Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis. (BioPredictor)

March 4, 2026 updated by: University Hospital, Angers

First-line treatment for patients with polycythemia vera, essential thrombocythemia, and pre-myelofibrosis is based on hydroxyurea or pegylated interferon. The objective of treatment is to prevent thrombotic complications and leukemic transformation. Despite overall good response rates, some patients do not respond to treatment and others lose their response over time. Both situations are associated with worse survival and there are to date no clear predictive factors for response although the existence of additional mutations seems unfavorable.

In this exploratory study, we hypothesize that biological factors at diagnosis are associated with hematological response at 12 months. We will more specifically study the association between mutational profile, assessed by next-generation sequencing, and cytokine profile with hematological response.

This study will help in identifying patients who will not respond to hydroxyurea or pegylated interferon and give the opportunity to try other treatments upfront, in the perspective of precision medicine. On the basic science side, this study will help in understanding the molecular and immunological factors involved in resistance to treatment.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

120

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis requiring first-line treatment with hydroxyurea or pegylated interferon.

Description

Inclusion Criteria:

  • Adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis.
  • Indication for first-line treatment with hydroxyurea or pegylated interferon.
  • Consent to participate.
  • Affiliated to social security.

Exclusion Criteria:

  • Previous treatment.
  • Other on-going malignancy, including overt myelofibrosis.
  • Other treatment such as phlebotomy solely, ruxolitinib, anagrelide, or pipobroman.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete hematological response
Time Frame: 12 months

ELN-2013 criteria by meeting all of the following:

  • Durable resolution of disease-related signs including palpable hepatosplenomegaly, large symptoms improvement, AND
  • Durable peripheral blood count remission, defined as: platelet count ≤400 ×109/L, WBC count <10 × 109/L, Ht lower than 45% without phlebotomies (for PV patients), absence of leukoerythroblastosis, AND
  • Without signs of progressive disease, and absence of any hemorrhagic or thrombotic events.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete hematological response
Time Frame: 24, 36, 48, and 60 months

ELN-2013 criteria by meeting all of the following:

  • Durable resolution of disease-related signs including palpable hepatosplenomegaly, large symptoms improvement, AND
  • Durable peripheral blood count remission, defined as: platelet count ≤400 ×109/L, WBC count <10 × 109/L, Ht lower than 45% without phlebotomies (for PV patients), absence of leukoerythroblastosis, AND
  • Without signs of progressive disease, and absence of any hemorrhagic or thrombotic events.
24, 36, 48, and 60 months
Molecular response
Time Frame: 12 and 24 months
ELN-2013 criteria: Complete response is defined as eradication of a preexisting abnormality (CALR, JAK2, or MPL mutations) by quantitative PCR. Partial response applies only to patients with at least 20% mutant allele burden at baseline. Partial response is defined as ≥50% decrease in allele burden by quantitative PCR.
12 and 24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Angers

Collaborators

Poitiers University Hospital
University Hospital, Brest
Rennes University Hospital
Nantes University Hospital
University Hospital, Tours

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 17, 2023

Primary Completion (Estimated)

February 17, 2027

Study Completion (Estimated)

February 17, 2032

Study Registration Dates

First Submitted

June 10, 2022

First Submitted That Met QC Criteria

June 27, 2022

First Posted (Actual)

July 1, 2022

Study Record Updates

Last Update Posted (Actual)

March 6, 2026

Last Update Submitted That Met QC Criteria

March 4, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2022-A01044-39

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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