Study title
PonaZero = Effect of Consolidation Treatment with Ponatinib on Treatment-free Remission Rate in Patients With Chronic Phase Chronic Myeloid Leukemia (CML) with Deep Molecular Response
Scientific title
Multicenter, open-label, randomized phase III pilot study to evaluate the effect of a one-year consolidation treatment with ponatinib at two-dose ranges on free-remission rate in patients with Philadelphia-positive chronic myeloid leukemia, who had previously achieved a deep molecular response (EudraCT no. 2017-004565-27)
Indication and most important inclusion criteria
This study includes patients who:
- have BCR-ABL positive chronic myeloid leukemia in chronic phase (CP-CML)
- have been on imatinib treatment for a minimum of 4 years with confirmed stable deep molecular response (MR4) for a minimum of 12 months prior to enrolment
- are at least 18 years old
- have not had a prior accelerated phase/blast crisis (AP/BC) or stem cell transplant (SCT)
- have an Eastern Co-Operative Group (ECOG) status of 0-2
Short description of intervention
This is a pilot study to determine the rate of successful treatment-free remission (TFR) in patients who achieved and maintained molecular response 4 (MR4) on ponatinib following at least 4 years of imatinib therapy. Patients who maintain MR4 during the 52-week ponatinib onsolidation phase can attempt stopping ponatinib and will continue in the 104-week TFR phase.
Type of study
7. Treatment discontinuation trials
Current status
Recruiting
Study sponsor
FUNDACIÓN TEÓFILO HERNANDO, Edificio Claid
Parque Científico de Madrid
Calle Faraday, 7, Campus de Cantoblanco, 28049 Madrid
Scientific lead / contact
Gutierrez, Garcia
Principal investigator
Multiple
Additional information
Study description in the EU Clinical Trials Register which is hosted by the European Medicines Agency (EMA)
Study centers / principal investigators
Spain
Badalona, Barcelona, 08916
Hospital Trials i Pujol
Dr. Blanca Xicoy
Barcelona, 08035
Hospital Vall D'Hebron
Dr. Guillermo Orti
Las Palmas De Gran Canaria, 35010
Hospital Universitario de Gran Canarias Dr. Negrin
Dr. Maria Teresa Gomez
Madrid, 28006
Hospital Unversitario de la Princesa
Juan Luis Steegmann, MD PhD
Madrid, 28034
Hospital Universitario Ramon y Cajal
Valentin Garcia Gutierrez, MD PhD
Madrid, 28041
Hospital Universitario Doce de Octubre
Rosa Ayala, MD PhD
Málaga, 29010
Hospital Regional de Malaga
Dr. Antonio Jimenez
Salamanca, 37007
Hospital Universitario de Salamanca
Fermin Sanchez-Guija, MD PhD
Valencia, 46010
Hospital Clinico Universitario de Valencia
Dr. Juan Carlos Hernandez Boluda
Chronic Myeloid Leukemia
also called: Chronic Myelogeneous Leukemia
A chronic disease of the blood and bone marrow that results from a transformation of a stem cell.
Inclusion criteria
Inlusion criteria define which subjects may participate in a clinical study. Study subjects must fulfill all inclusion criteria (e.g. with regard to sex, age, previous diseases). This ensures a uniform composition of the study population and minimizes the risk of influences that distort the study results.
Accelerated Phase
A phase of development of Chronic Myeloid Leukemia between chronic and blast phase. Untreated, the accelerated phase progresses to blast phase within a few months.
Chronic phase
The earliest phase of CML development.
Blast crisis
The third phase of development of CML after chronic and accelerated phases. It is characterized by the presence of increasing numbers of immature blood cells ("blasts") in the blood and bone marrow.
Open-label
A clinical trial in which researchers and participants know which treatment is being provided to each patient (opposite to "blinded trials" or "double-blind studies")
Indication
In medicine, a reason to use a certain diagnostic test, therapeutic procedure or medication. The opposite of indication is contraindication.
Ponatinib
Trade name: Iclusig, development name: AP24534; a third-generation tyrosine kinase inhibitor
Other names: AP24534|Iclusig.
Imatinib
Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.
Other names: Gleevec|Glivec
BCR-ABL
The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22
Chronic
Long-lasting, slowly developping
Blast
An immature white blood cell that normally represents an early phase of the development process of a blood stem cell in the bone marrow
ECOG
Eastern Cooperative Oncology Group Index to classify the quality of life of cancer patients on a scale ranging from 0 (fully active, able to carry on all predisease activities without restriction) to 5 (death).
Also often referred to as ECOG performance status.
CML
Chronic Myeloid Leukemia, also called Chronic Myelogenous Leukemia
A chronic disease of the blood and bone marrow that results from a transformation of a stem cell.
MR4
Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)
CHR
Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.
CAM
Complementary and Alternative Medicine
TFR
Abbreviation for Treatment-Free Remission. In CML is referred to having a stable deep molecular response without the need for ongoing Tyrosine Kinase Inhibitor (TKI) treatment after having been in deep molecular residual disease over a longer period of time.
TFR
Abbreviation for Treatment-Free Remission. In CML is referred to having a stable deep molecular response without the need for ongoing Tyrosine Kinase Inhibitor (TKI) treatment after having been in deep molecular residual disease over a longer period of time.
