Polycythemia VeraPhiNESSNCT02823184
Endoplasmic Reticulum Stress and Resistance to Treatments in Ph-negative Myeloproliferative Neoplasms
An observational study of Polycythemia Vera and Essential Thrombocythemia, run by University Hospital, Bordeaux at 7 sites in France.
Trial summary
The aim of this study is to evaluate the endoplasmic reticulum stress markers as predictive for response to hydroxyurea in polycythemia vera (PV) and essential thrombocythemia (ET). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycythemia Vera, Essential Thrombocythemia
- What is being tested?
- RNA sample of total leukocytes before start of treatment
- How many people
- 148 participants
- Where
- 7 sitesFrance
- Study dates
- 27 Apr 2017to 27 Apr 2019
- Phase
- Observational
- 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
Study population: Patients with Polycythemia Vera or Essential Thrombocythemia
Who may be able to join
- ET or PV patients diagnosed before acceleration phase and treated by hydroxyurea with a follow up period of at least 6 months following treatment start.
- Diagnosis criteria of PV :
- WHO criteria of PV with :
- Acquired JAK2V617F mutation > 5%
- Absence of evident cause of secondary polycythemia
- Diagnosis criteria of ET :
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- Platelet count > 450 G/L
- Absence of PV or Chronic Myeloid Leukemia
- Bone marrow biopsy preferred but not necessary in absence of reactional causes (CRP and ferritin levels normal) and/or presence of acquired JAK2V617F, CALR exon 9 or MPL exon 10
- Availability of RNA sample of total leukocytes before start of treatment.
Who cannot join
- In absence of clonality marker, presence of secondary cause of :
- Thrombocytosis :
- Inflammatory syndrom (CRP or SV increased)
- Iron deficiency (decreased ferritin level or increased soluble transferrin receptor level)
- Polycythemia :
- Increased or normal level of EPO in context of :
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- Hypoxia, respiratory insufficiency
- Sleep apnea syndrome
- Hyperaffin hemoglobin
- Absence of treatment by hydroxyurea
- Treatment by anagrelide, P32, pipobroman, interferon without subsequent hydroxyurea treatment.
- Concommitant treatment by other cancer chemotherapy (in a context of solid cancer for example).
- Diagnostic during transformation to acute leukemia
- Treatment by hydroxyurea during less than 6 months
- Bad observance of the cytotoxic treatment
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
France7 sites
Chu Angers
Angers
Ch de La Cöte Basque
Bayonne
Chu de Bordeaux
Bordeaux
Crlcc Bergonie
Bordeaux
Chu de Brest
Brest
Ch de Dax
Dax
Ch de Libourne
Libourne
About this study
The aim of this study is to evaluate the endoplasmic reticulum stress markers as predictive for response to hydroxyurea in polycythemia vera (PV) and essential thrombocythemia (ET).
Read the detailed description
The recent discovery of calreticulin mutations in myeloproliferative neoplasms point to the unexpected role of the endoplasmic reticulum biology in the pathophysiology in these diseases. Otherwise, the association of endoplasmic reticulum stress with solid cancers, in particular in resistance to chemotherapy, is well documented, contrary to hematological malignancies. The study aims to evaluate endoplasmic reticulum stress markers as predictors for the response to hydroxyurea in polycythemia vera and essential thrombocythemia patients. The main objective is to correlate endoplasmic reticulum stress (defined as splicing of XBP1 above 30%) to the rate of complete response after 6 months according to the 2009 ELN criteria. This is an observational retrospective study.
- Conditions
- Polycythemia Vera, Essential Thrombocythemia
- Being tested
- RNA sample of total leukocytes before start of treatment
- Keywords
- Myeloproliferative neoplasms, Endoplasmic reticulum stress
- Medical subject headings
- Polycythemia Vera, Thrombocythemia, Essential, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RNA sample of total leukocytes before start of treatmentBiological
RNA sample of total leukocytes before start of treatment
| Study group | Role | What they receive |
|---|---|---|
| Patients | ET or PV patients diagnosed before acceleration phase and treated by hydroxyurea with a follow up period of at least 6 months following treatment start, with a RNA sample of total leukocytes before start of treatment available Receives: Biological: RNA sample of total leukocytes before start of treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
To correlate endoplasmic reticulum stress (defined as splicing of XBP1 above 30%) to the rate of complete response after 6 months according to the 2009 ELN criteria
Measured: After 6 months of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2016 (estimated)
- Study start27 Apr 2017
- Primary completion27 Apr 2019
- Study completion27 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycythemia VeraStudies of the same condition, open and under way.
- More trials run by University Hospital, BordeauxThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 148 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bordeaux
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Olivier MANSIER, Doctor, Principal investigator, University Hospital, Bordeaux
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02823184
- Sponsor's protocol ID
- CHUBX 2014/27
- Acronym
- PhiNESS
- Status last verified
- Jun 2020
- First submitted
- 24 Jun 2016
- Last updated
- 19 Jun 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Endoplasmic Reticulum Stress and Resistance to Treatments in Ph-negative Myeloproliferative Neoplasms
- ClinicalTrials.gov ID
- NCT02823184
- Acronym / sponsor ID
- PhiNESS / CHUBX 2014/27
- Conditions
- Polycythemia Vera, Essential Thrombocythemia
- Keywords
- Myeloproliferative neoplasms, Endoplasmic reticulum stress
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 148 participants (actual)
- Lead sponsor
- University Hospital, BordeauxUniversity, hospital or other
- Study start
- 27 Apr 2017 (actual)
- Primary completion
- 27 Apr 2019 (actual)
- Study completion
- 27 Apr 2019 (actual)
- First posted
- 6 Jul 2016 (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.
The aim of this study is to evaluate the endoplasmic reticulum stress markers as predictive for response to hydroxyurea in polycythemia vera (PV) and essential thrombocythemia (ET).
Detailed description
The recent discovery of calreticulin mutations in myeloproliferative neoplasms point to the unexpected role of the endoplasmic reticulum biology in the pathophysiology in these diseases. Otherwise, the association of endoplasmic reticulum stress with solid cancers, in particular in resistance to chemotherapy, is well documented, contrary to hematological malignancies. The study aims to evaluate endoplasmic reticulum stress markers as predictors for the response to hydroxyurea in polycythemia vera and essential thrombocythemia patients. The main objective is to correlate endoplasmic reticulum stress (defined as splicing of XBP1 above 30%) to the rate of complete response after 6 months according to the 2009 ELN criteria. This is an observational retrospective study.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients | – | Biological: RNA sample of total leukocytes before start of treatmentET or PV patients diagnosed before acceleration phase and treated by hydroxyurea with a follow up period of at least 6 months following treatment start, with a RNA sample of total leukocytes before start of treatment available |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with Polycythemia Vera or Essential Thrombocythemia
Key inclusion criteria (10 listed)
- ET or PV patients diagnosed before acceleration phase and treated by hydroxyurea with a follow up period of at least 6 months following treatment start.
- Diagnosis criteria of PV :
- Diagnosis criteria of ET :
- Availability of RNA sample of total leukocytes before start of treatment.
Key exclusion criteria (15 listed)
- Thrombocytosis :
- Polycythemia :
- Absence of treatment by hydroxyurea
- Treatment by anagrelide, P32, pipobroman, interferon without subsequent hydroxyurea treatment.
- Concommitant treatment by other cancer chemotherapy (in a context of solid cancer for example).
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 |
|---|---|---|
| RNA sample of total leukocytes before start of treatmentRNA sample of total leukocytes before start of treatment | Biological | Patients |
Primary outcome
To correlate endoplasmic reticulum stress (defined as splicing of XBP1 above 30%) to the rate of complete response after 6 months according to the 2009 ELN criteria
Time frame: After 6 months of treatment
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 19 Jun 2020.
- Individual participant dataSharing planned: No
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RecruitingPhase 2
A Study of Ropeg-Interferon in People With Polycythemia Vera
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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.
Olivier MANSIER, Doctor principal investigator
University Hospital, Bordeaux
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Polycythemia Vera and Essential Thrombocythemia, run by University Hospital, Bordeaux at 7 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 10 inclusion and 15 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 19 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 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.
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?
To correlate endoplasmic reticulum stress (defined as splicing of XBP1 above 30%) to the rate of complete response after 6 months according to the 2009 ELN criteria (time frame: After 6 months of treatment). 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 19 Jun 2020.