Mesenchymal Stem Cells in NPM1 Mutated Low Risk Acute Myeloid Leukemia: a Study of the Tumor Microenvironment and Its Contribution to the Outcome

NCT07379528 | Completed

• 1 other identifier: AML-MSC
• Study Type: observational
• Target: 70
• Locations: 1 country
• Sites: 1

Brief Summary

Acute Myeloid Leukemia (AML) arises from the somatic acquisition of genetic alterations in hematopoietic progenitor or stem cells. One of the main hallmarks of AML is heterogeneity in terms of morphology, immunophenotype, cytogenetics, and molecular abnormalities, this heterogeneity leads to an important clinical heterogeneity in term of response to chemotherapy and prognosis. , The European Leukemia Net recognizes three different prognostic risk group (favorable, intermediate and high). Patients with favorable or intermediate risk AML, theoretically, should be cured with pharmacological treatment only (chemo and in some cases targeted therapies). However, more of the 50% of patients with favorable or intermediate risk AML experience relapse. This heterogeneity in outcome is not only explained by genetics and it's probably due to the persistence of chemo-resistant leukemic stem cell (LSC) clone, and to its interaction with the bone marrow (BM) microenvironment. This research project is focused on the analysis of the mesenchymal stem cells (MSCs) of the BM in order to deepen their connections with the LSC and their correlation with different genetic AML subgroups, and to evaluate their contribution to the outcome of favorable risk AML with Nucleophosmin 1 (NPM1) gene mutation.

Conditions

Acute Myeloid Leukaemia (AML)

Keywords

Acute myeloid leukemia; Mesenchimal cells; NPM1

Outcome Measures

Primary Outcomes (1)

• To characterize the BM-MSCs of subjects affected by AML NPM1mut as possible novel indicators of patient clinical outcome

  detection of CD146+ MSCs percentage at baseline

  Baseline

Secondary Outcomes (5)

• Characterize AML-MSCs in patients with AML NPM1mut vs AML NPM1wt

  Baseline

• Characterize AML-MSCs in patients with AML NPM1mut vs AML NPM1wt

  baseline

• Characterize AML-MSCs in patients with AML NPM1mut vs AML NPM1wt

  baseline

• Characterize AML-MSCs in patients with AML NPM1mut vs AML NPM1wt

  baseline

• Characterize AML-MSCs in patients with AML NPM1mut vs AML NPM1wt

  baseline

Study Arms (1)

pazients with diagnosis of acute myeloid leukemia

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Adult patients with new onset Acute Myeloid Leukemia diagnosed at Clinica Ematologica of the Fondazione IRCCS Policlinico San Matteo di Pavia.

You may qualify if:

• Adult patients with new onset Acute Myeloid Leukemia diagnosed.

Sponsors & Collaborators

• Fondazione IRCCS Policlinico San Matteo di Pavia: lead

Study Sites (1)

Fondazione IRCCS Policlinico San Matteo

Pavia, Pavia, 27100, Italy

Location

Biospecimen

Retention: SAMPLES WITH DNA

Bone Marrow

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 27, 2025
• First Posted: January 30, 2026
• Study Start: March 15, 2023
• Primary Completion: February 1, 2025
• Study Completion: March 15, 2025
• Last Updated: January 30, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: The individual participant data will be made available after the publication of the study results in a peer-reviewed journal.
• Access Criteria: The data will be shared exclusively with researchers for scientific research purposes

Locations

IT (1)