OPTIC = Ponatinib in resistant chronic phase CML [Africa, Asia, Australia, Europe, North- and South America]

Study title

OPTIC = Ponatinib in Patients With Resistant Chronic Phase Chronic Myeloid Leukemia (CML) to Characterize the Efficacy and Safety of a Range of Doses

Scientific title

OPTIC (Optimising Ponatinib Treatment In CML) – 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 (EudraCT 2014-001617-12, NCT02467270, AP24534-14-203)

Type of study

Therapy optimization trials

Current status

No longer recruiting

What is the purpose of the study

To characterize the efficacy of ponatinib administered in three starting doses (45mg, 30mg, and 15mg daily) in patients with CP-CML who are resistant to at least two TKIs, as measured by major cytogenetic response (MCyR) by 12 months.

Key inclusion criteria

This study includes patients who:
– have chronic myeloid leukemia in chronic phase (CP-CML) and are resistant to at least two prior tyrosine kinase inhibitors (TKI)
– are at least 18 years old, have an Eastern Co-Operative Group (ECOG) status of 0-2, have given written informed consent and comply with scheduled visits and study procedures
– have fully recovered from the acute effects of prior cancer therapy before initiation of study drug
– have adequate kidney, liver and pancreas function
– have a negative pregnancy test, use a highly effective form of contraception from randomization through at least 4 months after the end of treatment (for female and male patients who are fertile)

Additional criteria apply.

Key exclusion criteria

Where can I find additional information

Study description in the US register ClinicalTrials.gov, a service of the U. S. National Institutes of Health

Study sponsor

Ariad Pharmaceuticals (acquired by Takeda)

Scientific lead / contact

Takeda Study Registration Call Center 866-835-2233

Principal investigator

See site contact information

Study centers / principal investigators


Buenos Aires, Argentina, 1221
Hospital Ramos Mejia (Site 826)
Contact: Claudio Merlo
Principal Investigator: Elena B Moiraghi, MD

Caba, Buenos Aires, C1114AAN
Fundaleu (Site 825)
Contact: Miguel Frau
Principal Investigator: Carolina Pavlovsky, M.D.

La Plata, Buenos Aires, B1900AXI
Hospital Italiano La Plata


New South Wales
St. Leonards, New South Wales, 2065
Royal North Shore Hospital (Site 941)
Contact: Vicki Katsioulas
Principal Investigator: Christopher K Arthur, MD

Adelaide, 5000
Royal Adelaide Hospital, Haematology Clinical Trials (Site 951)
Principal Investigator: Professor Timothy Peter Hughes


Toronto, Ontario, M5G2M9
Princess Margaret Cancer Centre (Site 083)
Principal Investigator: Jeffrey Howard Lipton

Montreal, Quebec, H3T IE2
Jewish General Hospital (Site 129)
Contact: Marie Pascale Guay
Principal Investigator: Sarit Assouline, MD

Regina, Saskatchewan, S4T 1A5
Pasqua Hospital (Site 227)
Principal Investigator: Haji Chalchal, MD


Providencia, Santiago, 7500922
Hospital del Salvador (Site 829)
Contact: Maria Gianna Capurro
Principal Investigator: Maria Soledad Undurraga Sutton, MD

Vina del Mar, Valparaiso, 254-0364
Centro de Investigaciones Clínicas Viña del Mar (Site 828)
Contact: Gina Castagneto
Principal Investigator: Christine Rojas Hopkins, MD

Czech Republic
Olomouc, 77520
Fakultni nemocnice Olomoue (Site 515)
Principal Investigator: prof. Karel Indrak, MD, PhD

Praha 2, 128 20
Ustav Hematologie a Krevni Transfuze Praha (UHKT) (Site 516)
Contact: Jana Vedrova
Principal Investigator: Hana Klamova


Aarhus C, 8000
Haematologisk Afdeling, Arahus Universitehospital (Site 683)
Principal Investigator: Prof. Jesper Stentoft, M.D.


Angers Cedex 9, 44993 CHU Angers
Service Maladies du Sang (Site 685)
Principal Investigator: Dr. Martine Gardembas

Bordeaux, 33076
Centre de Lutte Contre le Cancer – Institut Bergonie (Site 772)
Principal Investigator: Dr. Gabriel Etienne

Lille, Nord, 59037 CEDEX Hopital Claude Huriez CHRU (Site 952)
Contact: Sylvie Brice
Principal Investigator: Valerie Coiteux, MD

Nantes Cedex, 44093
CHU Hotel-Dieu, Service d’Hematologie Clinique (Site 521)
Principal Investigator: Dr. Viviane Dubruille

Nice Cedex 3, 06202
Centre Hospitalier Universitaire de Nice Hopital l’Archet (Site 509)
Contact: Marie-Christine Rigualt
Principal Investigator: Laurence Legros

Poitiers, B.p. 577, 86021
CHRU de Poitiers, Department of Oncology-Hematology and Cell Therapy (Site 954)
Principal Investigator: Prof. Francois Guilhot

Toulouse Cedex 9, Midi-pyrenees, 31059
Institut Universitaire du Cancer de Toulouse Oncopole

Vandoeuvre-les-Nancy Cedex, 54511
University Teaching Hospital – CHU Brabois (Site 953)
Principal Investigator: Dr. Agnes-Paule Guerci-Bresler


Aachen, 52074
Universitätsklinikum Aachen, AOR (Site 513)
Contact: Sabrina Holst
Principal Investigator: Martina Crysandt, MD

Berlin, 13353
Universitätsmedizin Berlin – Charité (CVK) (Site 701)
Contact: Peggy Grill
Principal Investigator: Philipp Le Coutre, MD

Essen, 45147
Essen University Hospital (Site 686)
Contact: Ilona Vester
Principal Investigator: Jürgen Novotny, MD

Hamburg, 20246
University Cancer Center Hamburg (Site 524)
Hubertus Wald Tumorzentrum
Contact: Juliane Granzow
Principal Investigator: Philippe Schafhausen, MD

Jena, 07747
Universitätsklinikum Jena (Site 946)
Contact: Denise Mathes
Principal Investigator: Andreas Hochhaus, PhD, MD

Mannheim, 68169
University of Heidelberg (Site 947)
Contact: Regina Pleil-Losch
Principal Investigator: Susanne Saussele, MD

Rostock, 18057
Universitatsmedizin Rostock

Ulm, 89081
University Hospital of Ulm (Site 694)
Contact: Beate Böltau
Principal Investigator: Frank Stegelmann, MD

Hong Kong

Hong Kong, 852
Queen Mary Hospital


Catania, 95124
University Hospital Policlinico (Site 530)
Contact: Giovanna Pappalardo
Principal Investigator: Francesco Di Raimondo, MD

Genova, 16132
Azienda Ospedaliera Universitaria San Martino
V.O. Clinica Ematologica (Site 528)
Contact: Maria Attilia Grassi
Principal Investigator: Marco Gobbi

Monza, 20090
ASST San Gerardo Hospital (Site 355)
Contact: Dario Cerri
Principal Investigator: Carlo Gambacorti-Passerini, MD

Pesaro, Pesaro e Urbino, 61100
A.O. Ospedali Riuniti Marche Nord (Site 689)
Contact: Maurizio Nardelli
Principal Investigator: Giuseppe Visani, MD

Pescara, 65124
Pescara Civil Hospital (Site 688)
Contact: Giuseppina Di Florio
Principal Investigator: Paolo Di Bartolomeo, MD

Roma, 00161
University La Spienza (Site 511)
Contact: Enrica Arduini
Principal Investigator: Giuliana Alimena

Verona, 37134
Ospedale Borgo Roma – Verona (Site 687)
Contact: Giorgia Barbiero
Principal Investigator: Massimiliano Bonifacio, MD

Korea, Republic of

Seoul, Korea, Republic of, 137-701
The Catholic University of Korea Seoul
St. Mary’s Hospital (Site 938)
Principal Investigator: Dong-Wook Kim, M.D., PhD


Gdansk, Pomorskie, Poland, 80-952
Uniwersyteckie Centrum Kliniczne

Krakow, 30-510
Malopolskie Medical Center (Site 546)
Contact: Marta Kowalowka
Principal Investigator: Tomasz Sacha, MD

Łódź, Poland, 93-510
Klinika Hematologii Uniwersytetu Medycznego w Łodzi (Site 550)
Contact: Magdalena Przybysz
Principal Investigator: Tadeusz Robak, MD

Warszawa, Poland, 02-776
Klinika Hematologii (Site 691)
Contact: Urszula Szustkiewicz
Principal Investigator: Krzysztof Warzocha, MD

Wrocław, Poland, 50-369
Independent Public Clinical Hospital No. 1 in Wroclaw (Site 547)
Contact: Mariusz Grajcar
Principal Investigator: Ewa Medras, MD


Lisboa, 1090-023
Instituto Portugues de Oncologia de Lisboa (Site 545)
Contact: Ana Inacio
Principal Investigator: Antonio M Almeida, MD/PhD

Porto, 4200-319
Centro Hospitalar de S. João (Site 559)
Contact: Raquel Ribeiro
Principal Investigator: Manuel S Simões

Russian Federation

Chelyabinsk, 454076
Chelyabinsk Regional Clinical Hospital

Kemerovo, 650066
Kemerovo Regional Clinical Hospital (Site 455)
Contact: Ludmila Chervova
Principal Investigator: Marina Kosinova, MD

Moscow, 111123
GBUZ Moscow Clinical Scientific Center (Site 453)
Contact: Galina Dudina
Principal Investigator: Mikhail Y Byakhov, MD

Moscow, 125167
Russian Academy of Medical Science
Haematological Scientific Center (Site 452)
Contact: Nemchenko Irina, MD
Principal Investigator: Anna Turkina, MD

Rostov-na-Donu, Rostov, 344022
Rostov State Medical University

Saint Petersburg, 191024
FGU Russian Scientific Research Institute of Hematology and Transfusiology

Saint-Petersburg, 197341
Federal State Budgetary Institution V. A. Almazov
Federal North-West Medical (Site 454)
Contact: Irina Shakhomirova
Principal Investigator: Elza Lomaia, MD

Samara, 443099
Samara State Medical University

Saratov, Russian Federation, 355018
Saratov State Medical University


Outram Road, Singapore, 169608
Singapore General Hospital (Site 939)
Principal Investigator: Dr. Charles Chuah Thuan Heng


Barcelona, Cataluna, 08036
Hospital Clinic, Service de Hematologia (Site 963)
Principal Investigator: Francisco Cervantes

Las Palmas de Gran Canaria, Islas Canarias, 35010
Hospital Universitario de Gran Canaria Dr Negrin (Site 692)
Contact: Mireya A Lopez
Principal Investigator: Maria Teresa Gomez Casares, MD

Madrid, 28006
Hospital Universitario de la Princesa
Hematology Service, 2a Planta (Site 555)
Principal Investigator: Juan Luis Steegmann Olmedillas

Madrid, 28034
Hospital Universitario Ramon y Cajal (Site 538)
Principal Investigator: Valentin Garcia Gutierrez
Hospital Clinico Universitario de Valencia (Site 964)

Malaga, Andalucia, 29010
Hospital Regional Universitario Carlos Haya (Site 698)
Principal Investigator: Antonio Jimenez Velasco

Valencia, Spain, 46010
Hospital Clinico Universitario de Valencia (Site 964)
Principal Investigator: Juan Carlos Hernandez Boluda


Uppsala, SE-751 85
University Hospital Uppsala (Site 945)
Contact: Aime Mukenyuzi
Principal Investigator: Ulla Olsson-Stromberg, M.D., Ph.D.


Zurich, 8091
University Hospital Zurich (Site 562)
Contact: Isabell Pieper-Scholz
Principal Investigator: Stefan Balabanov, MD


Taipei, 100
National Taiwan University Hospital (Site 979)
Principal Investigator: Jih-Luh Tang, M.D., PhD.

United Kingdom

Glasgow, G12 0YN
NHS Greater Glasgow & Clyde
The Beatson West of Scotland Cancer Centre (Site 797)
Principal Investigator: Professor Tessa Holyoake

Liverpool, L7 8XP
The Royal Liverpool University Hospital (Site 969)
Principal Investigator: Professor Richard Clark

London, SE5 9RS
King’s College Hospital NHS Foundation Trust (Site 693)
Principal Investigator: Dr. Hugues de Lavallade

London, W12 0NN
Imperial College Healthcare NHS Trust (Site 967)
Contact: Regina Storch
Principal Investigator: Jane F Apperley, MD

Nottingham, NG5 1PB
The Centre for Clinical Haematology
Notthingham University Hospitals NHS Trust (Site 968)
ontact: Maria Scott
Principal Investigator: Jennifer Byrne

Oxford, OX3 7LE
Churchill Hospital

United States

Atlanta, Georgia, 30322
Emory University Hospital Winship Cancer Institute (Site 058)
Principal Investigator: Vamsi Kota, MD

Indianapolis, Indiana, 46237
Indiana Blood and Marrow Transplantation Clinic (Site 138)
Principal Investigator: Luke Akard, MD

Baltimore, Maryland, 21201
University of Maryland Medical Center (Site 040)
Principal Investigator: Maria Baer, MD

Ann Arbor, Michigan, 48109
University of Michigan Health System (Site 011)
Principal Investigator: Moshe Talpaz, MD

Detroit, Michigan, 48201
Karmanos Cancer Institute (Site 034)
Principal Investigator: Charles Schiffer, MD

Minneapolis, Minnesota, 55455
University of Minnesota Medical School

Omaha, Nebraska, 68198-7680
University of Nebraska Medical Center (Site 235)
Principal Investigator: Lori Maness-Harris, MD

New Jersey
Hackensack, New Jersey, 07601
Hackensack University Medical Center (Site 128)
Principal Investigator: James McCloskey II

New York
New York, New York, 10065
Memorial Sloan Kettering Cancer Center (Site 078)
Contact: Gerald O’Neill
Principal Investigator: Michael Mauro, MD

New York, New York, 10065
Weill Medical College of Cornell University and New York Presbyterian Hospital (Site 006)
Contact: Rachael Alettie
Principal Investigator: Ellen Ritchie, MD

North Carolina
Durham, North Carolina, 27710
Duke University Medical Center (Site 003)
Contact: Cheryl M Maxey
Principal Investigator: Joseph Moore, MD

Cleveland, Ohio, 44195
Cleveland Clinic Foundation (Site 004)
Principal Investigator: Dr. Sudipto Mukherjee

Portland, Oregon, 97239
Oregon Health & Science University (Site 048)
Contact: Jeanne Liming
Principal Investigator: Michael Heinrich

Philadelphia, Pennsylvania, 19104
Abramson Cancer Center (Site 013)
Principal Investigator: Selena Luger, MD

Houston, Texas, 77030
MD Anderson Cancer Center (Site 005)
Principal Investigator: Jorge E. Cortes, MD

Salt Lake City, Utah, 84112
Huntsman Cancer Institute (Site 043)
Principal Investigator: Michael Deiningeri, MD