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

Myeloproliferative Neoplasm Clinical Trial

— BioPredictor  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05440838
• Other study ID #: 2022-A01044-39
• Status: Not yet recruiting
• Start date: September 8, 2022
• Est. completion date: October 4, 2029

Study information

• Verified date: June 2022
• Source: University Hospital, Angers
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 120
• Est. completion date: October 4, 2029
• Est. primary completion date: September 7, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Myeloproliferative Disorders
• Myeloproliferative Neoplasm
• Polycythemia
• Polycythemia Vera
• Primary Myelofibrosis
• Thrombocythemia, Essential
• Thrombocytosis

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (6)

Lead Sponsor	Collaborator
University Hospital, Angers	Nantes University Hospital, Poitiers University Hospital, Rennes University Hospital, University Hospital, Brest, University Hospital, Tours

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Complete hematological response	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	Complete hematological response	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
Secondary	Molecular response	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