Study of Azacitidine and Drug Combination Before Stem Cell Transplantation in Patients with High-Risk Myelodysplastic Syndrome

3 1 1 1

What is this study about?

This study focuses on patients with Higher-risk Myelodysplastic Syndromes, a group of blood disorders where bone marrow does not produce enough healthy blood cells. The study evaluates two different treatment approaches: immediate hematopoietic stem cell transplantation (a procedure where damaged bone marrow is replaced with healthy stem cells) or transplantation after receiving other treatments.

The main treatment being studied is Azacitidine, which is given as an injection under the skin. Other medications that may be used in the study include Fludarabine, Daunorubicin, Busulfan, Cytarabine, and Thiotepa. These medications are given through an intravenous line directly into the bloodstream.

The purpose of this research is to determine whether it is better to perform stem cell transplantation immediately or to first treat patients with medications like Azacitidine or traditional chemotherapy, depending on the number of abnormal cells in their bone marrow. The study will monitor patients’ health and recovery throughout their treatment period and follow-up care.

1 Initial evaluation

A medical assessment will confirm if you have higher-risk myelodysplastic syndrome (a bone marrow disorder)

Your bone marrow will be examined to determine the percentage of abnormal cells (blasts)

Your overall health status and ability to perform daily activities will be evaluated

2 Treatment assignment

Based on your bone marrow blast percentage, you will be assigned to one of two treatment paths:

If blast percentage is below 10%: direct stem cell transplantation

If blast percentage is 10% or higher: pre-treatment before transplantation

3 Pre-transplant treatment (if required)

You may receive one of these treatments:

Azacitidine – given by injection under the skin

Conventional chemotherapy combination including: fludarabine, daunorubicin, cytarabine – given through intravenous infusion

4 Stem cell transplantation

Preparation with conditioning medications: busulfan and thiotepa given through intravenous infusion

Stem cell transplantation procedure

Hospital stay for monitoring and recovery

5 Follow-up evaluations

Bone marrow samples will be collected before transplant and 6 months after

Quality of life assessments will be conducted

Regular health monitoring will continue for several years

Final study evaluation will be completed by September 2031

Who Can Join the Study?

  • Must be diagnosed with higher-risk myelodysplastic syndromes (HR-MDS) that has not been treated before – this is a blood disorder affecting bone marrow
  • Must be between 18 and 70 years old
  • Must be eligible for HSCT (stem cell transplantation – a procedure where damaged bone marrow is replaced with healthy cells)
  • Must have a life expectancy of at least 3 months
  • Must be able to perform daily activities with minimal assistance – specifically an ECOG Performance Status of 0-2 (a scale that measures a person’s ability to perform daily activities and self-care)
  • Must be willing and able to provide written informed consent to participate in the study
  • Must not have received any previous treatment for HR-MDS

Who Cannot Join the Study?

  • Age below 18 years or above 65 years
  • Previous history of bone marrow transplant (a procedure where damaged bone marrow is replaced with healthy cells)
  • Active uncontrolled infections
  • Presence of more than 10% blast cells (immature blood cells) in bone marrow
  • Severe heart conditions including:
    • Uncontrolled heart failure
    • Recent heart attack (within last 6 months)
    • Unstable heart rhythm problems
  • Severe kidney or liver problems
  • Pregnancy or breastfeeding
  • Active other types of cancer
  • Mental conditions that prevent understanding of study requirements
  • Participation in other clinical trials within past 30 days
  • Known allergic reactions to study medications
  • Inability to follow study procedures

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Azienda Sanitaria Locale Di Taranto Taranto Italy
Ospedale San Raffaele S.r.l. Milan Italy
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Ospedale S. Eugenio, ASL Roma 2 Rome Italy
University Hospital Consorziale Policlinico Bari Italy
Azienda Ospedaliera Universitaria Senese Siena Italy
Ftqxbpprrn Ibjes Cz Gqsxsi Oqhqbgql Mpgkgyez Pmipgdzeieg Milan Italy
Aeey Giaftz Owdtodeb Mpduthbxtifqo Nrskrcym Milan Italy
Uztqtslhavzzmibru Ducog Sxjkz Df Vggnip Verona Italy
Aqdabhh Oxoiygmlkon Usbkmitdqibel Crjssyiaadjw Dnpsf Sfcqqo E Dxfqr Sclgzuk Dh Tncgxh Turin Italy
Aeogqyp Ofxiwenacll Uvephfkkkvlxw Fpgriofe Iv Df Nvznzc Naples Italy
Ahitqqn Okuvoimpcnk Uvhoxvhhxwydl Omxlzxzi Refqmif Foggia Italy
Atpcrdu Uqznt Sybctklbf Lyfczv Dl Bwzsfsy Bologna Italy
Icvxgr Iupdatpt Fapqqligaiyrg Odugujaajsk Rome Italy
Uyrsihfugh Dvslp Sqhfu Dw Rzms Lx Sznokzic Rome Italy
Awymnln Sycpffymb Uljjirfbpgcuf Fisycr Cgimosta Udine Italy
Ubkezgchtk Djlhk Snpeo Do Birhlsk Brescia Italy
Uxgjzgjaaqpewrrxv Dphgq Sugta Db Fbthjxs Ferrara Italy
Awkprvq Owzvbdmhizx S Gywzeqzp Aggahaeaeg Rome Italy
Fszvglxgao Ikvsk Sfc Gjjkwdl Dam Tzhlgrz Monza Italy
Alagoop Ofzpztypqsc Uicuzyhyehfvd Psktrk Pisa Italy
Iaetvdrh Ohbhtlchfh Vyvbxk Padua Italy
Arpqlgv Omgiqxlcfwz Uiwuotdhgyxbk Oqjumbxz Ryzlxxe Ubmbyxw I G M Lypzwcu G Slthzh Ancona Italy
Outxeqav Vhuu Ffunk Lqtpl Lecce Italy
Adjuhwq Oovlrwotoajlgfkhqtjcqfmho Pcvcbnujwum Gj Rxcdtiwmxtat Mpmsd Dm Cuqwhel Catania Italy
Gxtcdw Ozyxiuoe Mymqldrjpjvah Bdjqqmi Mrycvpueu Mwcbeay Reggio Calabria Italy
Akwcvdp Sxozjqags Lecppo Vxcljre Viterbo Italy
Psb Fjxgasuaxt Dp Cqfuv E Rzidvaceu Ctyy G Pedtis Tricase Italy
Ueoukrorziboitfgb Cqlvre Buddisxsao Dh Rjgn Rome Italy
Aasbgqg Ovmctpjetfw Npgcuaqcc Su Arxqxhl E Bfnyyo E C Akkgej Aayludphwce Alexandria Italy
Axkbjlv Uuv Ibnpe Dg Rhsjoy Ezaejz Reggio Emilia Italy
Aktqmsa Uickb Sszyyfnoq Llvpxf Duquh Rluwhix Faenza Italy
Auvyqat Szltvxgpy Trkidpgbpqel Dq Arqvzc Pyxxle Ascoli Piceno Italy
Afqzmuc Oujmopgacfgeyhhvkhbkcivud Szfi Aeajf Rome Italy
Aaiefht Uemkj Sgnevisns Lsbmap Dx Pfndpxhs Piacenza Italy
Svk Coipfnb Fveosabbh Huwbamqn Rome Italy
Anxuupv Ojkbsyxnifx Uqqqrxpwgttjk Pgndovzardm Pndmw Gcfnaxbh Palermo Italy
Oonbeuox Spcio Mssum Ggjaxyr Luxmsu Latina Italy
Atlhrtk Opulbtlziop Uzmlileetyuso Shu Gbypazno Dc Drj E Royjg devkkaciugblnby Salerno Italy
Alnuofh Shkhdlkdl Lucpcv Da Pfufuhx Pescara Italy
Aiatdsm Smqbwwcyh Lkwmoo Dt Sexmtwi Battipaglia Italy

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Italy Italy
Recruiting
27.11.2020

Trial locations

Azacitidine is a medication used in treating myelodysplastic syndromes (MDS). It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. It’s given as an injection under the skin or through an intravenous line.

Allogeneic stem cell transplantation is a procedure where healthy blood-forming stem cells from a matched donor are transferred to the patient. These cells help create new, healthy blood cells in the patient’s body. This is not a medication but rather a therapeutic procedure that can potentially cure myelodysplastic syndromes.

Conventional chemotherapy refers to standard cancer-fighting drugs that are used to treat myelodysplastic syndromes. These medications work by killing rapidly dividing cells, including cancer cells. They are typically given through an intravenous line.

Myelodysplastic Syndromes (Higher-risk) – A group of bone marrow disorders where the bone marrow doesn’t produce enough healthy blood cells. In higher-risk forms, the bone marrow contains an increased number of immature blood cells called blasts. The condition affects the production of red blood cells, white blood cells, and platelets, leading to decreased blood cell counts. The bone marrow may produce blood cells that are abnormally shaped and don’t function properly. This condition can gradually worsen over time as the number of abnormal cells increases and healthy cells decrease.

Trial ID:
2023-510515-19-00
Protocol code:
MDS0519
NCT ID:
NCT04184505
Trial Phase:
Therapeutic confirmatory (Phase III)

Other Trials to Consider

  • A Phase 3 Study of Elritercept Versus Epoetin Alfa in ESA‑Naïve Adults with Lower‑Risk Myelodysplastic Syndrome Requiring Red Blood Cell Transfusions

    Recruiting

    3 1 1 1
    Investigated Drugs:
    Belgium Bulgaria France Germany Greece Hungary +8
  • Phase 1/2 Study of LB-208 in Adults with Relapsed or Refractory Acute Myeloid Leukaemia and High‑Risk Myelodysplastic Syndrome

    Recruiting

    1 1 1
    Spain