Chronic Myeloproliferative NeoplasmsPhase 2NCT01761968
Long-term Study Evaluating the Effect of Givinostat in Patients With Chronic Myeloproliferative Neoplasms
A Phase 2 study testing givinostat in people with Chronic Myeloproliferative Neoplasms, run by Italfarmaco at 15 sites in 3 countries.
Trial summary
This is a multicenter, open label, long-term study testing the long-term safety, tolerability and efficacy of givinostat in patients with Polycythemia Vera, Essential Thrombocythemia, primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis following core protocols in… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloproliferative Neoplasms
- What is being tested?
- givinostat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 15 sitesin 3 countries
- Study dates
- Mar 2013to Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have completed givinostat treatment on at least one core study in chronic myeloproliferative neoplasms, or patients must be participating in a compassionate use program with givinostat AND Patients must have tolerated previous givinostat treatment and achieved a clinical benefit at the end of core protocols or compassionate use program with givinostat, assessed by the Investigator according to the revised clinico-haematological ELN response criteria (for PV and ET) and EUMNET response criteria (for MF);
- Patients must be able to provide informed consent and be willing to sign an informed consent form;
- Adult patients (age ≥ 18 years) of both genders with established diagnosis of chronic myeloproliferative neoplasms according to the revised WHO criteria;
- Patients must have an Eastern Cooperative Oncology Group performance status < 3 at baseline;
- Acceptable organ function within 7 days of initiating study drug;
- Use of an effective means of contraception for women of childbearing potential and men with partners of childbearing potential;
- Willingness and capability to comply with the requirements of the study.
Who cannot join
- Pregnancy or nursing (lactating) women, where pregnancy is defined as the state of a female after conception, confirmed by a positive human Chorionic Gonadotropin (hCG) laboratory test (i.e. > 5 mIU/mL) and until the termination of gestation;
- A clinically significant corrected QT interval prolongation at baseline;
- Use of concomitant medications known to prolong the corrected QT interval;
- Clinically significant cardiovascular disease including:
- Uncontrolled hypertension, myocardial infarction, unstable angina at screening;
- New York Heart Association Grade II or greater congestive heart failure;
Show all 19Show fewer
- History of any cardiac arrhythmia requiring medication (irrespective of its severity);
- A history of additional risk factors for Torsade de Point;
- Active virus infection including human HIV, HBV and HCV;
- Platelets count < 100 x109/L within 14 days before enrolment (i.e. the receipt of the Patient ID);
- Absolute neutrophil count < 1.2 x109/L within 14 days before enrolment (i.e. the receipt of the Patient ID);
- Total serum bilirubin > 1.5 1.5 x ULN except in case of Gilbert's disease or pattern consistent with Gilbert's disease;
- Serum Aspartate aminotransferase/Alanine aminotransferase (AST/ALT) > 3 times the upper normal limit;
- Serum Cystatin C > 2 x ULN for two subsequent evaluations (i.e. if the value of serum Cystatin C is >2 x ULN, the test will be repeated once, and if the value is again > 2 x ULN, this becomes an exclusioncriterion);
- Uncontrolled hypertriglyceridemia at baseline, i.e. triglycerides ˃ 1.5 x ULN in fasting state.
- History and/or presence 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 or significantly alter the absorption of the study drug;
- Any investigational drug other than givinostat within 28 days before enrolment. Notably, the use of such medications within 28 days or 6 half-lives - whichever is longer - prior to the first dose of study drugs (i.e. Day 1) and during the study through all the study conduct (including any safety follow-up [FU] visit) is prohibited;
- Use of concomitant medications known to inhibit Pgp;
- Patients with known hypersensitivity to the components of potential study therapy.
Trial locations and contacts
15 sites in 3 countries.
15 sites in 3 countries
Italy11 sites
Azienda Ospedaliero-Universitaria Policlinico Consorziale, Bari
Bari, BA, 70124
Istituto Tumori Giovanni Paolo II IRCCS Ospedale Oncologico di Bari
Bari, BA, 70125
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, BG, 24127
Azienda Ospedaliero-Universitaria Careggi, Florence
Florence, FI, 50134
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico UOS Oncoematologia anziano
Milan, MI, 20122
Azienda Ospedaliera Universitaria Università degli Studi "Federico II", Naple
Naples, 80131
Fondazione I.R.C.C.S.-Policlinico San Matteo, Pavia
Pavia, PV, 27100
Azienda Unità Sanitaria Locale - Presidio Ospedaliero "Spirito Santo", Pescara
Pescara, PE, 65124
Azienda Ospedaliera "Bianchi-Melacrino-Morelli"
Reggio Calabria, RC, 89124
Università "Campus Bio-Medico", Rome
Rome, 00128
Ospedale San Bortolo, Vicenza
Vicenza, VI, 36100
Germany2 sites
Charite Research Organization GmbH
Berlin, 10117
Universitaetsklinikum Freiburg, Innere Medizin I - Haematologie und Onkologie
Freiburg im Breisgau, 79106
United Kingdom2 sites
Belfast City Hospital
Belfast, BT9 7BL
Royal Cornwall Hospital
Truro, TR1 3LJ
About this study
Official title: Long-term Study Evaluating the Effect of Givinostat in Patients With JAK2V617F Positive Chronic Myeloproliferative Neoplasms
This is a multicenter, open label, long-term study testing the long-term safety, tolerability and efficacy of givinostat in patients with Polycythemia Vera, Essential Thrombocythemia, primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis following core protocols in chronic myeloproliferative neoplasms and/or patient-named compassionate use program (if regulated/allowed by the local regulations, e.g. for Italy D.M. 8/5/2003 "Uso terapeutico di medicinale sottoposto a sperimentazione clinica" published on G.U. n. 173 of 28 July 2003, and the following amendments). Patients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. If patients previously received givinostat in combination with other drugs during a core protocol or a compassionate use program (if regulated/allowed by the local regulations, e.g. for Italy D.M. 8/5/2003 "Uso terapeutico di medicinale sottoposto a sperimentazione clinica" published on G.U. n. 173 of 28 July 2003, and the following amendments), they will be treated at the last tolerable dose of the combination. Assessment of safety and efficacy will be performed at each quarterly visit and each visit will also include laboratory tests and ECG examination. During the visits the clinical benefit will be assessed by Investigator according to the revised European LeukemiaNet response criteria (for PV and ET) and EUMNET response criteria (for MF). The dose of Givinostat will be modified for protocol specified toxicities. The treatment may continue up to Marketing Authorization of givinostat, currently planned in the next 5 years (note: only for Germany, this long-term study is initially limited up to 2 years of treatment). Patients may discontinue study treatment at any time and remain on study therapy as long as they derive clinical benefit. Safety will be monitored at each visit throughout the entire duration of the study. In case the approved label will not cover the whole study population, givinostat will be provided by the Sponsor to those patients not fulfilling the criteria for the approved label of the drug that are still deriving benefit from givinostat at the time of its commercial availability.
Read the detailed description
This is a multicenter, open label, long-term study testing the long-term safety, tolerability and efficacy of givinostat in patients with PV, ET, primary MF, Post-PV MF, Post-ET MF following core protocols in cMPN (i.e. Study DSC/07/2357/28, Study DSC/08/2357/38, Study DSC/12/2357/45 and/or all further core protocols in cMPN), and/or patient-named compassionate use program (if regulated/allowed by the local regulations, e.g. for Italy D.M. 8/5/2003 "Uso terapeutico di medicinale sottoposto a sperimentazione clinica" published on G.U. n. 173 of 28 July 2003, and the following amendments).
Eligible patients will have tolerated previous givinostat treatment and achieved a clinical benefit at the end of the core protocols; alternatively, eligible patients are participating in compassionate use program (if regulated/allowed by the local regulations, e.g. for Italy D.M. 8/5/2003 "Uso terapeutico di medicinale sottoposto a sperimentazione clinica" published on G.U. n. 173 of 28 July 2003, and the following amendments). In addition, patients must have tolerated previous givinostat treatment and achieved a clinical benefit at the end of core protocols or compassionate use program with givinostat, assessed by the Investigator according to the revised clinico-haematological ELN response criteria (for PV and ET) and EUMNET response criteria (for MF).
After providing informed written consent before undertaking any protocol-related procedure, an unique patient identification code (patient ID) will be assigned to each patient, will identify the patient throughout his/her participation in the study and can never be reused in case of premature drop-out.
Patients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. If patients previously received givinostat in combination with other drugs (e.g. hydroxyurea) during a core protocol or a compassionate use program , they will be treated at the last tolerable dose of the combination. In general, the dose of study drug may not be increased during this study. However, since the Maximum Tolerated Dose (MTD) of givinostat as chronic treatment has been recently identified and proven tolerable in Polycythenia Vera patients (i.e. 100 mg b.i.d.), selected patients may be eligible for escalation of their givinostat dose up to this level upon consultation with and written permission by Italfarmaco S.p.A. or its designee.
Assessment of safety and efficacy will be performed at each quarterly visit and each visit will also include laboratory tests and ECG examination. During the visits the clinical benefit will be assessed by Investigator according to the revised clinico-haematological ELN response criteria (for PV and ET) and EUMNET response criteria (for MF). Patients will be considered for continued treatment on the basis of their clinical response:
- if patients continue to derive clinical benefit from participating in the study, they will be allowed to continue study medication;
- if patients do not derive clinical benefit from participating in the study, they will discontinue study treatment.
For patients who previously participated in a compassionate use program with Givinostat the decision to continue treatment will be based on the assessment of continued clinical benefit derived from study therapy.
Of note, since this is a long-term study, at each quarterly visit the Investigator should include in his/her clinical evaluation the patient's benefit-risk assessment, taking into account both the clinical course of the patient in the Study until the visit time (i.e. the "clinical benefit", as above defined in this paragraph) and also any possible treatment option available at the time when the visit is performed.
The dose of givinostat will be modified for protocol specified toxicities. The treatment may continue up to Marketing Authorization of Givinostat, currently planned in the next 5 years (note: only for Germany, this long-term study is initially limited up to 2 years of treatment).
Patients may discontinue study treatment at any time and remain on study therapy as long as they derive clinical benefit. 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.
In case the approved label will not cover the whole study population, givinostat will be provided by the Sponsor to those patients not fulfilling the criteria for the approved label of the drug that are still deriving benefit from Givinostat at the time of its commercial availability.
- Conditions
- Chronic Myeloproliferative Neoplasms
- Being tested
- givinostat
- Keywords
- chronic myeloproliferative neoplasms, Polycythemia Vera, Essential Thrombocythemia, Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis, Givinostat
- Medical subject headings
- Myeloproliferative Disorders, Polycythemia Vera, Thrombocythemia, Essential, Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
givinostatDrug
Also called givinostat (ITF2357)
Patients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. Givinostat is a histone-deacetylases inhibitor. The product will be supplied as hard gelatine capsules for oral administration at the strength of 50 mg, 75 mg and/or 100 mg each.
If patients previously received givinostat in combination with other drugs during a core protocol or a compassionate use program, they will be treated at their last tolerable dose of this combination.
| Study group | Role | What they receive |
|---|---|---|
| givinostat | Experimental | Patients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. Givinostat is a histone-deacetylases inhibitor. The product will be supplied as hard gelatine capsules for oral administration at the strength of 50 mg, 75 mg and/or 100 mg each.
If patients previously received givinostat in combination with other drugs during a core protocol or a compassionate use program, they will be treated at their last tolerable dose of this combination. Receives: Drug: givinostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long-term safety and efficacy
To obtain information on the long-term safety and tolerability of givinostat in patients with chronic myeloproliferative neoplasms following core protocols or compassionate use program:
- Number of patients experiencing adverse events;
- Type, incidence, and severity of treatment-related adverse events.
To determine the long term efficacy of givinostat in patients with chronic myeloproliferative neoplasms following core protocols or compassionate use program:
- For Polycythemia Vera and Essential Thrombocythemia, Complete response and partial response rate according to the revised clinico-haematological European LeukemiaNet response criteria;
- For Myelofibrosis, complete response, major response, moderate response and minor response rate according to European Myelofibrosis Network 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.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2013 (estimated)
- Study startMar 2013
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloproliferative NeoplasmsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Italfarmaco
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alessandro Rambaldi, MD, Principal investigator, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01761968
- Sponsor's protocol ID
- DSC/11/2357/44
- Other IDs
- 2012-003499-37
- Status last verified
- Apr 2025
- First submitted
- 18 Dec 2012
- Last updated
- 30 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Study Evaluating the Effect of Givinostat in Patients With JAK2V617F Positive Chronic Myeloproliferative Neoplasms
- ClinicalTrials.gov ID
- NCT01761968
- Acronym / sponsor ID
- DSC/11/2357/44
- Conditions
- Chronic Myeloproliferative Neoplasms
- Keywords
- chronic myeloproliferative neoplasms, Polycythemia Vera, Essential Thrombocythemia, Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis, Givinostat
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- ItalfarmacoIndustry (pharma, biotech, devices)
- Study start
- Mar 2013 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 7 Jan 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, open label, long-term study testing the long-term safety, tolerability and efficacy of givinostat in patients with Polycythemia Vera, Essential Thrombocythemia, primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis following core protocols in chronic myeloproliferative neoplasms and/or patient-named compassionate use program (if regulated/allowed by the local regulations, e.g. for Italy D.M. 8/5/2003 "Uso terapeutico di medicinale sottoposto a sperimentazione clinica" published on G.U. n. 173 of 28 July 2003, and the following amendments). Patients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. If patients previously received givinostat in combination with other drugs during a core protocol or a compassionate use program (if regulated/allowed by the local regulations, e.g. for Italy D.M. 8/5/2003 "Uso terapeutico di medicinale sottoposto a sperimentazione clinica" published on G.U. n. 173 of 28 July 2003, and the following amendments), they will be treated at the last tolerable dose of the combination. Assessment of safety and efficacy will be performed at each quarterly visit and each visit will also include laboratory tests and ECG examination. During the visits the clinical benefit will be assessed by Investigator according to the revised European LeukemiaNet response criteria (for PV and ET) and EUMNET response criteria (for MF). The dose of Givinostat will be modified for protocol specified toxicities. The treatment may continue up to Marketing Authorization of givinostat, currently planned in the next 5 years (note: only for Germany, this long-term study is initially limited up to 2 years of treatment). Patients may discontinue study treatment at any time and remain on study therapy as long as they derive clinical benefit. Safety will be monitored at each visit throughout the entire duration of the study. In case the approved label will not cover the whole study population, givinostat will be provided by the Sponsor to those patients not fulfilling the criteria for the approved label of the drug that are still deriving benefit from givinostat at the time of its commercial availability.
Detailed description
This is a multicenter, open label, long-term study testing the long-term safety, tolerability and efficacy of givinostat in patients with PV, ET, primary MF, Post-PV MF, Post-ET MF following core protocols in cMPN (i.e. Study DSC/07/2357/28, Study DSC/08/2357/38, Study DSC/12/2357/45 and/or all further core protocols in cMPN), and/or patient-named compassionate use program (if regulated/allowed by the local regulations, e.g. for Italy D.M. 8/5/2003 "Uso terapeutico di medicinale sottoposto a sperimentazione clinica" published on G.U. n. 173 of 28 July 2003, and the following amendments).
Eligible patients will have tolerated previous givinostat treatment and achieved a clinical benefit at the end of the core protocols; alternatively, eligible patients are participating in compassionate use program (if regulated/allowed by the local regulations, e.g. for Italy D.M. 8/5/2003 "Uso terapeutico di medicinale sottoposto a sperimentazione clinica" published on G.U. n. 173 of 28 July 2003, and the following amendments). In addition, patients must have tolerated previous givinostat treatment and achieved a clinical benefit at the end of core protocols or compassionate use program with givinostat, assessed by the Investigator according to the revised clinico-haematological ELN response criteria (for PV and ET) and EUMNET response criteria (for MF).
After providing informed written consent before undertaking any protocol-related procedure, an unique patient identification code (patient ID) will be assigned to each patient, will identify the patient throughout his/her participation in the study and can never be reused in case of premature drop-out.
Patients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. If patients previously received givinostat in combination with other drugs (e.g. hydroxyurea) during a core protocol or a compassionate use program , they will be treated at the last tolerable dose of the combination. In general, the dose of study drug may not be increased during this study. However, since the Maximum Tolerated Dose (MTD) of givinostat as chronic treatment has been recently identified and proven tolerable in Polycythenia Vera patients (i.e. 100 mg b.i.d.), selected patients may be eligible for escalation of their givinostat dose up to this level upon consultation with and written permission by Italfarmaco S.p.A. or its designee.
Assessment of safety and efficacy will be performed at each quarterly visit and each visit will also include laboratory tests and ECG examination. During the visits the clinical benefit will be assessed by Investigator according to the revised clinico-haematological ELN response criteria (for PV and ET) and EUMNET response criteria (for MF). Patients will be considered for continued treatment on the basis of their clinical response:
- if patients continue to derive clinical benefit from participating in the study, they will be allowed to continue study medication;
- if patients do not derive clinical benefit from participating in the study, they will discontinue study treatment.
For patients who previously participated in a compassionate use program with Givinostat the decision to continue treatment will be based on the assessment of continued clinical benefit derived from study therapy.
Of note, since this is a long-term study, at each quarterly visit the Investigator should include in his/her clinical evaluation the patient's benefit-risk assessment, taking into account both the clinical course of the patient in the Study until the visit time (i.e. the "clinical benefit", as above defined in this paragraph) and also any possible treatment option available at the time when the visit is performed.
The dose of givinostat will be modified for protocol specified toxicities. The treatment may continue up to Marketing Authorization of Givinostat, currently planned in the next 5 years (note: only for Germany, this long-term study is initially limited up to 2 years of treatment).
Patients may discontinue study treatment at any time and remain on study therapy as long as they derive clinical benefit. 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.
In case the approved label will not cover the whole study population, givinostat will be provided by the Sponsor to those patients not fulfilling the criteria for the approved label of the drug that are still deriving benefit from Givinostat at the time of its commercial availability.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| givinostat | Experimental | Drug: givinostatPatients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. Givinostat is a histone-deacetylases inhibitor. The product will be supplied as hard gelatine capsules for oral administration at the strength of 50 mg, 75 mg and/or 100 mg each. If patients previously received givinostat in combination with other drugs during a core protocol or a compassionate use program, they will be treated at their last tolerable dose of this combination. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients must have completed givinostat treatment on at least one core study in chronic myeloproliferative neoplasms, or patients must be participating in a compassionate use program with givinostat AND Patients must have tolerated previous givinostat treatment and achieved a clinical benefit at the end of core protocols or compassionate use program with givinostat, assessed by the Investigator according to the revised clinico-haematological ELN response criteria (for PV and ET) and EUMNET response criteria (for MF);
- Patients must be able to provide informed consent and be willing to sign an informed consent form;
- Adult patients (age ≥ 18 years) of both genders with established diagnosis of chronic myeloproliferative neoplasms according to the revised WHO criteria;
- Patients must have an Eastern Cooperative Oncology Group performance status < 3 at baseline;
- Acceptable organ function within 7 days of initiating study drug;
Key exclusion criteria (19 listed)
- Pregnancy or nursing (lactating) women, where pregnancy is defined as the state of a female after conception, confirmed by a positive human Chorionic Gonadotropin (hCG) laboratory test (i.e. > 5 mIU/mL) and until the termination of gestation;
- A clinically significant corrected QT interval prolongation at baseline;
- Use of concomitant medications known to prolong the corrected QT interval;
- Clinically significant cardiovascular disease including:
- Active virus infection including human HIV, HBV and HCV;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| givinostatAlso: givinostat (ITF2357)Patients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. Givinostat is a histone-deacetylases inhibitor. The product will be supplied as hard gelatine capsules for oral administration at the strength of 50 mg, 75 mg and/or 100 mg each. If patients previously received givinostat in combination with other drugs during a core protocol or a compassionate use program, they will be treated at their last tolerable dose of this combination. | Drug | givinostat |
Primary outcome
Long-term safety and efficacy
Time frame: 3 months
To obtain information on the long-term safety and tolerability of givinostat in patients with chronic myeloproliferative neoplasms following core protocols or compassionate use program: * Number of patients experiencing adverse events; * Type, incidence, and severity of treatment-related adverse events. To determine the long term efficacy of givinostat in patients with chronic myeloproliferative neoplasms following core protocols or compassionate use program: * For Polycythemia Vera and Essential Thrombocythemia, Complete response and partial response rate according to the revised clinico-haematological European LeukemiaNet response criteria; * For Myelofibrosis, complete response, major response, moderate response and minor response rate according to European Myelofibrosis Network 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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alessandro Rambaldi, MD principal investigator
Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy
15 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing givinostat in people with Chronic Myeloproliferative Neoplasms, run by Italfarmaco at 15 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Italy, Germany, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long-term safety and efficacy (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2025.