Study Comparing Oral Azacitidine and Cedazuridine with Subcutaneous Azacitidine for Patients with Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia

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What is this study about?

This clinical trial is focused on studying the effects of a new treatment for certain blood-related diseases. The diseases being studied are Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), and Acute Myeloid Leukemia (AML). These are conditions where the bone marrow does not produce enough healthy blood cells. The treatment being tested is a combination of two drugs, Azacitidine and Cedazuridine, which are taken orally in capsule form. This combination is referred to by the code name ASTX030. The study will compare this oral treatment to an existing treatment where Azacitidine is given as an injection under the skin.

The purpose of the study is to see if the new oral treatment is as effective as the injection form. Participants in the study will receive either the oral treatment or the injection, and then switch to the other form during the study. This is known as a crossover study. The study will take place over several phases, starting with a dose-escalation phase to determine the best dose of the oral treatment, followed by a phase where the effectiveness of the oral treatment is compared to the injection.

Throughout the study, researchers will monitor participants for any side effects and measure how well the treatment works in controlling the diseases. The study aims to provide a more convenient treatment option for patients with these conditions, potentially improving their quality of life by offering an oral medication instead of injections. The study is expected to continue until 2027.

1 initial assessment

Upon joining the study, an initial assessment will be conducted to confirm eligibility. This includes verifying age, medical history, and current health status.

The assessment ensures that the participant has a confirmed diagnosis of myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), or acute myeloid leukemia (AML).

2 treatment phase 1

Participants will begin the first phase of treatment, which involves taking the oral medication ASTX030, a combination of cedazuridine and azacitidine.

The dosage and frequency will be determined based on the dose-escalation study results. Participants will be monitored for any side effects or adverse reactions.

3 treatment phase 2

In the second phase, participants will continue with the oral medication ASTX030 at the selected dose from phase 1.

This phase aims to establish the equivalence of the oral medication to the subcutaneous form of azacitidine.

4 crossover phase

Participants will switch to the subcutaneous form of azacitidine, administered at 75 mg/m² per day.

This phase allows for comparison between the oral and subcutaneous forms of the medication.

5 monitoring and follow-up

Throughout the study, participants will undergo regular monitoring to assess the effectiveness of the treatment and any potential side effects.

Follow-up visits will be scheduled to evaluate the overall health and response to the treatment.

Who Can Join the Study?

Must be 18 years of age or older at the time of signing the informed consent.
Must follow sex and contraceptive barrier requirements as defined in the study protocol.
Is capable of giving legally effective informed consent, which means understanding and agreeing to the study’s requirements and restrictions, and is willing to participate in the study.
Has confirmed Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML) and is a candidate to receive and benefit from single-agent azacitidine treatment. This includes specific subtypes of MDS such as refractory anemia or refractory anemia with ringed sideroblasts (if accompanied by low white blood cells, low platelets, or requiring transfusions), refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and CMML or MDS with intermediate-2 or high risk according to the International Prognostic Scoring System (IPSS).
Has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1, which means being fully active or restricted in physically strenuous activity but able to carry out light work.
Has adequate organ function, which means:
- Liver function: Total or direct bilirubin levels are less than or equal to 2 times the upper limit of normal; AST/SGOT and ALT/SGPT levels are less than or equal to 2.5 times the upper limit of normal.
- Kidney function: Calculated creatinine clearance is greater than 50 mL/min, which is a measure of how well the kidneys are working.
For those with a prior allogeneic stem cell transplant, there must be no evidence of graft-versus-host disease, and they must be off systemic immunosuppressive therapy for at least 3 weeks before starting the study treatment.
Has had no major surgery within 3 weeks before the first study treatment.
Has had no cytotoxic chemotherapy (except hydroxyurea) within 4 weeks before the first study treatment.
Is able to swallow the required number of tablets or capsules within a 10-minute period and tolerate 4 hours of fasting.
Has a projected life expectancy of at least 12 weeks.

Who Cannot Join the Study?

Patients with any other serious medical condition that might affect their participation in the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial within the last 30 days.
Patients with known allergies to the study medication or its ingredients.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients with uncontrolled infections or other active diseases that could interfere with the study.
Patients who are unable to comply with the study procedures or follow-up visits.
Patients with a history of drug or alcohol abuse that could interfere with their ability to participate in the study.
Patients who are currently receiving treatment with other investigational drugs.
Patients with a history of certain heart conditions that could pose a risk during the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Hospital Universitario De Salamanca	Salamanca	Spain
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Oncopole Claudius Regaud	Toulouse	France
Hospital Universitario De Navarra	Pamplona	Spain
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA	Rome	Italy

Other Sites

Site Name	City	Country
Staedtisches Klinikum Braunschweig gGmbH	Brunswick	Germany
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hospital San Pedro De Alcantara	Caceres	Spain
Hospital Universitario Virgen De Las Nieves	Granada	Spain
Azienda Ospedaliera Ordine Mauriziano Di Torino	Turin	Italy
MD Anderson Cancer Center	Madrid	Spain
Hospital Quironsalud Malaga	Malaga	Spain
University Of Debrecen	Debrecen	Hungary
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Hospital Clinic De Barcelona	Barcelona	Spain
Fakultni Nemocnice Brno	Brno	Czechia
Semmelweis University	Budapest	Hungary
Fakultni Nemocnice Kralovske Vinohrady	Prague	Czechia
Hospital Universitari De Girona Doctor Josep Trueta	Girona	Spain
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Samodzielny Publiczny Szpital Kliniczny Nr 1 W Lublinie	Lublin	Poland
Centre Hospitalier Universitaire De Nice	Nice	France
Azienda Ospedaliero-Universitaria Maggiore Della Carita	Novara	Italy
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
Azienda Unita Sanitaria Locale Della Romagna	Faenza	Italy
Hopital Beaujon	Clichy	France
University Of Szeged	Szeged	Hungary
University Hospital Ostrava	Ostrava	Czechia
Ircvpecq Cveblj Dpjxmuwpykuniiaex	L'hospitalet De Llobregat	Spain
Wjspqpfxtgn Wkqhwjrvbhnoyatizdrb Czpjzee Oftjafgvt I Tzyrxrvzqoyxg Iq Mndetzdawvt W Lrcur	Lodz	Poland
Sftiymfj Pxtumvxvl Sqj z ottv	Gdynia	Poland
Apxxlrw Orwramorqqn Ufuutpesdslnq Ctpahceiuejk Diqei Surxjg E Dfhyj Sgvatkt Df Tnwxai	Turin	Italy
Ghsnbofowevrizcmx Vzwprjsez Pcyg Awqjyc Egbhidxu Oohsni Ksqhgq	Gyor	Hungary
Akieeom Uwtli Ssklvpodr Lmqlvs De Biygwma	Bologna	Italy
Uypjgxhbex Dqrqu Shjlz Da Rmzj Lg Spvuppof	Rome	Italy
Majqbwvxibefhtqxmnezvjpihr Hgoonkpeznfyszle	Halle (Saale)	Germany
Fsmqwqhgn Przq Lr Ievxnnywskyvg Bqwwosxnl Drk Hceeuoee Upeofszmgzpqs Lr Pca	Madrid	Spain
Hplkdndz Vivy ductzdsc	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Czechia	Recruiting	16.06.2025
France	Recruiting	16.06.2025
Germany	Recruiting	16.06.2025
Hungary	Recruiting	16.06.2025
Italy	Recruiting	16.06.2025
Poland	Recruiting	16.06.2025
Spain	Recruiting	16.06.2025

Trial locations

Investigated drugs:

ASTX030 is a combination of two medications, Cedazuridine and Azacitidine, taken by mouth. This combination is being studied to see if it can be as effective as the standard treatment given by injection under the skin. The goal is to make treatment easier for patients by allowing them to take the medication orally instead of through injections.

Azacitidine is a medication that is usually given by injection under the skin. It is used to treat certain blood disorders and types of leukemia by helping to slow down the growth of abnormal blood cells. In this study, it is being compared to the oral combination of ASTX030 to see if they work equally well.

Investigated diseases:

Myelodysplastic Syndromes (MDS) – Myelodysplastic Syndromes are a group of disorders caused by poorly formed or dysfunctional blood cells. These disorders occur 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 anemia, infections, or bleeding. The progression of MDS can vary, with some cases remaining stable for years, while others may progress to more severe forms of blood disorders. The condition is characterized by a shortage of one or more types of blood cells, such as red blood cells, white blood cells, or platelets.

Chronic Myelomonocytic Leukemia (CMML) – Chronic Myelomonocytic Leukemia is a type of cancer that starts in blood-forming cells of the bone marrow and invades the blood. It is characterized by an increase in monocytes, a type of white blood cell, in the blood and bone marrow. CMML can lead to symptoms such as fatigue, fever, and weight loss due to the overproduction of abnormal cells. Over time, the disease can progress, causing a decrease in normal blood cells, which may result in anemia, increased risk of infections, and bleeding issues. The disease may remain stable for a period but can eventually transform into acute leukemia.

Acute Myeloid Leukemia (AML) – Acute Myeloid Leukemia is a cancer of the blood and bone marrow characterized by the rapid growth of abnormal white blood cells. These cells accumulate in the bone marrow, interfering with the production of normal blood cells. AML progresses quickly and can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. The disease is marked by the presence of immature white blood cells, known as myeloblasts, in the blood and bone marrow. As the disease advances, it can cause a significant reduction in red blood cells, white blood cells, and platelets.

Trial ID:

2024-515098-93-00

Protocol code:

ASTX030-01

NCT ID:

NCT04256317

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study Comparing Oral Azacitidine and Cedazuridine with Subcutaneous Azacitidine for Patients with Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?