 fashion, stratified by Sokal risk score at diagnosis (low, intermediate, high), to receive once daily oral administration of either ponatinib or imatinib. Efficacy measures include molecular, cytogenetic, and hematologic response rates at various timepoints; time to, duration of, and durability of responses; and survival follow-up. Safety measures include clinical laboratory testing, adverse event monitoring, vital signs, physical exams, ECGs, and ECHOs. Other measures include two patient-reported health outcomes questionnaires (FACT-Leu and EQ-5D-5L), determination of mutation status, and, for ponatinib only, measurement of steady-state plasma concentration. Accrual is expected to take approximately 2 years, and patients will be followed for survival for up to 8 years after the last patient's first dose; therefore, patient participation may last up to 10 years.

Conditions

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Chronic Myeloid Leukemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ponatinib

Group Type EXPERIMENTAL

ponatinib

Intervention Type DRUG

45 mg tablet, taken orally once daily

imatinib

Group Type ACTIVE_COMPARATOR

imatinib (Gleevec/ Glivec)

Intervention Type DRUG

400 mg tablet, taken orally once daily

Interventions

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ponatinib

45 mg tablet, taken orally once daily

Intervention Type DRUG

imatinib (Gleevec/ Glivec)

400 mg tablet, taken orally once daily

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. CP CML within 6 months of diagnosis

* CP-CML will be defined by (i) \<15% blasts in bone marrow; (ii) \<30% blasts plus promyelocytes in bone marrow; (iii) \<20% basophils in peripheral blood; (iv) ≥100 × 10\^9/L platelets (≥100,000/mm\^3); (v) No evidence of extramedullary disease except hepatosplenomegaly; AND (vi) No prior diagnosis of AP-CML or BP-CML
2. Cytogenetic assessment must demonstrate the BCR-ABL fusion by presence of the t(9;22) Philadelphia chromosome

* (a)Variant translocations are only allowed provided they are assessable for cytogenetic response utilizing conventional cytogenetic techniques; (b) Conventional chromosome banding must be performed; AND (c) A minimum of 20 metaphases must be assessable at entry
3. Eastern Cooperative Oncology Group (ECOG) Performance Status of 0, 1, or 2
4. Adequate hepatic function as defined by the following criteria:

(a) Total serum bilirubin ≤1.5 x upper limit of normal (ULN), unless due to Gilbert's syndrome; (b) Alanine aminotransferase (ALT) ≤2.5 × ULN; AND (c) Aspartate aminotransferase (AST) ≤2.5 × ULN
5. Adequate renal function as defined as defined by serum creatinine \<1.5 x ULN
6. Adequate pancreatic function as defined by serum lipase and amylase ≤1.5 × ULN

Exclusion Criteria

1. Received prior imatinib therapy
2. Received prior dasatinib therapy
3. Received prior nilotinib therapy
4. Received, for CML, any other systemic anticancer therapy, experimental therapy, or radiation therapy with the exception of anagrelide or hydroxyurea
5. Major surgery within 28 days prior to initiating therapy
6. History of bleeding disorder unrelated to CML
7. History of acute pancreatitis within 1 year of study or history of chronic pancreatitis
8. History of alcohol abuse
9. Have uncontrolled hypertriglyceridemia (triglycerides \>450 mg/dL)
10. Clinically significant, uncontrolled, or active cardiovascular disease, specifically including, but not restricted to:

1. Myocardial infarction, within 6 months prior to randomization
2. Unstable angina within 6 months prior to randomization
3. Congestive heart failure within 6 months prior to randomization
4. History of clinically significant (as determined by the treating physician) atrial arrhythmia or any ventricular arrhythmia
5. Any history of ventricular arrhythmia
6. Cerebrovascular accident or transient ischemic attack within 6 months prior to randomization
7. Any history of peripheral arterial occlusive disease requiring revascularization
8. Any history of venous thromboembolism including deep venous thrombosis or pulmonary embolism
11. Uncontrolled hypertension (diastolic blood pressure \>90 mm Hg; systolic \>140 mm Hg). Patients with hypertension should be under treatment on study entry to effect blood pressure control
12. Taking medications that are known to be associated with Torsades de Pointes
13. Ongoing or active infection. The requirement for intravenous (IV) antibiotics is considered active infection
14. Known history of human immunodeficiency virus (HIV). Testing is not required in the absence of history
15. Pregnant or breastfeeding
16. Malabsorption syndrome or other gastrointestinal illness that could affect oral absorption of study drugs
17. Diagnosed with or received anticancer therapy for another primary malignancy within 3 years prior to entry (except for non-melanoma skin cancer or cervical cancer in situ)
18. Any condition or illness that, in the opinion of the Investigator, would compromise patient safety or interfere with the evaluation of the drug
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ariad Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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US Oncology - Providence Health System, Site #167

Burbank, California, United States

Site Status

UCLA Department of Medicine, Site #027

Los Angeles, California, United States

Site Status

Bay Area Cancer Research Group, Site #156

Pleasant Hill, California, United States

Site Status

Bay Area Cancer Research Group, Site #157

Pleasant Hill, California, United States

Site Status

Rocky Mountain Cancer Centers, Site #191

Boulder, Colorado, United States

Site Status

Cancer Center of Central Connecticut, Site #147

Southington, Connecticut, United States

Site Status

Christiana Care Health Services, Site #155

Newark, Delaware, United States

Site Status

University Cancer Institute, Site #149

Boynton Beach, Florida, United States

Site Status

Florida Cancer Specialists, Site #180

Fort Meyers, Florida, United States

Site Status

Florida Cancer Specialists, Site #179

St. Petersburg, Florida, United States

Site Status

Emory University, Site #058

Atlanta, Georgia, United States

Site Status

John H. Stroger, Jr. Hospital of Cook County, Site #192

Chicago, Illinois, United States

Site Status

University of Chicago, Site #001

Chicago, Illinois, United States

Site Status

Loyola University Chicago, Site #054

Maywood, Illinois, United States

Site Status

Franciscan St. Francis Health, Site #138

Indianapolis, Indiana, United States

Site Status

University of Iowa Hospitals and Clinics, Site #050

Iowa City, Iowa, United States

Site Status

Siouxland Hematology-Oncology Associates, Site #198

Sioux City, Iowa, United States

Site Status

US Oncology - Cancer Center of Kansas, Site #168

Wichita, Kansas, United States

Site Status

Willis-Knighton Cancer Center, Site #196

Shreveport, Louisiana, United States

Site Status

University of Maryland, Greenebaum Cancer Center, Site #040

Baltimore, Maryland, United States

Site Status

Greater Baltimore Medical Center, Site #140

Baltimore, Maryland, United States

Site Status

St. Agnes Healthcare, Site #185

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital, Site #047

Boston, Massachusetts, United States

Site Status

Dana Farber Cancer Institute, Site #008

Boston, Massachusetts, United States

Site Status

University of Massachusetts Worcester, Site #152

Worcester, Massachusetts, United States

Site Status

University of Michigan Medical Center, Site #011

Ann Arbor, Michigan, United States

Site Status

Providence Cancer Institute, Site #197

Southfield, Michigan, United States

Site Status

Mayo Clinic, Site #044

Rochester, Minnesota, United States

Site Status

Oncology Research Park Nicollet Institute, Site #195

Saint Louis Park, Minnesota, United States

Site Status

Saint Luke's Hospital, Site #162

Kansas City, Missouri, United States

Site Status

Mercy Clinic - Cancer & Hematology, Site #151

Springfield, Missouri, United States

Site Status

Nebraska Hematology-Oncology, P.C., Site # 133

Lincoln, Nebraska, United States

Site Status

US Oncology - Comprehensive Cancer Center of Nevada, Site #169

Las Vegas, Nevada, United States

Site Status

John Theurer Cancer Center, Site #128

Hackensack, New Jersey, United States

Site Status

University of New Mexico Cancer Center, Site #166

Albuquerque, New Mexico, United States

Site Status

Maimonides Cancer Center, Site #177

Brooklyn, New York, United States

Site Status

Winthrop University Hospital, Site #153

Mineola, New York, United States

Site Status

Beth Israel Medical Center, Site #145

New York, New York, United States

Site Status

Mount Sinai School of Medicine, Site #189

New York, New York, United States

Site Status

Memorial Sloan-Kettering Cancer Center, Site #078

New York, New York, United States

Site Status

Weill Cornell Medical College, Site #006

New York, New York, United States

Site Status

New York Medical College, Site #146

Valhalla, New York, United States

Site Status

Southeastern Medical Oncology Center, Site #188

Goldsboro, North Carolina, United States

Site Status

Signal Point Clinical Research Center, Site #139

Middletown, Ohio, United States

Site Status

University of Oklahoma, Site #028

Oklahoma City, Oklahoma, United States

Site Status

Providence Cancer Center Oncology and Hematology Care Eastside, Site #194

Portland, Oregon, United States

Site Status

Kaiser Permanente Northwest, Site #200

Portland, Oregon, United States

Site Status

Oregon Health & Science University, Site #048

Portland, Oregon, United States

Site Status

Gettysburg Cancer Center, Site #160

Gettysburg, Pennsylvania, United States

Site Status

Western Pennsylvania Hospital, Site #159

Pittsburgh, Pennsylvania, United States

Site Status

Medical University of South Carolina, Site #148

Charleston, South Carolina, United States

Site Status

Carolina Hematology Oncology, Site #143

Sumter, South Carolina, United States

Site Status

Associates in Oncology & Hematology, Site #186

Chattanooga, Tennessee, United States

Site Status

Sarah Cannon Research Institute, Site #076

Nashville, Tennessee, United States

Site Status

US Oncology - Texas Oncology Austin, Site #172

Austin, Texas, United States

Site Status

US Oncology - Texas Oncology Dallas, Site #171

Dallas, Texas, United States

Site Status

University of Texas Southwestern Medical Center, Site #178

Dallas, Texas, United States

Site Status

Baylor College of Medicine, Site #063

Houston, Texas, United States

Site Status

US Oncology - Texas Oncology Midland, Site #173

Midland, Texas, United States

Site Status

US Oncology - Cancer Care Center of South Texas, Site #170

San Antonio, Texas, United States

Site Status

Huntsman Cancer Institute, Site #043

Salt Lake City, Utah, United States

Site Status

VCU Massey Cancer Center, Dalton Oncology Clinic, Site #069

Richmond, Virginia, United States

Site Status

Seattle Cancer Care Alliance, Site #100

Seattle, Washington, United States

Site Status

US Oncology - Northwest Cancer Specialists, Site #174

Vancouver, Washington, United States

Site Status

West Virginia University, Site #154

Morgantown, West Virginia, United States

Site Status

Green Bay Oncology, Ltd. / St. Mary's Hospital Medical Center, Site #193

Green Bay, Wisconsin, United States

Site Status

University of Wisconsin, Site #030

Madison, Wisconsin, United States

Site Status

Canberra Hospital, Site #971

Garran, Australian Capital Territory, Australia

Site Status

Royal North Shore Hospital, Site #941

Sydney, New South Wales, Australia

Site Status

Royal Adelaide Hospital, Site #951

Adelaide, South Australia, Australia

Site Status

The Peter MacCallum Cancer Center, Site #950

East Melbourne, Victoria, Australia

Site Status

Box Hill Hospital, Site #940

Melbourne, Victoria, Australia

Site Status

Royal Perth Hospital, Site #972

Perth, Western Australia, Australia

Site Status

Medizinische Universitat Wien / AKH, Universitatsklinik fur Inniere Medizin I, Site #561

Vienna, , Austria

Site Status

Clinique Universitaire de Saint-Luc, Department of Haematology, Site #508

Brussels, , Belgium

Site Status

UZ Brussel - Department Hematology, Site #544

Brussels, , Belgium

Site Status

UZ Gent - Department Hematology, Site #756

Ghent, , Belgium

Site Status

UZ Gasthuisberg - Department of Hematology, Site #700

Leuven, , Belgium

Site Status

University Health Network, Princess Margaret Hospital, Site #083

Toronto, Ontario, Canada

Site Status

Jewish General Hospital, Site #129

Montreal, Quebec, Canada

Site Status

Fakultni nemocnice Brno, Interni hematologicka a onkologicka klinika, Site #514

Brno, , Czechia

Site Status

FN Hradec Kralove, Site #517

Hradec Králové, , Czechia

Site Status

Fakultni nemocnice Olomouc, Hematoonkologicka klinika, Site #515

Olomouc, , Czechia

Site Status

Ustav hematologie a krevni transfuse, Site #516

Prague, , Czechia

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Helsinki University Central Hospital, Site #542

Helsinki, , Finland

Site Status

Institut Bergonie, Site #772

Bordeaux, , France

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CHRU de Brest, Hopital Morvan, Site #523

Brest, , France

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CHU Henri Mondor, Site #520

Créteil, , France

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Centre Hospitalier de Versailles, Site #958

Le Chesnay, , France

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Hospital Claude Huriez, Site #952

Lille, , France

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Institut Paoli Calmette, Site #519

Marseille, , France

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CHU de Brabois, Site #953

Nancy, , France

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CHU de Nantes, Site #521

Nantes, , France

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Service Hematologie - Hospital Archet I, Site #509

Nice, , France

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Hopital Saint-Louis, Site #957

Paris, , France

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Hospital Saint Antoine, Site #518

Paris, , France

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Centre Hospitalier Lyon Sud, Site #956

Pierre-Bénite, , France

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CHU de Poitiers, Site #954

Poitiers, , France

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CHU Purpan, Site #955

Toulouse, , France

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Universitätsklinikum Aachen, AÖR, Site #513

Aachen, , Germany

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Charite - Universitatsmedizin Berlin, Site #701

Berlin, , Germany

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Universitatsklinikum Koln-AOR, Site #525

Cologne, , Germany

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Universitatsklinikum Carl Gustav Carus an der TU Dresden, Site #526

Dresden, , Germany

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Universitatsklinikum Freiburg, Site #527

Freiburg im Breisgau, , Germany

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Universitatsklinikum Hamburg-Eppendorf, Site #524

Hamburg, , Germany

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Universitatsklinikum Jena, Site #946

Jena, , Germany

Site Status

Universitat Heidelberg, CML - Studienzentrale III. Medizinische Klinik, Site #947

Mannheim, , Germany

Site Status

Klinikum rechts der Isar, Site #949

München, , Germany

Site Status

Prince of Wales Hospital, Site #974

Hong Kong, , Hong Kong

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Queen Mary Hospital, Site #973

Hong Kong, , Hong Kong

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Unita Operativa di Ematologia con Trapianto, Site #529

Bari, , Italy

Site Status

Istituto di Ematologia "L. & A. Seragnoli", Site #959

Bologna, , Italy

Site Status

A.O. Universitaria Policlinico Vittorio Emanuele di Catania, Site #530

Catania, , Italy

Site Status

Clinica Ematologica, Site #528

Genova, , Italy

Site Status

Ospedale Niguarda Ca' Granda di Milano, Site #531

Milan, , Italy

Site Status

S.C. Ematologia, Site #960

Modena, , Italy

Site Status

San Gerardo Hospital, Site #961

Monza, , Italy

Site Status

U.O.C Ematologia con trapianto di midollo osseo, Site #560

Napoli, , Italy

Site Status

Universita Federico II, Site #510

Napoli, , Italy

Site Status

SCDU Medicina Interna II - Indirizzo Ematologico, Site #785

Orbassano, , Italy

Site Status

Dipartimento di Biotecnologie Cellulari ed Ematologia Universita La Sapienza - Policlinico Umberto I, Site #511

Rome, , Italy

Site Status

U.O. di Ematologia - Ospedale S. Eugenio, Site #962

Rome, , Italy

Site Status

VU Medical Centre - Department Haematology, Site #948

Amsterdam, , Netherlands

Site Status

Auckland City Hospital, Site #921

Grafton, Auckland, New Zealand

Site Status

Christchurch Hospital, Site #922

Christchurch, , New Zealand

Site Status

Waikato Hospital, Site #977

Hamilton, , New Zealand

Site Status

North Shore Hospital, Site #976

Takapuna, , New Zealand

Site Status

Klinika Hematologii i Transplantologii, Uniwersyteckie Centrum Medyczne, Site #548

Gdansk, , Poland

Site Status

Malopolskie Centrum Medyczne, Site #546

Krakow, , Poland

Site Status

Klinika Hematologii Wojewodzkiego Szpitala Specjalistycznego, Site #550

Lodz, , Poland

Site Status

Oddzial Hematologii, Site #551

Rzeszów, , Poland

Site Status

Katedra i Klinika Hematologii, Site #547

Wroclaw, , Poland

Site Status

Instituto Portugues de Oncologia, Site #545

Lisbon, , Portugal

Site Status

Fundacion de Investigacion de Diego, Site #199

San Juan, , Puerto Rico

Site Status

Singapore General Hospital, Site #939

Singapore, , Singapore

Site Status

Narodny onkologicky ustav, Site #532

Bratislava, , Slovakia

Site Status

Univerzitna nemocnica Martin, Site #533

Martin, , Slovakia

Site Status

The Catholic University of Korea, Site #938

Seocho-gu, Seoul, South Korea

Site Status

Complejo Hospitalario Universitario A Coruna, Hospital "Teresa Herrera," Site #554

A Coruña, , Spain

Site Status

Hospital Universitari Germans Trias i Pujol, Site #512

Badalona, , Spain

Site Status

Hospital Clinic, Site #963

Barcelona, , Spain

Site Status

Institut Catala d' Oncologia de Girona, S. de Hematologia Clinica, Site #734

Girona, , Spain

Site Status

Hospital Universitari Son Espases, Site #553

Islas Baleares, , Spain

Site Status

Hospital Universitario La Princesa, Site #555

Madrid, , Spain

Site Status

Hospital Gregorio Maranon, Site #536

Madrid, , Spain

Site Status

H.U. Ramon y Cajal, Site #538

Madrid, , Spain

Site Status

Hospital Universitario 12 de Octubre, Site #537

Madrid, , Spain

Site Status

Hospital La Paz, Site #966

Madrid, , Spain

Site Status

Hospital Universitario Central de Asturias, Site #535

Oviedo, , Spain

Site Status

Hospital Universitario de Salamanca, Site #965

Salamanca, , Spain

Site Status

Hospital Clinico Universitario de Valencia, Site #964

Valencia, , Spain

Site Status

Skane University Hospital, Site #944

Lund, , Sweden

Site Status

Karolinska University Hospital Huddinge, Site #534

Stockholm, , Sweden

Site Status

Karolinska University Hospital Solna, Site #763

Stockholm, , Sweden

Site Status

Uppsala University Hospital, Site #945

Uppsala, , Sweden

Site Status

Kantonsspital Aarau, Site #541

Aarau, , Switzerland

Site Status

Kantonsspital St. Gallen, Site #707

Sankt Gallen, , Switzerland

Site Status

Kaohsiung Chang Gung Memorial Hospital, Site #980

Kaohsiung City, , Taiwan

Site Status

China Medical University Hospital, Site #978

Taiching, , Taiwan

Site Status

National Taiwan University Hospital, Site #979

Taipei, , Taiwan

Site Status

Western General Hospital, Site #556

Edinburgh, , United Kingdom

Site Status

Kent and Medway Cancer Research Network, Site #558

Gillingham, , United Kingdom

Site Status

University of Glasgow, Site #797

Glasgow, , United Kingdom

Site Status

St. James University Hospital, Site #540

Leeds, , United Kingdom

Site Status

Royal Liverpool University Hospital, Site #969

Liverpool, , United Kingdom

Site Status

Hammersmith Hospital, Site #967

London, , United Kingdom

Site Status

Newcastle University, Site #970

Newcastle, , United Kingdom

Site Status

Norfolk & Norwich University Hospital Foundation Trust, Site #557

Norwich, , United Kingdom

Site Status

Nottingham University Hospitals NHS Trust, Site #968

Nottingham, , United Kingdom

Site Status

Oxford University Hospitals NHS Trust, Site #543

Oxford, , United Kingdom

Site Status

Countries

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United States Australia Austria Belgium Canada Czechia Finland France Germany Hong Kong Italy Netherlands New Zealand Poland Portugal Puerto Rico Singapore Slovakia South Korea Spain Sweden Switzerland Taiwan United Kingdom

References

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Hanley MJ, Diderichsen PM, Narasimhan N, Srivastava S, Gupta N, Venkatakrishnan K. Population Pharmacokinetics of Ponatinib in Healthy Adult Volunteers and Patients With Hematologic Malignancies and Model-Informed Dose Selection for Pediatric Development. J Clin Pharmacol. 2022 Apr;62(4):555-567. doi: 10.1002/jcph.1990. Epub 2021 Dec 16.

Reference Type DERIVED
PMID: 34699069 (View on PubMed)

Lipton JH, Chuah C, Guerci-Bresler A, Rosti G, Simpson D, Assouline S, Etienne G, Nicolini FE, le Coutre P, Clark RE, Stenke L, Andorsky D, Oehler V, Lustgarten S, Rivera VM, Clackson T, Haluska FG, Baccarani M, Cortes JE, Guilhot F, Hochhaus A, Hughes T, Kantarjian HM, Shah NP, Talpaz M, Deininger MW; EPIC investigators. Ponatinib versus imatinib for newly diagnosed chronic myeloid leukaemia: an international, randomised, open-label, phase 3 trial. Lancet Oncol. 2016 May;17(5):612-21. doi: 10.1016/S1470-2045(16)00080-2. Epub 2016 Apr 12.

Reference Type DERIVED
PMID: 27083332 (View on PubMed)

Other Identifiers

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AP24534-12-301

Identifier Type: -

Identifier Source: org_study_id