Ponatinib: Ponatinib 45 mg/day x 4 weeks x 3 courses.
+/- chemotherapy:
* vincristine or
* L-VAMP (leucovorin, vincristine, aracytin, methotrexate, prednisone)
Study summary
This is a phase II interventional trial to evaluate if the use of ponatinib, with or without chemotherapy, can induce a molecular remission in MRD-positive patients, in patients in hematologic and extra-hematologic relapse and in the few patients who never achieved an hematologic remission after whatever prior treatment.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Ph+ ALL patients with evidence of MRD disease or in hematologic and extra-hematologic relapse/refractoriness after any previous treatment, will be considered eligible to enter the study.
2. Age ≥18 years old with no upper age limit.
3. Adequate hepatic function as defined by the following criteria:
* total serum bilirubin ≤1.5 x upper limit of normal (ULN), unless due to Gilbert's syndrome
* alanine aminotransferase (ALT) ≤2.5 × ULN
* aspartate aminotransferase (AST) ≤2.5 × ULN.
4. Adequate pancreatic function as defined by the following criterion:
\- serum lipase and amylase ≤1.5 × ULN.
5. For females of childbearing potential, a negative pregnancy test must be documented prior to enrollment.
6. Female and male patients who are fertile must agree to use an effective form of contraception with their sexual partners from enrollment through 4 months after the end of treatment.
7. Signed written informed consent according to ICH/EU/GCP and national local laws.
Exclusion Criteria:
1. WHO performance status ≤ 50% (Karnofsky) or ≥ 3 (ECOG).
2. Uncontrolled active HBV or HCV hepatitis, or AST/ALT ≥ 2.5 x ULN and bilirubine ≥ 1.5 x ULN not due to the disease.
3. History of acute pancreatitis within 1 year of study or history of chronic pancreatitis.
4. History of alcohol abuse.
5. Ongoing or active uncontrolled infections.
6. Uncontrolled hypertriglyceridemia (triglycerides \>450 mg/dL).
7. Clinically significant, uncontrolled or active cardiovascular disease, specifically including, but not restricted to:
* any history of myocardial infarction, stroke, or revascularization
* unstable angina or transient ischemic attack within 6 months prior to enrollment
* congestive heart failure within 6 months prior to enrollment, or left ventricular ejection fraction (LVEF) less than lower limit of normal per local institutional standards within 6 months prior to enrollment
* history of clinically significant (as determined by the treating physician) atrial arrhythmia
* any history of ventricular arrhythmia
* any history of venous thromboembolism including deep venous thrombosis or pulmonary embolism
* uncontrolled hypertension (diastolic blood pressure \>90 mm Hg; systolic \>140 mm Hg). Patients with hypertension should be under treatment on study entry to effect blood pressure control.
8. Taking medications that are known to be associated with Torsades de Pointes.
9. Taking any medications or herbal supplements that are known to be strong inhibitors of CYP3A4 within at least 14 days before the first dose of ponatinib.
10. Creatinine level \>2.5mg/dl or glomerular filtration rate (GFR) \<20 ml/min or proteinuria \>3.5 g/day.
11. Patients who are currently receiving treatment with any of the medications listed in Appendix E if the medications cannot be either discontinued or switched to a different medication prior to starting study drug. The medications listed in Appendix E have the potential to prolong QT.
Primary outcome measure(s)
MRD negativity/reduction rate — After 3 months of treatment Rate of patients who achieve a MRD negativity/MRD reduction following treatment with either Ponatinib alone or in combination with systemic chemotherapy
Trial sites (22)
Facility
City
Region
Status
Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica
Ancona
Italy
Area Vasta N. 5 Ascoli Piceno - S. Benedetto Del Tronto, Presidio Ospedaliero Av5 Osp. Gen. Prov.Le "C.G.Mazzoni" - Uoc Ematologia
Ascoli Piceno
Italy
Ao Di Rilievo Nazionale E Di Alta Specialità "San Giuseppe Moscati" - Avellino - Uoc Ematologia Con Unità Di Trapianto
Avellino
Italy
Aou Consorziale Policlinico - Bari - Uo Ematologia Con Trapianto
Bari
Italy
Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia
Bergamo
Italy
Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
Bologna
Italy
Asst Degli Spedali Civili Di Brescia - Uo Ematologia
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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