Study on the Effectiveness and Safety of Oral Azacitidine for Patients with Low or Intermediate-Risk Myelodysplastic Syndrome

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What is this study about?

This clinical trial is focused on studying a condition known as Myelodysplastic Syndrome (MDS), which affects the blood and bone marrow. Specifically, the study targets individuals with low- or intermediate-risk MDS, as classified by a system called the International Prognostic Scoring System-Revised (IPSS-R). The treatment being tested is an oral medication called Azacitidine, available in 200 mg and 300 mg tablets. This medication is being compared to a placebo, with both groups receiving the best supportive care available.

The purpose of the study is to evaluate the safety and effectiveness of Azacitidine in treating MDS. The study is divided into two parts. In the first part, different doses of the medication will be tested to find the most effective and safe dose. In the second part, the chosen dose will be compared to a placebo to see how well it works in improving the condition. Participants will take the medication orally, and the study will monitor their response over several cycles of treatment.

Throughout the study, participants will be closely observed to assess any changes in their condition and to ensure their safety. The study aims to determine if Azacitidine can help achieve a complete response, which means a significant improvement in blood cell counts and overall health. The results will help in understanding the potential benefits of this treatment for people with low- or intermediate-risk Myelodysplastic Syndrome.

1 joining the study

Upon joining the study, you will be randomly assigned to one of two groups. One group will receive the medication oral azacitidine, and the other group will receive a placebo. A placebo is a substance with no active medication, used to compare the effects of the actual drug.

2 medication administration

If you are in the group receiving oral azacitidine, you will take either a 200 mg or 300 mg tablet. The exact dosage will be determined during the study. The medication is taken by mouth, and you will follow the specific instructions provided by the study team regarding how often and for how long you need to take the medication.

3 monitoring and assessments

Throughout the study, you will undergo regular monitoring and assessments. This includes checking your vital signs, such as blood pressure and heart rate, and performing laboratory tests to evaluate your health and the effects of the medication. These assessments help ensure your safety and measure the effectiveness of the treatment.

4 evaluation of results

The study aims to evaluate the complete response (CR) within 6 cycles of treatment. A complete response means that there are no signs of the disease after treatment. The study will also assess other outcomes, such as the duration of response and any side effects you may experience.

5 completion of the study

At the end of the study, you will have a final evaluation to assess your overall health and the outcomes of the treatment. The study team will provide you with information about your participation and any next steps, if necessary.

Who Can Join the Study?

  • Participants must be 18 years of age or older at the time of signing the consent form.
  • Participants must have a documented diagnosis of Myelodysplastic Syndrome (MDS) according to the 2016 World Health Organization (WHO) classification. This should meet the International Prognostic Scoring System-Revised (IPSS-R) classification of low- or intermediate-risk disease.
  • Participants must have at least one cytopenia, which means a low count of blood cells. This can include anemia (low red blood cells), thrombocytopenia (low platelets), or neutropenia (low white blood cells).
  • Participants must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2. This is a scale used to assess how a patient’s disease is progressing, including their level of functioning and ability to care for themselves.

Who Cannot Join the Study?

  • Patients with a different type of blood disorder than the one being studied cannot participate.
  • Patients who have had another type of cancer in the past, unless it was treated and has not come back for at least 5 years, cannot participate.
  • Patients who are currently receiving treatment for another type of cancer cannot participate.
  • Patients who have had a heart attack or severe heart problems in the past 6 months cannot participate.
  • Patients with uncontrolled high blood pressure cannot participate.
  • Patients with severe liver disease cannot participate.
  • Patients with severe kidney disease cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are not willing to use effective birth control during the study cannot participate.
  • Patients who have participated in another clinical trial within the last 30 days cannot participate.
  • Patients who have an allergy to the study medication or similar drugs cannot participate.
  • Patients with any other medical condition that the study doctors think would make it unsafe for them to participate cannot participate.

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 Ospedaliera di Padova Padua Italy
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Region Skane Skanes Universitetssjukhus Lund Sweden
Fakultni Nemocnice Brno Brno Czechia
Fakultni Nemocnice Hradec Kralove Novy Hradec Kralove Czechia
Centre Hospitalier Universitaire De Nice Nice France
University General Hospital Of Alexandroupoli Alexandroupoli Greece
Pratia Hematologia Sp. z o.o. Katowice Poland
Grande Ospedale Metropolitano Bianchi Melacrino Morelli Reggio Calabria Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Karolinska University Hospital Solna Sweden
Marien Hospital Duesseldorf GmbH Duesseldorf Germany
Hanusch Krankenhaus Der Wiener Gebietskrankenkasse Vienna Austria
Aalborg University Hospital Aalborg Denmark
Region Midtjylland Aarhus Denmark
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum) Munich Germany
Region Oerebro Laen Orebro Sweden
HELIOS Klinikum Duisburg GmbH Duisburg Germany
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Geniko Nosokomeio Thessalonikis George Papanikolaou Thessaloniki Greece
Hospital Universitario Virgen De Las Nieves Granada Spain
Szpital Kliniczny Ministerstwa Spraw Wewnetrznych I Administracji Z Warminsko-Mazurskim Centrum Onkologii W Olsztynie Olsztyn Poland
OncoResearch Lerchenfeld GmbH Hamburg Germany
Odense University Hospital Odense Denmark
Uniklinikum Salzburg Salzburg Austria
Klinikverbund Allgau gGmbH Immenstadt im Allgäu Germany
Apedudsovu Pnyhnife Helwvqje Dr Piyfw Paris France
Cunuri Htuglfcjkcq Rcdfdtra Udhozawrluicg Dh Tbvxq Tours France
Ailrxgg Uxfzh Sqgpkfmxj Lquwsl Du Bdxmbxu Bologna Italy
Otajmseagwczzj Lbxg Gbfp Linz Austria
Hootxqed Ueteiozrmmjga Dt Lk Prpndyrd Madrid Spain
Ihvflswz Clddkv Dhuuuilwstxjflkog L'hospitalet De Llobregat Spain
Cdnaej Hxjjjtilper Rrczpald Danoxivizlzpcd Angers France
Krglrdxr Ocydsl goudbcaspapmys kahyzhivb Ahtcczp dfo oivqsrrrsfuwl Ruppkq Aalen Germany
Hjoqtmyr Vbqr dcewdmte Barcelona Spain
Iijihawj Pgagczaztnknjpg Cqnxyp Ckwipp Marseille France
Uknvcorfjf Gzbwytc Hvebcnnf Anevzsn Athens Greece

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

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
30.03.2023
Czechia Czechia
Not recruiting
30.03.2023
Denmark Denmark
Not recruiting
30.03.2023
France France
Not recruiting
30.03.2023
Germany Germany
Not recruiting
30.03.2023
Greece Greece
Not recruiting
30.03.2023
Italy Italy
Not recruiting
30.03.2023
Poland Poland
Not recruiting
30.03.2023
Spain Spain
Not recruiting
30.03.2023
Sweden Sweden
Not recruiting
30.03.2023

Trial locations

Investigated drugs:

Oral Azacitidine (Oral-Aza, ONUREG®) is a medication used in this clinical trial to treat a condition called myelodysplastic syndrome (MDS), which affects the blood and bone marrow. This medication is taken by mouth and works by helping to slow down or stop the growth of abnormal blood cells. The goal of using Oral Azacitidine in this study is to see if it can improve the health of patients with low- or intermediate-risk MDS by reducing the number of abnormal cells and improving blood cell counts. The study aims to find the best dose that balances effectiveness and safety for patients.

Myelodysplastic Syndrome (MDS) – Myelodysplastic Syndrome is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when something goes wrong in the bone marrow, where blood cells are produced. Over time, the bone marrow becomes less effective at producing healthy blood cells, leading to a shortage of red blood cells, white blood cells, and platelets. This can result in symptoms such as fatigue, infections, and easy bruising or bleeding. The disease can progress slowly or quickly, and in some cases, it may transform into acute myeloid leukemia. The progression of MDS varies greatly among individuals, depending on the specific type and severity of the syndrome.

Trial ID:
2022-500479-29-00
Protocol code:
CA055-026
NCT ID:
NCT05469737
Trial Phase:
Therapeutic use (Phase IV)

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