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

Sponsor
University Hospital, Angers (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT05440838
Collaborator
University Hospital, Brest (Other), Nantes University Hospital (Other), Poitiers University Hospital (Other), Rennes University Hospital (Other), University Hospital, Tours (Other)
120
84.9

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase
  • Diagnostic Test: Next-generation sequencing

Study Design

Study Type:
Observational
Anticipated Enrollment :
120 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.
Anticipated Study Start Date :
Sep 8, 2022
Anticipated Primary Completion Date :
Sep 7, 2024
Anticipated Study Completion Date :
Oct 4, 2029

Outcome Measures

Primary Outcome Measures

  1. Complete hematological response [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 Outcome Measures

  1. Complete hematological response [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 [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.

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

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

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
University Hospital, Angers
ClinicalTrials.gov Identifier:
NCT05440838
Other Study ID Numbers:
  • 2022-A01044-39
First Posted:
Jul 1, 2022
Last Update Posted:
Jul 1, 2022
Last Verified:
Jun 1, 2022
Individual Participant Data (IPD) Sharing Statement:
Undecided
Plan to Share IPD:
Undecided
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Jul 1, 2022