Molecular Mechanisms of Disease Relapse After Allogenic Stem Cell Transplantation in MDS Patients

Not Applicable

Recruiting

• Conditions: Myelodysplastic Syndromes
• Interventions: Other: bone marrow withdrawal

• Registration Number: NCT04587856
• Lead Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Brief Summary

This is a biological study for adult MDS patients who undergo HSCT procedure. Viable bone marrow samples will be collected and cryopreserved from MDS patients before transplantation and at clinical disease recurrence. CD34+ blast cells at disease relapse after HSCT will be compared with CD34+ cells collected before transplant to study genomic and transcriptomic changes.

Detailed Description

This is a biological study for adult MDS patients who undergo HSCT procedure.The research is focused on the study of biological mechanisms of disease relapse after HSCT in MDS to provide the rationale to develop pre-emptive strategies in patients with high risk of transplant failure.

Viable bone marrow samples will be collected and cryopreserved from MDS patients before transplantation and at clinical disease recurrence.

CD34+ blast cells at disease relapse after HSCT will be compared with CD34+ cells collected before transplant by using Single-cell sequencing.

To study genomic and transcriptomic changes in CD34+ blast cells at disease relapse after HSCT will be used TARGET-seq in parallel with unbiased whole-transcriptome analysis.

Study Timeline

• Study First Submitted: 2020-09-24
• Study First Submitted QC: 2020-10-12
• Study First Posted: 2020-10-14
• Study Start: 2021-02-12
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-21
• Last Update Posted: 2024-10-22
• Primary Completion: 2025-03
• Study Completion: 2025-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 38

Inclusion Criteria

• Diagnosis of MDS according to 2016 WHO criteria
• Aged 18y or older
• Patients who will receive allogeneic stem cell transplantation (HSCT)
• Signed written informed consent according to ICH/EU/GCP and national local laws

Exclusion Criteria

• Second HSCT procedure.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Biological evaluation	bone marrow withdrawal	evaluation of molecular changes in CD34+ blast cells at the time of relapse after allo-HSCT.

Primary Outcome Measures

Name	Time	Method
CD34+ blast cells modifications	at 24 months	Difference in frequency distribution of CD34+ blast cells mutations between transplant and relapse.

Trial Locations

Locations (7)

Aou Consorziale Policlinico - Bari - Uo Ematologia Con Trapianto; 🇮🇹 Bari, Italy

Unità di Ricerca e di Malattie del sangue - Ematologia S. Luca Vecchio; 🇮🇹 Firenze, Italy

Sezione di Ematologia - Ospedale S.Maria della Misericordia; 🇮🇹 Perugia, Italy

Dip. di Medicina Trasl. E di Precisione - U.O.C. Ematologia; 🇮🇹 Roma, Italy

Policlinico Tor Vergata; 🇮🇹 Roma, Italy

AOU San Martino-IST-Ematologia 1; 🇮🇹 Genova, Italy

Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia; 🇮🇹 Rozzano, Italy