CompletedNot Applicable

JAK2V617F Mutated Endothelium Has a Stem Cell Potential in Primary Myelofibrosis: a New Disease Ontogenesis for Chronic Myeloproliferative Disorders

Italy30 participantsStarted 2023-05-02

Plain-language summary

The present project aims to experimentally test the hypothesis that JAK2V617F mutated endothelium in primary myelofibrosis (PMF) we discovered in 2013 represents an embryonic-like hemogenic endothelium and is part of the malignant stem cell pool that give rise to clonal hematopoiesis. The hypothesis stems from the recent evidence that JAK2 mutation in PMF patients is acquired in utero or childhood. The experimental plan of the project is 1) to separate mature endothelial cells from peripheral blood of PMF patients for morphological, molecular, functional characterization and clonogenic assays; 2) to assay the megakaryocyte, erythroid, granulocyte and endothelial clonogenic potential of the mature endothelial cells of patients with PMF. The potential to form colonies will be the documentation that a fraction of mature endothelial cells of patients with PMF have the characteristics of embryonic hemogenic endothelium and might act as the cell of origin of PMF.

Who can participate

Age range

18 Years

Sex

ALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * availability to understand and sign the informed consent Exclusion Criteria: * no known concurrent hematologic disease or condition that can affect the interpretation of the results

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

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Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Isolation of endothelial cells

Timeframe: from month 1 to month 18

Genotyping of sorted endothelial cells

Timeframe: from month 6 to month 30 of the project

in vito culture of endothelail cells

Timeframe: from month 6 to month 36 of the project

Trial details

NCT IDNCT07607392

SponsorFondazione IRCCS Policlinico San Matteo di Pavia

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2026-02-28

View on ClinicalTrials.gov More Primary Myelofibrosids trials