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CompletedNCT02823184PhiNESSUpdated Jun 19, 2020

Endoplasmic Reticulum Stress and Resistance to Treatments in Ph-negative Myeloproliferative Neoplasms

An observational study in Polycythemia Vera and Essential Thrombocythemia, sponsored by University Hospital, Bordeaux. Completed at 7 sites in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-06-19.

Sponsored by University Hospital, Bordeaux · Observational

Study type
Observational
Model
Cohort
Time perspective
Retrospective
Enrollment
148
Ages
18 Years and older
Sex
All
01

Study 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 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.

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Conditions studied

  • Polycythemia Vera
  • Essential Thrombocythemia

Keywords

  • Myeloproliferative neoplasms
  • Endoplasmic reticulum stress
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In context

Neoplasms

9,365 studies on the registry are indexed under Neoplasms; 2,489 are open to participants now.

This study's enrollment of 148 is below the median of 204 across 1,683 observational studies indexed under Neoplasms.

Browse Neoplasms studies →

Lead sponsor

University Hospital, Bordeaux is the lead sponsor of 783 studies on the registry; 188 are open to participants now.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Probability sample

Study population

Patients with Polycythemia Vera or Essential Thrombocythemia

Inclusion criteria

  • 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 :

    • 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.

Exclusion criteria

Exclusion Criteria:

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 :

      • 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
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Study design

Observational model
Cohort
Time perspective
Retrospective
Enrollment
148 participants (actual)
Patient registry
No

Groups and cohorts

  • 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

    Biological: RNA sample of total leukocytes before start of treatment

Interventions

  • BiologicalRNA sample of total leukocytes before start of treatment

    RNA sample of total leukocytes before start of treatment

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What researchers measure

Primary outcomes

  1. 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

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Study locations

7 sites
  • Chu Angers
    Angers, France
  • Ch de La Cöte Basque
    Bayonne, France
  • Chu de Bordeaux
    Bordeaux, France
  • Crlcc Bergonie
    Bordeaux, France
  • Chu de Brest
    Brest, France
  • Ch de Dax
    DAX, France
  • Ch de Libourne
    Libourne, France
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References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 19, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT02823184
Lead sponsor
University Hospital, Bordeaux
Responsible party
Sponsor
First posted
Jul 6, 2016
Start date
Apr 27, 2017
Primary completion
Apr 27, 2019
Completion
Apr 27, 2019
Last update
Jun 19, 2020

Study contacts

Olivier MANSIER, Doctor
principal investigator · University Hospital, Bordeaux

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jun 2020. You cannot join it, but the record below documents what was studied.

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