Study title
PONAZA - A combination of ponatinib and subcutaneous azacitidine in chronic myelogenous leukemia patients in accelerated phase or in myeloid blast crisis
Scientific title
Open Label Phase 2 Study on the efficacy and tolerance of a combination of ponatinib and 5-azacitidine in chronic myelogenous leukemia in accelerated phase or in myeloid blast crisis (ClinicalTrials.gov NCT03895671)
Indication and most important inclusion criteria
This study includes patients who:
- are at least 18 years old
- have Philadelphia chromosome positive (Ph+) chronic myeloid leukemia in blast crisis (CML-BP) or accelerated phase (CML-AP)
- have an Eastern Co-Operative Group (ECOG) status of 0-3
- have adequate kidney and liver function and normal pancreas status
Other criteria may apply.
Short description of intervention
In this study, azacitidine will be given in addition to the tyrosine kinase inhibitor ponatinib to treat patients with either CML in advanced phase or myeloid blast crisis. The aim of the trial is to evaluate the activity of azacitidine in combination with ponatinib and to improve the survival of patients with CML in advanced phase and myeloid blast crisis. This treatment schedule will also reduce toxicity as compared to intensive chemotherapy + tyrosine kinase inhibitors.
Type of study
Treatment of advanced phases
Current status
Recruiting
Study sponsor
Hôpitaux de Versailles, France
Scientific lead / contact
Philippe Rousselot, Hôpitaux de Versailles
Principal investigator
Philippe Rousselot, Hôpitaux de Versailles
Additional information
Study description in the US register ClinicalTrials.gov, a service of the U. S. National Institutes of Health
Scientific abstract on the combination of a TKI and azacytidine in CML patients in advanced phases
Study centers / principal investigators
France
Amiens
Centre Hospitalier Universitaire D'Amiens
Delphine Lebon
Chambéry
Centre Hospitalier Metropole Savoie
Gian Matteo Pica
Grenoble
Centre Hospitalier Universitaire de Grenoble
Stéphane Courby
Paris
Hôpital St Louis
Emmanuel Raffoux
Versailles
Centre Hospitalier de Versailles
Philippe Rousselot
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.
Philadelphia chromosome
A certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene)
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.
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.
Chemotherapy
Chemotherapy is the use of drugs to treat cancer by destroying or slowing the growth of fast-growing cancer cells. The term chemotherapy also includes treatment with antibiotics.
Chromosome
A chromosome is a structure of DNA that carries the genetic makeup in the nucleus of the cell. Chromosomes contain giant chain molecules of DNA, coiled and folded as aggregates with specific proteins. Chromosomes ensure that during cell division the hereditary information is evenly distributed to the daughter cells. Normal human body cells have 46 chromosomes. Cancer cells can have a different number and/or structure of chromosomes.
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.
Toxicity
The quality of being poisonous; harm resulting from drugs, e.g. side effects of chemotherapy.
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.
Ph+
Abbreviation for "Philadelphia-Chromosome-positive", meaning the presence of a certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene).
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.
