Polycythemia VeraPhase 2NCT00241241
Efficacy and Safety of Pegylated Interferon Alfa in Polycythemia Vera
A Phase 2 study testing pegylated interferon-alfa 2a in people with Polycythemia Vera, run by PV-Nord at 5 sites in France.
Trial summary
Interferon alfa is an effective treatment of polycythemia vera (PV), but about 20% of patients discontinue their treatment because of side effects and treatment schedule (three times per week administration). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Polycythemia Vera
- What is being tested?
- pegylated interferon-alfa 2a
- How many people
- 40 participants
- Where
- 5 sitesFrance
- Study dates
- Sep 2004to Jan 2008
- Phase
- Phase 2
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- polycythemia vera diagnosed according to PVSG criteria, modified by Pearson
- Previously untreated patients or patients treated by phlebotomy only or HU or pipobroman for less than 2 years
- Age 18 to 65 years
- Signed informed consent
Who cannot join
- Contra indication for interferon
- Severe renal or liver disease
- ECOG performance status > 2
- Pregnancy
- Uncontrolled endocrine disorders except well regulated hyperthyroidism and diabetes
- Severe concomitant heart failure or psychiatric disorder
- Patients receiving an other investigational treatment
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
France5 sites
Hopital Avicenne
Bobigny
Hopital Huriez
Lille
Hopital Dupuytren
Limoges
Hopital Lariboisiere
Paris
Hopital Saint-Louis
Paris
About this study
Official title: Multicenter Phase 2 Study of Efficacy and Safety of Pegylated Interferon-alfa 2a in Polycythemia Vera Patients
Interferon alfa is an effective treatment of polycythemia vera (PV), but about 20% of patients discontinue their treatment because of side effects and treatment schedule (three times per week administration). The pegylated form of interferon alfa-2a has shown a better tolerance in hepatitis patients and is administered only once a week. The purpose of this study is to determine efficacy and safety of pegylated interferon alfa-2a in the treatment of PV patients.
Read the detailed description
The aim of PV treatment is to reduce the risk of vascular thrombosis without enhancing the long-term risk of evolution toward myelofibrosis or MDS/AL. Although currently controversial, phlebotomies have been shown in the old PVSG01 study to increase the risk of both thrombosis and myelofibrosis. On the other hand, currently available cytoreductive treatments have been shown to efficiently reduce the thrombotic risk, but were demonstrated (32P, busulfan, chlorambucil) or suspected (pipobroman, hydroxyurea) to enhance the risk of evolution to MDS/AL. In fact, the main widely used cytoreductive treatment, when indicated, is hydroxyurea (HU). This drug is very efficient to control myeloproliferation with a response rate of 80 to 90%. It is generally well tolerated, even if long term toxicity leads to treatment change in 10% of cases. Although no prospective study has yet clearly demonstrated its leukemogenic potential in PV, a non-leukemogenic alternative treatment is highly warranted, especially for younger patient.
Interferon (IFN) alpha is a promising agent in PV both because of good efficacy and absence of leukemogenic risk. Expanded experience with IFN-alpha was recently reported, showing a control of erythrocytosis in approximately 75% of patients. A similar percentage of patients also have resolution of disease-related symptoms, in particular a reduction in spleen size and relief from intractable pruritus. In some cases, long-term persisting remissions after treatment discontinuation have been observed as well as demonstration of eradication of the myeloproliferative clone. However, 20% of patients may not tolerate the treatment because of side effects. Furthermore, the treatment schedule (three times per week administration) may be a factor reducing long-term compliance to this drug.
In this regard, pegylated-IFN could be a major drug in PV. The weekly administration and better tolerance by comparison to IFN reported in hepatitis patients could allow to obtain results similar to chemotherapy in terms of compliance to treatment and efficacy, with a major advantage, its lack of mutagenicity.
- Conditions
- Polycythemia Vera
- Being tested
- pegylated interferon-alfa 2a
- Keywords
- myeloproliferative disorder, polycythemia vera, interferon
- Medical subject headings
- Polycythemia Vera, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pegylated interferon-alfa 2aDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
response rate after one year of treatment
Secondary outcomes (2)
safety
molecular response
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2005 (estimated)
- Study startSep 2004
- Primary completionOct 2006
- Study completionJan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants
Sponsor and investigators
- Lead sponsor
- PV-Nord
- Sponsor type
- University, hospital or other
- Investigators
- Jean-Jacques Kiladjian, MD, Principal investigator, PV-NordPierre Fenaux, MD, PhD, Study chair, PV-NordChristine Chomienne, MD, PhD, Study chair, PV-NordSylvia Bellucci, MD, Study chair, PV-NordBruno Cassinat, MD, Study chair, PV-NordMarie-Jose Grange, MD, Study chair, PV-NordNathalie Cambier, MD, Study chair, PV-NordJean-Francois Bernard, MD, Study chair, PV-Nord
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00241241
- Sponsor's protocol ID
- PVN1
- Status last verified
- Oct 2015
- First submitted
- 17 Oct 2005
- Last updated
- 21 Oct 2015
- Results posted
- No
Publications
Berk PD, Goldberg JD, Donovan PB, Fruchtman SM, Berlin NI, Wasserman LR. Therapeutic recommendations in polycythemia vera based on Polycythemia Vera Study Group protocols. Semin Hematol. 1986 Apr;23(2):132-43.
PubMed 3704665Fruchtman SM, Mack K, Kaplan ME, Peterson P, Berk PD, Wasserman LR. From efficacy to safety: a Polycythemia Vera Study group report on hydroxyurea in patients with polycythemia vera. Semin Hematol. 1997 Jan;34(1):17-23. No abstract available.
PubMed 9025158Kiladjian JJ, Bernard JF, Fenaux P. Is life expectancy of polycythemia vera patients clearly different from that of the general population? Am J Med. 2005 May;118(5):565; author reply 565-6. doi: 10.1016/j.amjmed.2004.12.026. No abstract available.
PubMed 15866263Lengfelder E, Berger U, Hehlmann R. Interferon alpha in the treatment of polycythemia vera. Ann Hematol. 2000 Mar;79(3):103-9. doi: 10.1007/s002770050563.
PubMed 10803930Streiff MB, Smith B, Spivak JL. The diagnosis and management of polycythemia vera in the era since the Polycythemia Vera Study Group: a survey of American Society of Hematology members' practice patterns. Blood. 2002 Feb 15;99(4):1144-9. doi: 10.1182/blood.v99.4.1144.
PubMed 11830459Kiladjian JJ, Cassinat B, Chevret S, Turlure P, Cambier N, Roussel M, Bellucci S, Grandchamp B, Chomienne C, Fenaux P. Pegylated interferon-alfa-2a induces complete hematologic and molecular responses with low toxicity in polycythemia vera. Blood. 2008 Oct 15;112(8):3065-72. doi: 10.1182/blood-2008-03-143537. Epub 2008 Jul 23.
PubMed 18650451Kiladjian JJ, Cassinat B, Turlure P, Cambier N, Roussel M, Bellucci S, Menot ML, Massonnet G, Dutel JL, Ghomari K, Rousselot P, Grange MJ, Chait Y, Vainchenker W, Parquet N, Abdelkader-Aljassem L, Bernard JF, Rain JD, Chevret S, Chomienne C, Fenaux P. High molecular response rate of polycythemia vera patients treated with pegylated interferon alpha-2a. Blood. 2006 Sep 15;108(6):2037-40. doi: 10.1182/blood-2006-03-009860. Epub 2006 May 18.
PubMed 16709929
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Phase 2 Study of Efficacy and Safety of Pegylated Interferon-alfa 2a in Polycythemia Vera Patients
- ClinicalTrials.gov ID
- NCT00241241
- Acronym / sponsor ID
- PVN1
- Conditions
- Polycythemia Vera
- Keywords
- myeloproliferative disorder, polycythemia vera, interferon
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants
- Lead sponsor
- PV-NordUniversity, hospital or other
- Study start
- Sep 2004
- Primary completion
- Oct 2006 (actual)
- Study completion
- Jan 2008 (actual)
- First posted
- 18 Oct 2005 (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.
Interferon alfa is an effective treatment of polycythemia vera (PV), but about 20% of patients discontinue their treatment because of side effects and treatment schedule (three times per week administration). The pegylated form of interferon alfa-2a has shown a better tolerance in hepatitis patients and is administered only once a week. The purpose of this study is to determine efficacy and safety of pegylated interferon alfa-2a in the treatment of PV patients.
Detailed description
The aim of PV treatment is to reduce the risk of vascular thrombosis without enhancing the long-term risk of evolution toward myelofibrosis or MDS/AL. Although currently controversial, phlebotomies have been shown in the old PVSG01 study to increase the risk of both thrombosis and myelofibrosis. On the other hand, currently available cytoreductive treatments have been shown to efficiently reduce the thrombotic risk, but were demonstrated (32P, busulfan, chlorambucil) or suspected (pipobroman, hydroxyurea) to enhance the risk of evolution to MDS/AL. In fact, the main widely used cytoreductive treatment, when indicated, is hydroxyurea (HU). This drug is very efficient to control myeloproliferation with a response rate of 80 to 90%. It is generally well tolerated, even if long term toxicity leads to treatment change in 10% of cases. Although no prospective study has yet clearly demonstrated its leukemogenic potential in PV, a non-leukemogenic alternative treatment is highly warranted, especially for younger patient.
Interferon (IFN) alpha is a promising agent in PV both because of good efficacy and absence of leukemogenic risk. Expanded experience with IFN-alpha was recently reported, showing a control of erythrocytosis in approximately 75% of patients. A similar percentage of patients also have resolution of disease-related symptoms, in particular a reduction in spleen size and relief from intractable pruritus. In some cases, long-term persisting remissions after treatment discontinuation have been observed as well as demonstration of eradication of the myeloproliferative clone. However, 20% of patients may not tolerate the treatment because of side effects. Furthermore, the treatment schedule (three times per week administration) may be a factor reducing long-term compliance to this drug.
In this regard, pegylated-IFN could be a major drug in PV. The weekly administration and better tolerance by comparison to IFN reported in hepatitis patients could allow to obtain results similar to chemotherapy in terms of compliance to treatment and efficacy, with a major advantage, its lack of mutagenicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- polycythemia vera diagnosed according to PVSG criteria, modified by Pearson
- Previously untreated patients or patients treated by phlebotomy only or HU or pipobroman for less than 2 years
- Age 18 to 65 years
- Signed informed consent
Key exclusion criteria (7 listed)
- Contra indication for interferon
- Severe renal or liver disease
- ECOG performance status > 2
- Pregnancy
- Uncontrolled endocrine disorders except well regulated hyperthyroidism and diabetes
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 |
|---|---|---|
| pegylated interferon-alfa 2a | Drug | – |
Primary outcome
response rate after one year of treatment
2 secondary outcomes
safety
molecular response
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2015.
- Background: PubMed 3704665Berk PD, Goldberg JD, Donovan PB, Fruchtman SM, Berlin NI, Wasserman LR. Therapeutic recommendations in polycythemia vera based on Polycythemia Vera Study Group protocols. Semin Hematol. 1986 Apr;23(2):132-43.
- Background: PubMed 9025158Fruchtman SM, Mack K, Kaplan ME, Peterson P, Berk PD, Wasserman LR. From efficacy to safety: a Polycythemia Vera Study group report on hydroxyurea in patients with polycythemia vera. Semin Hematol. 1997 Jan;34(1):17-23. No abstract available.
- Background: PubMed 15866263Kiladjian JJ, Bernard JF, Fenaux P. Is life expectancy of polycythemia vera patients clearly different from that of the general population? Am J Med. 2005 May;118(5):565; author reply 565-6. doi: 10.1016/j.amjmed.2004.12.026. No abstract available.
- Background: PubMed 10803930Lengfelder E, Berger U, Hehlmann R. Interferon alpha in the treatment of polycythemia vera. Ann Hematol. 2000 Mar;79(3):103-9. doi: 10.1007/s002770050563.
- Background: PubMed 11830459Streiff MB, Smith B, Spivak JL. The diagnosis and management of polycythemia vera in the era since the Polycythemia Vera Study Group: a survey of American Society of Hematology members' practice patterns. Blood. 2002 Feb 15;99(4):1144-9. doi: 10.1182/blood.v99.4.1144.
- Derived from this study: PubMed 18650451Kiladjian JJ, Cassinat B, Chevret S, Turlure P, Cambier N, Roussel M, Bellucci S, Grandchamp B, Chomienne C, Fenaux P. Pegylated interferon-alfa-2a induces complete hematologic and molecular responses with low toxicity in polycythemia vera. Blood. 2008 Oct 15;112(8):3065-72. doi: 10.1182/blood-2008-03-143537. Epub 2008 Jul 23.
- Derived from this study: PubMed 16709929Kiladjian JJ, Cassinat B, Turlure P, Cambier N, Roussel M, Bellucci S, Menot ML, Massonnet G, Dutel JL, Ghomari K, Rousselot P, Grange MJ, Chait Y, Vainchenker W, Parquet N, Abdelkader-Aljassem L, Bernard JF, Rain JD, Chevret S, Chomienne C, Fenaux P. High molecular response rate of polycythemia vera patients treated with pegylated interferon alpha-2a. Blood. 2006 Sep 15;108(6):2037-40. doi: 10.1182/blood-2006-03-009860. Epub 2006 May 18.
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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.
Jean-Jacques Kiladjian, MD principal investigator
PV-Nord
Pierre Fenaux, MD, PhD study chair
PV-Nord
Christine Chomienne, MD, PhD study chair
PV-Nord
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing pegylated interferon-alfa 2a in people with Polycythemia Vera, run by PV-Nord at 5 sites in France.
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 4 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: finished, as of the sponsor's last update on 21 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in France. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
response rate after one year of treatment. 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 NCT00241241, which also lists 7 linked publications.
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 21 Oct 2015.