Study title
OPTIC-2L = A Study Comparing Ponatinib and Nilotinib in Patients With Chronic Myeloid Leukemia
Scientific title
A Randomized, Open-label Study of Ponatinib Versus Nilotinib in Patients With Chronic Myeloid Leukemia in Chronic Phase Following Resistance to Imatinib (AP24534-15-303) (EudraCT2015-001318-92, NCT02627677)
Indication and most important inclusion criteria
This study includes patients who:
- have chronic myeloid leukemia in chronic phase (CP-CML) and are resistant to first-line imatinib treatment
- are at least 18 years old, have an Eastern Co-Operative Group (ECOG) status of 0-1, have given written informed consent and comply with scheduled visits and study procedures
- have adequate kidney and liver function as well as normal pancreatic status
- 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)
- have fully recovered from the acute effects of prior cancer therapy (hydroxyurea or imatinib) before initiation of study drug
Additional criteria apply.
Short description of intervention
This is a study to demonstrate the efficacy and safety of 2 starting doses of ponatinib (30 and 15 mg once daily) as a treatment for CP-CML compared to nilotinib (400 mg twice daily), as measured by major molecular response (MMR) by 12 months.
Type of study
Second line trial after treatment failure
Current status
Active, not recruiting
Study sponsor
Takeda (Previously Ariad Pharmaceuticals)
Scientific lead / contact
Heinrich Farin, MD; Heinrich.Farin@ARIAD.com
Principal investigator
Dr. Violaine Havelange, Cliniques Universitaire Saint-Luc (Site 058), Bruxelles, Belgium, 1200
Additional information
Study description in the US register ClinicalTrials.gov, a service of the U. S. National Institutes of Health
Study centers / principal investigators
The study is being performed in the following countries:
Argentina, Austria, Belgium, Canada, Czech Republic, Denmark, France, Germany, Hong Kong, Hungary, Israel, Italy, Korea, Netherlands, Poland, Portugal, Romania, Russia, Singapore, Spain, Switzerland, UK
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.
Chronic phase
The earliest phase of CML development.
Randomization
In a clinical trial, patients are assigned by chance (often by a computer) to receive either one or the other treatment. This is to avoid bias in recruiting patients with specific characteristics into single arms of a trial.
Resistance
The ability to withstand the effects of a drug, e.g. resistance of cancer cells to a specific therapy.
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.
Nilotinib
Trade name: Tasigna, development name: AMN107, inhibits BCR-ABL tyrosine kinase. Authorized for marketing in the EU since 2007 for the treatment of CML and Ph+ALL.
Other names: |AMN107|Tasigna
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
Chronic
Long-lasting, slowly developping
Acute
Of sudden onset, severe; of short duration.
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.
MMR
Major Molecular Response (MMR), a level of response to CML therapy where the BCR-ABL ratio is equal or below 0,1%
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.
