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Long-term Study Evaluating the Effect of Givinostat in Patients With JAK2V617F Positive Chronic Myeloproliferative Neoplasms

Phase 2
18 Years
Open (Enrolling)
Chronic Myeloproliferative Neoplasms (cMPN)

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Trial Information

Long-term Study Evaluating the Effect of Givinostat in Patients With JAK2V617F Positive Chronic Myeloproliferative Neoplasms

Inclusion Criteria:

1. Patients must have completed Givinostat treatment on at least one core study in cMPN
(i.e. Study DSC/07/2357/28, Study DSC/08/2357/38 and/or any further core protocols
in cMPN), or Patients must be participating in a compassionate use program with

2. Patients must be able to provide informed consent and be willing to sign an informed
consent form;

3. Adult patients (age ≥ 18 years) of both genders with established diagnosis of JAK2
V617F positive cMPN according to the revised WHO criteria;

4. Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status <

5. Acceptable organ function within 7 days of initiating study drug;

6. Use of an effective means of contraception for women of childbearing potential and
men with partners of childbearing potential;

7. Willingness and capability to comply with the requirements of the study.

Exclusion Criteria:

1. Active bacterial or mycotic infection requiring antimicrobial treatment;

2. Pregnancy or nursing;

3. A clinically significant QTc prolongation at baseline (e.g. repeated demonstration of
a QTc interval > 450 ms);

4. Use of concomitant medications known to prolong the QTc interval;

5. Clinically significant cardiovascular disease including:

- Uncontrolled hypertension, myocardial infarction, unstable angina within 6
months from study start;

- New York Heart Association (NYHA) Grade II or greater congestive heart failure;

- History of any cardiac arrhythmia requiring medication (irrespective of its

- A history of additional risk factors for TdP (e.g., heart failure, hypokalemia,
family history of Long QT Syndrome);

6. History of virus infection including HIV, HBV and HCV;

7. Platelets count < 100 x109/L within 14 days before enrolment;

8. Absolute neutrophil count < 1.2 x109/L within 14 days before enrolment;

9. Serum creatinine > 2 xULN;

10. Total serum bilirubin > 1.5 xULN;

11. Serum AST/ALT > 3 xULN;

12. History of other diseases, metabolic dysfunctions, physical examination findings, or
clinical laboratory findings giving reasonable suspicion of a disease or condition
that contraindicates use of an investigational drug or that might affect
interpretation of the results of the study or render the patient at high risk from
treatment complications;

13. Any investigational drug other than Givinostat within 28 days before enrolment;

14. Patients with known hypersensitivity to the components of potential study therapy.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Long-term safety and efficacy

Outcome Description:

To obtain information on the long-term efficacy of Givinostat in patients with cMPN following core protocols or compassionate use program: Number of patients experiencing adverse events; Type, incidence, and severity of treatment-related adverse events, graded according to CTCAE v. 4.03. To determine the long term safety and tolerability of Givinostat in patients with cMPN following core protocols or compassionate use program: For PV and ET, Complete response (CR) and partial response (PR) rate according to the revised clinico-haematological European LeukemiaNet (ELN) response criteria; For MF, complete response, major response, moderate response and minor response rate according to European Myelofibrosis Network (EUMNET) response criteria. Note that these assessment will be repeated periodically (each 3 months) during the study. In fact, the treatment will continue up to Marketing Authorisation of Givinostat.

Outcome Time Frame:

3 months

Safety Issue:


Principal Investigator

Alessandro Rambaldi, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy


Italy: Ethics Committee

Study ID:




Start Date:

March 2013

Completion Date:

March 2016

Related Keywords:

  • Chronic Myeloproliferative Neoplasms (cMPN)
  • chronic myeloproliferative neoplasms
  • Polycythemia Vera
  • Essential Trombocytemia
  • Primary Myelofibrosis
  • Post-PV Myelofibrosis
  • Post-ET Myelofibrosis
  • Givinostat
  • ITF-2357
  • Neoplasms
  • Myeloproliferative Disorders