Polycythemia VeraPhase 3CYTO-PVNCT01645124
Large-scale Trial Testing the Intensity of CYTOreductive Therapy in Polycythemia Vera (PV)
A Phase 3 study testing Hydroxyurea and Phlebotomy in people with Polycythemia Vera, run by Consorzio Mario Negri Sud at 26 sites in Italy.
Trial summary
CYTO-PV is a phase III Prospective, Randomized, Open-label, with Blinded Endpoint evaluation (PROBE), multi-center, clinical trial in patients with diagnosis of Polycythemia vera (PV) treated at the best of recommended therapies (e.g.adequate control of standard cardiovascular risk factors). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycythemia Vera
- What is being tested?
- Hydroxyurea and Phlebotomy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 365 participants
- Where
- 26 sitesItaly
- Study dates
- May 2008to Jul 2012
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Males and females aged 18 years or more are eligible for the study if they meet all the following inclusion criteria:
- New diagnosis of PV according to WHO 2007 diagnostic criteria including Jak 2 V617F mutation status;
- Old diagnosis of PV confirmed with JAK-2 positivity and clinical course of the disease;
- Ability and willingness to comply with all study requirements;
- Written informed consent (obtained before any study specific procedure).
Who cannot join
- Pregnant or lactating women or women of childbearing potential who are not protected from pregnancy by an accepted method of contraception;
- Known hypersensitivity or contraindication to study treatments;
- Significant liver (AST or ALT > 2.5 times ULN) or renal disease (creatinine > 2 mg/ml);
- Presence of any life-threatening condition or of any disease (e.g. cancer) that is likely to significantly shorten life expectancy;
- History of active substance or alcohol abuse within the last year;
- Any condition that in the opinion of the investigator would jeopardize the evaluation of efficacy or safety or be associated with poor adherence to the protocol - Baseline and FUP visits schedule and assessments
- Logistic problem related to the patient.
Trial locations and contacts
26 sites in 1 country.
26 sites in 1 country
Italy26 sites
Azienda Ospedaliera Universitaria Ospedali Riuniti di Ancona
Ancona, Ancona, 60020
Azienda Ospedaliera Universitaria Ospedale Consorziale Policlinico di Bari
Bari, Bari, 70124
Azienda Ospedali Riuniti di Bergamo
Bergamo, Bergamo, 24128
Azienda Unità Sanitaria Locale di Brindisi BR/1- Ospedale "Di Summa - Perrino"
Brindisi, Brindisi, 72100
Ospedale Armando Businco
Cagliari, Cagliari, 09121
Azienda Ospedaliera Universitaria-'Policlinico- Vittorio Emanuele'-Ospedale Ferrarotto Alessi di Catania
Catania, Catania, 95124
Azienda Ospedaliera S. Croce e Carle di Cuneo
Cuneo, Cuneo, 12100
Azienda Ospedaliera Universitaria Careggi di Firenze
Florence, Firenze, 50134
Azienda Ospedaliera Universitaria Policlinico Martino di Messina
Messina, Messina, 98122
Fondazione IRCSS Cà Granda- Ospedale Maggiore Policlinico
Milan, Milano, 20122
Ospedale S.Raffaele
Milan, Milano, 20132
Ospedale S.Gerardo di Monza
Monza, Monza, 20900
Azienda Ospedaliera Universitaria'Maggiore della Carità' di Novara
Novara, Novara, 28100
Azienda Ospedaliero-Universitaria San Luigi Gonzaga di Orbassano
Orbassano, Torino, 10043
Università di Padova
Padova, Padova, 35128
Azienda Ospedaliero-Universitaria Policlinico Giaccone di Palermo
Palermo, Palermo, 90127
IRCCS Policlinico S. Matteo di Pavia
Pavia, Pavia, 27100
Azienda Ospedaliera S. Salvatore, Presidio San Salvatore Muraglia
Pesaro, Pesaro, 61100
AUSL 4 Prato, Ospedale "Misericordia e Dolce" di Prato
Prato, Prato, 59100
Ospedale di S.Maria Nuova
Reggio Emilia, Reggio Emilia, 42100
IRCCS Centro di Riferimento Oncologico di Basilicata (CROB)
Rionero in Vulture, Potenza, 85028
IRCCS Istituto Regina Elena (IFO)
Roma, Roma, 00144
Università degli Studi di Roma "La Sapienza"
Roma, Roma, 00161
Policlinico Universitario Gemelli di Roma
Roma, Roma, 00168
IRCCS Ospedale Casa Sollievo della Sofferenza di San Giovanni Rotondo
San Giovanni Rotondo, Foggia, 71013
Ospedale San Bortolo di Vicenza
Vicenza, Vicenza, 36100
About this study
Official title: A Large-scale Trial Testing the Intensity of CYTOreductive Therapy to Prevent Cardiovascular Events In Patients With Polycythemia Vera (PV)
CYTO-PV is a phase III Prospective, Randomized, Open-label, with Blinded Endpoint evaluation (PROBE), multi-center, clinical trial in patients with diagnosis of Polycythemia vera (PV) treated at the best of recommended therapies (e.g.adequate control of standard cardiovascular risk factors). Irrespective of randomized interventions, all patients will be administered low-dose aspirin (when not contraindicated), i.e.the standard antithrombotic treatment in PV patients.
The purpose of this study to demonstrate that a more intensive cytoreductive therapy, plus low-dose aspirin when not contraindicated, with phlebotomy and/or hydroxyurea (HU), aimed at maintaining hematocrit (HCT) < 45% is more effective than a less intensive cytoreduction (either with phlebotomy or HU plus low-dose aspirin when not contraindicated) maintaining HCT in the range of 45-50% in the reduction of CV deaths plus thrombotic events (stroke, acute coronary syndrome [ACS], transient ischemic attack [TIA], pulmonary embolism [PE], splanchnic thrombosis, deep vein thrombosis [DVT], and any other clinically relevant thrombotic event), in patients with Polycythemia Vera treated at the best of recommended therapies (e.g. adequate control of standard cardiovascular risk factors).
Read the detailed description
Polycythemia vera (PV) is a chronic myeloproliferative disorder characterized by clonal proliferation of hematopoietic progenitors resulting in expansion of the erythrocyte mass, and its clinical course is affected by cardiovascular events, the main cause of morbidity and mortality. Arterial thrombotic events are predominant, particularly large vessel arterial events including cerebrovascular accidents, myocardial infarction, and peripheral arterial occlusion. Based on the complex relationship between thrombosis, hematocrit, and parameters of tissue perfusion and blood viscosity, the latter has been proved to be an exponential function of the hematocrit. Red cell aggregation increases at high hematocrit (HCT) levels, creating the potential for vascular stasis. As a result, enhanced interplay between platelet, leukocytes and vessel wall increases the risk of thrombosis.
Considering the lack of effective therapeutic strategy targeted at the mutated allele JAK2V617F, there is no known treatment that eradicates the abnormal clone, apart from anecdotal cases of bone marrow transplantation. Cytoreductive treatment by phlebotomy or chemotherapy, however, has dramatically reduced the number of thrombotic complications and substantially improved survival and today there is agreement that the goal of cytoreductive treatment should be to keep the HCT value below 0.45 in all PV patients.
This was suggested on the basis of a small, retrospective study of PV that more than 30 years ago showed a progressive increase in the incidence of vascular occlusive episodes at HCT levels higher than 44% and in patients treated according to the drugs and the therapeutic tenets of the time. However no clinical trial has confirmed such findings. The results of the two largest prospective studies currently available (namely PVSG-1 and ECLAP) suggest no difference in the risk of thrombosis among patients kept at HCT below 50%.
An association between relevant outcome events (namely. thrombotic events, mortality, and haematological progression) and HCT in the evaluable range of 40-55% was found in the ECLAP population neither in the multivariate analysis at baseline nor in the time-dependent multivariate analysis. The ECLAP trial demonstrated the antithrombotic efficacy of low-dose aspirin in this setting and the use of this therapy in clinical practice is likely to decrease meaningfully, though not eliminate, the high risk of thrombosis of PV patients.
In conclusion, the high incidence of thrombotic events irrespective of low-dose aspirin administration as well as of haematological transformation in the long term which have been shown in PV patients study suggest the need to investigate in depth the benefit/risk profile of current therapeutic options for cytoreductive therapy. CYTO-PV is aimed at assessing the benefit risk profile of cytoreductive therapy with phlebotomy and/or HU aimed at maintaining HCT < 45% Vs. maintaining HCT in the range 45-50%. It is an independent, investigator-generated pragmatic trial with broad selection criteria to mimic clinical practice in order to strengthen the transferability of its results to the population of PV patients; it has been designed to be conducted, without need of special facilities, in the framework of the Italian Group of hematologic Adult diseases ("GIMEMA"). The optimization of therapeutic management of PV patients will allow to improve the prognosis of PV patients, the allocation of the resources the Italian National Health Service (IHS), and the knowledge about the benefit/risk profile of pharmacological cytoreduction in PV
- Conditions
- Polycythemia Vera
- Being tested
- Hydroxyurea, Phlebotomy
- Keywords
- Polycythemia, Hematocrit, Thrombosis
- Medical subject headings
- Polycythemia Vera, Polycythemia, Thrombosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
PhlebotomyProcedure or surgery
| Study group | Role | What they receive |
|---|---|---|
| Cytoreduction for HCT < 45% | Active comparator | Patients will be treated with phlebotomy and/or HU more intensively, with the goal to reach and maintain the target of hematocrit(HCT)below 45%. Phlebotomy should be performed initially by removing 250-500 ml of every other day or twice a week until the target HCT is obtained. Hydroxyurea (HU)should be administered initially at a dose of 0.5-1.0 g daily. Blood counts at regular intervals (monthly) will establish the frequency of future phlebotomies with the goal to maintain the target HCT. Supplemental iron therapy should not be given. Low-dose aspirin is the standard antithrombotic therapy in PV and will be administered to all patients with no contraindications to aspirin. Receives: Drug: Hydroxyurea; Procedure: Phlebotomy |
| Cytoreduction for HCT between 45 and 50% | Experimental | Patients will be treated with phlebotomy and/or HU less intensively, with the goal to reach and maintain the target of hematocrit(HCT)between 45% and 50%. Phlebotomy should be performed initially by removing 250-500 ml of every other day or twice a week until the target HCT is obtained. Hydroxyurea (HU)should be administered initially at a dose of 0.5-1.0 g daily. Blood counts at regular intervals (monthly) will establish the frequency of future phlebotomies with the goal to maintain the target HCT. Supplemental iron therapy should not be given. Low-dose aspirin is the standard antithrombotic therapy in PV and will be administered to all patients with no contraindications to aspirin. Receives: Drug: Hydroxyurea; Procedure: Phlebotomy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction of PEP (Primary End Point)defined as CV deaths plus thrombotic events
To demonstrate that in patients with PV treatment with aggressive cytoreductive therapy aimed at maintaining HCT < 45% is more effective than cytoreductive therapy aimed at maintaining HCT between 45 and 50% in the reduction CV deaths plus thrombotic events (PEP: stroke, acute coronary syndrome [ACS], transient ischemic attack [TIA], pulmonary embolism [PE], abdominal thrombosis, deep vein thrombosis [DVT], and peripheral arterial thrombosis). The minimum clinically relevant beneficial effect is set at a 30% reduction of risk of the PEP.
Measured: Expected average of 5 years
Secondary outcomes (1)
PEP plus minor thrombosis, hospitalization and malignancy
The events included in the PEP, arterial and venous thrombosis, major and minor thrombosis as well as hospitalization for any reason, hospitalization for CV reason, malignancy and PV-related malignancy (progression to myelofibrosis, myelodysplastic or leukemic transformation) will be analyzed separately to assess the full benefit/risk profile of experimental treatments.
Measured: Expected average of 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2012 (estimated)
- Study startMay 2008
- Primary completionJun 2012
- Study completionJul 2012 (estimated)
Why it stopped: Low accrual rate not allowing planned sample size leads to a futility condition
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Polycythemia VeraStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 365 participants (actual)
Sponsor and investigators
- Lead sponsor
- Consorzio Mario Negri Sud
- Sponsor type
- University, hospital or other
- Collaborators
- Agenzia Italiana del Farmaco, A.O. Ospedale Papa Giovanni XXIII
- Responsible party
- Sponsor
- Investigators
- Tiziano Barbui, MD, Study chair, A.O. Ospedale Papa Giovanni XXIII
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01645124
- Sponsor's protocol ID
- FARM6YNXAN
- Acronym
- CYTO-PV
- Status last verified
- May 2012
- First submitted
- 17 Jul 2012
- Last updated
- 20 Jul 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Marchioli R, Finazzi G, Specchia G, Cacciola R, Cavazzina R, Cilloni D, De Stefano V, Elli E, Iurlo A, Latagliata R, Lunghi F, Lunghi M, Marfisi RM, Musto P, Masciulli A, Musolino C, Cascavilla N, Quarta G, Randi ML, Rapezzi D, Ruggeri M, Rumi E, Scortechini AR, Santini S, Scarano M, Siragusa S, Spadea A, Tieghi A, Angelucci E, Visani G, Vannucchi AM, Barbui T; CYTO-PV Collaborative Group. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med. 2013 Jan 3;368(1):22-33. doi: 10.1056/NEJMoa1208500. Epub 2012 Dec 8.
PubMed 23216616
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Large-scale Trial Testing the Intensity of CYTOreductive Therapy to Prevent Cardiovascular Events In Patients With Polycythemia Vera (PV)
- ClinicalTrials.gov ID
- NCT01645124
- Acronym / sponsor ID
- CYTO-PV / FARM6YNXAN
- Conditions
- Polycythemia Vera
- Keywords
- Polycythemia, Hematocrit, Thrombosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low accrual rate not allowing planned sample size leads to a futility condition
- Enrolment
- 365 participants (actual)
- Lead sponsor
- Consorzio Mario Negri SudUniversity, hospital or other
- Collaborators
- Agenzia Italiana del Farmaco, A.O. Ospedale Papa Giovanni XXIII
- Study start
- May 2008
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jul 2012 (estimated)
- First posted
- 20 Jul 2012 (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.
CYTO-PV is a phase III Prospective, Randomized, Open-label, with Blinded Endpoint evaluation (PROBE), multi-center, clinical trial in patients with diagnosis of Polycythemia vera (PV) treated at the best of recommended therapies (e.g.adequate control of standard cardiovascular risk factors). Irrespective of randomized interventions, all patients will be administered low-dose aspirin (when not contraindicated), i.e.the standard antithrombotic treatment in PV patients.
The purpose of this study to demonstrate that a more intensive cytoreductive therapy, plus low-dose aspirin when not contraindicated, with phlebotomy and/or hydroxyurea (HU), aimed at maintaining hematocrit (HCT) < 45% is more effective than a less intensive cytoreduction (either with phlebotomy or HU plus low-dose aspirin when not contraindicated) maintaining HCT in the range of 45-50% in the reduction of CV deaths plus thrombotic events (stroke, acute coronary syndrome [ACS], transient ischemic attack [TIA], pulmonary embolism [PE], splanchnic thrombosis, deep vein thrombosis [DVT], and any other clinically relevant thrombotic event), in patients with Polycythemia Vera treated at the best of recommended therapies (e.g. adequate control of standard cardiovascular risk factors).
Detailed description
Polycythemia vera (PV) is a chronic myeloproliferative disorder characterized by clonal proliferation of hematopoietic progenitors resulting in expansion of the erythrocyte mass, and its clinical course is affected by cardiovascular events, the main cause of morbidity and mortality. Arterial thrombotic events are predominant, particularly large vessel arterial events including cerebrovascular accidents, myocardial infarction, and peripheral arterial occlusion. Based on the complex relationship between thrombosis, hematocrit, and parameters of tissue perfusion and blood viscosity, the latter has been proved to be an exponential function of the hematocrit. Red cell aggregation increases at high hematocrit (HCT) levels, creating the potential for vascular stasis. As a result, enhanced interplay between platelet, leukocytes and vessel wall increases the risk of thrombosis.
Considering the lack of effective therapeutic strategy targeted at the mutated allele JAK2V617F, there is no known treatment that eradicates the abnormal clone, apart from anecdotal cases of bone marrow transplantation. Cytoreductive treatment by phlebotomy or chemotherapy, however, has dramatically reduced the number of thrombotic complications and substantially improved survival and today there is agreement that the goal of cytoreductive treatment should be to keep the HCT value below 0.45 in all PV patients.
This was suggested on the basis of a small, retrospective study of PV that more than 30 years ago showed a progressive increase in the incidence of vascular occlusive episodes at HCT levels higher than 44% and in patients treated according to the drugs and the therapeutic tenets of the time. However no clinical trial has confirmed such findings. The results of the two largest prospective studies currently available (namely PVSG-1 and ECLAP) suggest no difference in the risk of thrombosis among patients kept at HCT below 50%.
An association between relevant outcome events (namely. thrombotic events, mortality, and haematological progression) and HCT in the evaluable range of 40-55% was found in the ECLAP population neither in the multivariate analysis at baseline nor in the time-dependent multivariate analysis. The ECLAP trial demonstrated the antithrombotic efficacy of low-dose aspirin in this setting and the use of this therapy in clinical practice is likely to decrease meaningfully, though not eliminate, the high risk of thrombosis of PV patients.
In conclusion, the high incidence of thrombotic events irrespective of low-dose aspirin administration as well as of haematological transformation in the long term which have been shown in PV patients study suggest the need to investigate in depth the benefit/risk profile of current therapeutic options for cytoreductive therapy. CYTO-PV is aimed at assessing the benefit risk profile of cytoreductive therapy with phlebotomy and/or HU aimed at maintaining HCT < 45% Vs. maintaining HCT in the range 45-50%. It is an independent, investigator-generated pragmatic trial with broad selection criteria to mimic clinical practice in order to strengthen the transferability of its results to the population of PV patients; it has been designed to be conducted, without need of special facilities, in the framework of the Italian Group of hematologic Adult diseases ("GIMEMA"). The optimization of therapeutic management of PV patients will allow to improve the prognosis of PV patients, the allocation of the resources the Italian National Health Service (IHS), and the knowledge about the benefit/risk profile of pharmacological cytoreduction in PV
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cytoreduction for HCT < 45% | Active comparator | Drug: Hydroxyurea, Procedure: PhlebotomyPatients will be treated with phlebotomy and/or HU more intensively, with the goal to reach and maintain the target of hematocrit(HCT)below 45%. Phlebotomy should be performed initially by removing 250-500 ml of every other day or twice a week until the target HCT is obtained. Hydroxyurea (HU)should be administered initially at a dose of 0.5-1.0 g daily. Blood counts at regular intervals (monthly) will establish the frequency of future phlebotomies with the goal to maintain the target HCT. Supplemental iron therapy should not be given. Low-dose aspirin is the standard antithrombotic therapy in PV and will be administered to all patients with no contraindications to aspirin. |
| Cytoreduction for HCT between 45 and 50% | Experimental | Drug: Hydroxyurea, Procedure: PhlebotomyPatients will be treated with phlebotomy and/or HU less intensively, with the goal to reach and maintain the target of hematocrit(HCT)between 45% and 50%. Phlebotomy should be performed initially by removing 250-500 ml of every other day or twice a week until the target HCT is obtained. Hydroxyurea (HU)should be administered initially at a dose of 0.5-1.0 g daily. Blood counts at regular intervals (monthly) will establish the frequency of future phlebotomies with the goal to maintain the target HCT. Supplemental iron therapy should not be given. Low-dose aspirin is the standard antithrombotic therapy in PV and will be administered to all patients with no contraindications to aspirin. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Males and females aged 18 years or more are eligible for the study if they meet all the following inclusion criteria:
- New diagnosis of PV according to WHO 2007 diagnostic criteria including Jak 2 V617F mutation status;
- Old diagnosis of PV confirmed with JAK-2 positivity and clinical course of the disease;
- Ability and willingness to comply with all study requirements;
- Written informed consent (obtained before any study specific procedure).
Key exclusion criteria (7 listed)
- Pregnant or lactating women or women of childbearing potential who are not protected from pregnancy by an accepted method of contraception;
- Known hypersensitivity or contraindication to study treatments;
- Significant liver (AST or ALT > 2.5 times ULN) or renal disease (creatinine > 2 mg/ml);
- Presence of any life-threatening condition or of any disease (e.g. cancer) that is likely to significantly shorten life expectancy;
- History of active substance or alcohol abuse within the last year;
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 |
|---|---|---|
| Hydroxyurea | Drug | Cytoreduction for HCT < 45%, Cytoreduction for HCT between 45 and 50% |
| Phlebotomy | Procedure or surgery | Cytoreduction for HCT < 45%, Cytoreduction for HCT between 45 and 50% |
Primary outcome
Reduction of PEP (Primary End Point)defined as CV deaths plus thrombotic events
Time frame: Expected average of 5 years
To demonstrate that in patients with PV treatment with aggressive cytoreductive therapy aimed at maintaining HCT < 45% is more effective than cytoreductive therapy aimed at maintaining HCT between 45 and 50% in the reduction CV deaths plus thrombotic events (PEP: stroke, acute coronary syndrome [ACS], transient ischemic attack [TIA], pulmonary embolism [PE], abdominal thrombosis, deep vein thrombosis [DVT], and peripheral arterial thrombosis). The minimum clinically relevant beneficial effect is set at a 30% reduction of risk of the PEP.
1 secondary outcome
PEP plus minor thrombosis, hospitalization and malignancy
Time frame: Expected average of 5 years
Results and safety
The study was stopped early (reason given: Low accrual rate not allowing planned sample size leads to a futility condition). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2012.
- Derived from this study: PubMed 23216616Marchioli R, Finazzi G, Specchia G, Cacciola R, Cavazzina R, Cilloni D, De Stefano V, Elli E, Iurlo A, Latagliata R, Lunghi F, Lunghi M, Marfisi RM, Musto P, Masciulli A, Musolino C, Cascavilla N, Quarta G, Randi ML, Rapezzi D, Ruggeri M, Rumi E, Scortechini AR, Santini S, Scarano M, Siragusa S, Spadea A, Tieghi A, Angelucci E, Visani G, Vannucchi AM, Barbui T; CYTO-PV Collaborative Group. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med. 2013 Jan 3;368(1):22-33. doi: 10.1056/NEJMoa1208500. Epub 2012 Dec 8.
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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.
Tiziano Barbui, MD study chair
A.O. Ospedale Papa Giovanni XXIII
26 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Hydroxyurea and Phlebotomy in people with Polycythemia Vera, run by Consorzio Mario Negri Sud at 26 sites in Italy.
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 5 inclusion and 7 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: stopped early, as of the sponsor's last update on 20 Jul 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in Italy. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Low accrual rate not allowing planned sample size leads to a futility condition). Results are not available in the source record.
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?
Reduction of PEP (Primary End Point)defined as CV deaths plus thrombotic events (time frame: Expected average of 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01645124, which also lists 1 linked publication.
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 20 Jul 2012.