A Two-part Study to Assess the Safety and Preliminary Efficacy of Givinostat in Patients With Polycythemia Vera
Status:
Completed
Trial end date:
2017-09-25
Target enrollment:
Participant gender:
Summary
This is a two-part, multicenter, open label, non-randomized, phase Ib/II study to assess the
safety and tolerability, Maximum Tolerated Dose and preliminary efficacy of Givinostat in
patients with JAK2V617F positive Polycythemia Vera. Part A is the dose finding part while
Part B is assessing the preliminary efficacy. Patients will be enrolled either in Part A or
Part B and transition from one part to the other is not allowed.
Eligible patients for this study will have a confirmed diagnosis of Polycythemia Vera
according to the revised World Health Organization criteria. Only if the enrolment in Part A
is slow (i.e. < 5 patients enrolled in 3 months), eligibility for this part of the study may
be expanded to all patients with chronic myeloproliferative neoplasms.
Study therapy will be administered in 28 day cycles (4 weeks of treatment). Disease response
will be evaluated according to the European LeukemiaNet criteria after 3 and 6 cycles (i.e.
at weeks 12 and 24, respectively) of treatment with Givinostat for both parts of the study.
All phlebotomies performed in the first 3 weeks of treatment will not be counted to assess
the clinico-haematological response.
The study will last up to a maximum of 24 weeks of treatment. However, after completion of
the trial, all patients achieving clinical benefit will be allowed to continue treatment with
Givinostat (at the same dose and schedule) in a long-term study.
Safety will be monitored at each visit throughout the entire duration of the study. Treatment
will be administered on an outpatient basis and patients will be followed regularly with
physical and laboratory tests, as specified in the protocol; in case of hospitalization, the
treatment will be continued or interrupted according to the Investigators' decision.