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NCT Number: NCT05440838

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

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.

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Key information

Conditions

Myeloproliferative Neoplasm Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myeloproliferative Disorders

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Chu Angers, Angers, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Next-Generation Sequencing

Diagnostic Test

Next-generation sequencing and cytokine profile will be established in all patients before the start of treatment.

Other names: Cytokines

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

Study contacts

Contact information is provided by the study sponsor or research team.

CORENTIN ORVAIN, DOCTOR

CONTACT

[email protected]

+3302 41 35 44 75

Sponsors and collaborators

Lead sponsor

University Hospital, Angers

Other Gov

Collaborators

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

Registry information

Acronym: BioPredictor

Important dates

Study start
2023
Primary completion
2027
Study completion
2032
First posted
Jul 1, 2022
Registry last updated
Mar 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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