Clinical trial · Interventional
Activity and Risk Profile of Ponatinib in Chronic Phase Patients With Chronic Myeloid Leukemia Resistant to Imatinib
Optimizing Ponatinib USe (OPUS). A GIMEMA Phase 2 Study of the Activity and Risk Profile of Ponatinib, 30 mg Once Daily, in Chronic Myeloid Leukemia (CML) Chronic Phase (CP) Patients Resistant to Imatinib
NCT02398825CI-TRIAL-00084823terminatedPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): low enrollment rate
Summary
Brief summary (as posted)
This study aims at evaluating the efficacy of treatment with ponatinib in patients with chronic myeloid leukemia who are in a chronic phase and who previously received treatment with imatinib but resulted to be resistant to it.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Adults | — | UNRESOLVED | — |
| Chronic Myeloid Leukemia | Chronic Myeloid Leukemia, BCR-ABL1 Positive | ALIAS | 0.90 |
| Chronic Phase | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Ponatinib | Drug | Ponatinib | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Ponatinib
- interventionNames
- Drug: Ponatinib
Primary outcomes (1)
- measure
- Number of patients with major cytogenetic response
- timeFrame
- After 52 weeks of ponatinib treatment start
- description
- Cytogenetic response (CyR) is defined based on the percentage of Ph pos metaphases, as evaluated by chromosome banding analysis (CBA) of at least 20 marrow cell metaphases: 1. Major Cytogenetic Response if Ph pos metaphases \< 35% 2. Complete (CCyR) if Ph pos metaphases 0 or FISH BCR-ABL1 nuclei minor or equal to 1% 3. Partial (PCyR) if Ph pos metaphases 1-34% 4. Minor (mCyR) if Ph pos metaphases 35-65% 5. Minimal or none (min/none CyR) if Ph pos metaphases \> 65% If marrow cell metaphases cannot be obtained or analysed, interphase fluorescence-in-situ-hybridization (FISH) can be used, but only to distinguish a CCyR (minor or equal to 1% positive nuclei out of at least 200 nuclei) from a non CCyR. FISH data cannot be used to classify a response as minimal, minor, or partial.
Secondary outcomes (10)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: 1. Cytogenetic and/or molecular confirmed diagnosis of Ph+ and/or BCR-ABL1+ CML 2. Age ≥ 18 years 3. Chronic phase CML 4. Prior treatment with imatinib, any dose 5. Resistance to imatinib, as defined by any one of the ELN 2013 failure criteria, as follows: * no complete hematologic response (CHR) at 3 months * no cytogenetic response (CyR) (Ph+ \> 95%) at 3 months * Less than partial CyR (PCyR, Ph+ \> 35%) at 6 months * BCR-ABL1 \> 10% at 6 months * Non complete CyR (CCyR) (Ph+ \> 0%) at 12 months * BCR-ABL1 \> 1% at 12 months * Loss of CHR, at any time * Loss of CCyR, at any time * Confirmed loss of major molecular response (MMR) (BCR-ABL1 bigger or equal to 0.1% in two consecutive tests, of which one bigger or equal to 1%), at any time * Any new BCR-ABL1 mutation, at any time 6. For females of childbearing potential, a negative pregnancy test must be documented prior to enrolment 7. An effective form of contraception with their sexual partners from enrolment through 4 months after the end of treatment 8. Signed written informed consent according to ICH/EU/GCP and national local laws prior to any study procedures 9. Willingness and ability to comply with scheduled visits and study procedures. Exclusion Criteria: 1. Accelerated or blastic phase CML 2. Patients previously treated with nilotinib or dasatinib 3. Patients with the T315I mutation 4. History of acute pancreatitis within 1 year of study or history of chronic pancreatitis or of alcohol abuse 5. Patients with history of acute myocardial infarction (AMI), unstable angina or coronary heart disease (CHD), congestive heart failure, cerebrovascular events (CVE) (stroke or transitory ischemic attack), or peripheral artery occlusive disease (PAOD) 6. Compelled to take medications that are known to be associated with Torsades de Pointes and/or with significant QTc prolongation 7. Pregnant or breastfeeding 8. Any condition or illness that, in the opinion of the Investigator, would compromise patient safety or interfere with the evaluation of the drug 9. Lack of informed consent
References
Publications (0)
Data not yet available
No reference posted for this study.