Using stem cell transplantation for high-risk MDS patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 058 - Aon Ss. Antonio E Biagio E C. Arrigo - Soc Ematologia
  Alessandria, Italy Recruiting
• Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi - Sod Clinica Ematologica
  Ancona, Italy Recruiting
• Presidio Ospedaliero Av5 Osp. Gen. Prov.Le "C.G.Mazzoni" - Uoc Ematologia
  Ascoli Piceno, Italy Recruiting
• Aou Consorziale Policlinico - Bari - Uo Ematologia Con Trapianto
  Bari, Italy Recruiting
• Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
  Bologna, Italy Recruiting
• Asst Degli Spedali Civili Di Brescia - Uo Ematologia
  Brescia, Italy Recruiting

Study contacts

• Principal investigator: Voso — Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali
• Study coordinator: Paola Fazi
• Email: p.fazi@gimema.it
• Phone: 0670390528

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• REGN17235 for CCUS and LR-MDS with SF3B1 mutation

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.