Phase IIA Study of the HDAC Inhibitor ITF2357 in Patients With JAK-2 V617F Positive Chronic Myeloproliferative Diseases
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|ClinicalTrials.gov Identifier: NCT00606307|
Recruitment Status : Completed
First Posted : February 1, 2008
Results First Posted : December 3, 2019
Last Update Posted : December 3, 2019
To evaluate efficacy and safety of ITF2357 in the treatment of patients with JAK2V617F positive myeloproliferative diseases [Polycythemia Vera (PV), Essential Thrombocytosis (ET), Myelofibrosis (MF)]. Efficacy was evaluated by ad hoc haematological and clinical criteria for PV and ET, and by internationally established response criteria (EUMNET criteria) for MF. Safety was evaluated by number of subjects experiencing an Adverse Event (AE), type, frequency, severity, timing and relatedness of AEs, including changes in vital signs and clinical laboratory results.
To evaluate the JAK2 mutated allele burden by quantitative Real-Time Polymerase Chain Reaction (qRTPCR).
|Condition or disease||Intervention/treatment||Phase|
|Myeloproliferative Diseases||Drug: ITF2357||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||29 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase IIA Study of the Histone-deacetylase Inhibitor ITF2357 in Patients With JAK-2 V617F Positive Chronic Myeloproliferative Diseases|
|Study Start Date :||December 2007|
|Actual Primary Completion Date :||December 2008|
|Actual Study Completion Date :||December 2008|
Initial dose of 50 mg b.i.d. that was subsequently escalated to 50 mg t.i.d in case of lack of significant toxicity.
50 mg b.i.d. PO every day. More precisely, ITF2357 was supplied as 50 mg hard gelatine capsules for oral administration.
Other Name: Givinostat
- Number of Patients With Objective Responses (Complete, Major, Moderate or Minor Responses), in Terms of Best Overall Response [ Time Frame: Every single week from week 1 to week 24 of treatment ]
Patients with Objective Response were defined as those patients achieving a complete, major, moderate or minor (only for Myelofibrosis patients) response during the experimental treatment course.
The "best response" is reported hereunder by intensity of response.
- Change in JAK2 Mutated Allele Burden [ Time Frame: At screening, at week 12, at week 24, at the end of treatment (EOT) visit ]
This outcome was assessed by quantitative real time Polymerase Chain Reaction (RT PCR).
At each time point, the number of patients is the following:
Screening: N=29 Week 12: N=20 Week 24: N=18 EOT: N=24. End of treatment corresponds to the last visit performed before treatment discontinuation.
- Number of Subject Experiencing an Adverse Event [ Time Frame: At weekly visits (Days 8, 15, 22, 36, 43, 50, 64, 71, 78, 99, 127, 155); At monthly visits (Days 29, 57, 85 113, 141,169); at end of treatment visit ]
An adverse event (AE) is any untoward occurrence in a patient or clinical investigation subject administered with a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavourable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
The adverse events must to be followed to the end of study (28 days after the last study drug intake).
A serious AE (SAE) is defined as an untoward (unfavourable) medical occurrence that at any dose results in death, or is life-threatening or requires inpatient hospitalisation or prolongation of existing hospitalisation, or results in persistent or significant disability/incapacity or is a congenital anomaly/birth defect.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00606307
|Bergamo, Italy, 24158|
|IRCCS - Pol. San Matteo|
|Pavia, Italy, 27100|
|Study Director:||tiziano oldoni, MD||Italfarmaco|
|Principal Investigator:||Alessandro Rambaldi, MD||A.O. Ospedale Papa Giovanni XXIII|