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Using stem cell transplantation for high-risk MDS patients

Prospective Randomized Study on the Feasibility of Allogeneic Stem Cell Transplantation in Higher-risk-myelodysplastic Syndromes, Performed Upfront or Preceded by Azacitidine or Conventional Chemotherapy According to the BM-blast Proportion

PHASE3 · Gruppo Italiano Malattie EMatologiche dell'Adulto · NCT04184505

This study is testing whether a stem cell transplant right away or after treatment with a specific drug is better for people with high-risk myelodysplastic syndromes.

Quick facts

PhasePHASE3
Study typeInterventional
Enrollment274 (estimated)
Ages18 Years to 70 Years
SexAll
SponsorGruppo Italiano Malattie EMatologiche dell'Adulto (other)
Drugs / interventionschemotherapy
Locations46 sites (Alessandria and 45 other locations)
Trial IDNCT04184505 on ClinicalTrials.gov

What this trial studies

This is an open-label, randomized multicenter phase III study designed to evaluate the feasibility of allogeneic stem cell transplantation (HSCT) in patients with higher-risk myelodysplastic syndromes (MDS). The study compares two approaches: one involving hematopoietic stem cell transplantation after treatment with azacitidine or standard chemotherapy, and the other involving upfront HSCT. The trial aims to determine whether these approaches are non-inferior in terms of feasibility for patients with different levels of bone marrow blasts. Participants will be monitored for their response to treatment and overall outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18-70 with newly diagnosed higher-risk MDS who have not received prior treatment.

Not a fit: Patients with acute myeloid leukemia or severe organ impairment may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment pathway for patients with high-risk MDS, potentially improving their survival rates.

How similar studies have performed: Other studies have shown promise in using stem cell transplantation for MDS, but this specific approach is being evaluated for the first time in this context.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Patients with newly diagnosed higher-risk MDS, including IPSS Intermediate-2 and high, and IPSS-R intermediate to very-high
2. Age 18-70 years
3. Previously untreated for HR-MDS
4. HSCT - eligible
5. Life expectancy ≥3 months;
6. Signed written informed consent according to ICH/EU/GCP and national local laws
7. Eastern Cooperative Oncology Group Performance Status Grade of 0-2

Exclusion Criteria:

1. Acute myeloid leukaemia with \>20% blasts in BM or peripheral blood (PB);
2. concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma);
3. severe renal, cardiac, liver or lung impairment;
4. pregnant or lactating or potentially fertile (both males and females), who have not agreed to avoid pregnancy during the trial period; Women of childbearing potential and men must agree to use effective contraception during and up to 3 months after treatment with azacitidine.
5. HIV infection; active, uncontrolled HCV or HBV infections or liver cirrhosis;
6. clinically relevant neurological or psychiatric diseases;
7. hypersensitivity (known or suspected) to AZA;
8. prior Treatments:

   1. prior investigational drugs (within 30 days);
   2. radiotherapy, chemotherapy, or cytotoxic therapy for non-MDS conditions within the previous 6 months;
   3. growth factors (EPO, G-CSF or GM-CSF) during the previous 21 days;
   4. androgenic hormones during the previous 14 days;
   5. prior transplantation or cytotoxic therapy, including azacitidine, AZA or chemotherapy, administered to treat MDS (a previous treatment with Lenalidomide is admitted, provided that lenalidomide had been stopped at least 60 days before enrolment).

Where this trial is running

Alessandria and 45 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: High-risk MDS, MDS, Transplant, Azacitidine

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.