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

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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
Ospedale Vito Fazzi Lecce Lecce Italy
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania Catania Italy
Azienda Ospedaliero-Universitaria Sant Andre Rome Italy
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Azienda Unita Sanitaria Locale Di Piacenza Piacenza Italy
Azienda Sanitaria Universitaria Friuli Centrale Udine Italy
San Camillo Forlanini Hospital Rome Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone Palermo Italy
Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d’Aragona Salerno Italy
Azienda Sanitaria Locale Di Pescara Pescara Italy
Azienda Ospedaliero Universitaria Ospedali Riuniti Umberto I G M Lancisi G Salesi Ancona Italy
Grande Ospedale Metropolitano Bianchi Melacrino Morelli Reggio Calabria Italy
Ospedale Santa Maria Goretti Latina Latina Italy
Istituto Oncologico Veneto Padua Italy
Azienda Sanitaria Locale Viterbo Viterbo Italy
Azienda Sanitaria Locale Di Taranto Taranto Italy
Pia Fondazione Di Culto E Religione Card G Panico Tricase Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Ospedale San Raffaele S.r.l. Milan Italy
ASST Grande Ospedale Metropolitano Niguarda Milan Italy
Universita’ Campus Bio-medico Di Roma Rome Italy
Universita’ Degli Studi Di Verona Verona Italy
Universita’ Di Pisa Pisa Italy
Azienda Unita Sanitaria Locale Della Romagna Faenza Italy
Azienda Sanitaria Territoriale Di Ascoli Piceno Ascoli Piceno Italy
Universita Degli Studi Di Brescia Brescia Italy
Universita’ Degli Studi Di Ferrara Ferrara Italy
Ospedale S. Eugenio, ASL Roma 2 Rome Italy
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Axmfsll Ocumyqmqtbz Ufepougjkfkjz Cibwstxkdfqi Dovpf Sodkph E Dotrc Smjybbn Dx Thjhxa Turin Italy
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Aajugaa Owlencwfwki Urfswtuojjzmu Ocpewkdr Riejnlo Foggia Italy
Akhzcdw Unb Ihlir Da Rfzudi Eenvbm Reggio Emilia Italy
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Ulxpyvnzox Dzske Ssvpm Dl Rxnb Ln Snyablqh Rome 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.

Investigated diseases:

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)

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