Study on Oral Arsenic Trioxide for Patients with Low-Risk Myelodysplastic Syndromes Not Responding to Erythropoiesis Stimulating Agents and Luspatercept

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What is this study about?

This clinical trial is focused on studying a group of blood disorders known as Myelodysplastic Syndromes (MDS). These are conditions where the bone marrow does not produce enough healthy blood cells. The study specifically targets low-risk MDS patients who have not responded to treatments called Erythropoiesis Stimulating Agents and Luspatercept, or who are not eligible for these treatments. The trial will use a medication called Arsenic Trioxide, which will be given in capsule form to be taken orally.

The purpose of the study is to evaluate the safety and effectiveness of Arsenic Trioxide in these patients. The study will be conducted in two parts. The first part will focus on determining the highest dose of the medication that can be given safely. The second part will assess how well the medication works in improving the production of red blood cells over a 12-week period. Participants will take the medication for up to 12 months, with regular check-ups to monitor their health and response to the treatment.

Throughout the study, participants will be closely monitored for any side effects and their overall health will be assessed. The trial aims to provide valuable information on the potential benefits of Arsenic Trioxide for patients with low-risk Myelodysplastic Syndromes who have limited treatment options. This research could lead to new ways to manage and treat this condition, improving the quality of life for those affected.

1 initial treatment phase

The study begins with the administration of oral arsenic trioxide in capsule form. The available dosages are 5 mg, 3 mg, and 1 mg.

The medication is taken by mouth. The specific dosage and frequency will be determined by the study team based on individual needs and responses.

2 observation period

An observation period occurs from day 28 to day 42 following the start of the first cycle. This period is used to monitor for any dose-limiting side effects of the treatment.

3 expansion phase

After the initial phase, the study enters an expansion phase. The main goal is to evaluate the response of red blood cells to the treatment over a 12-week period.

The response is assessed to determine the effectiveness of the treatment in improving blood cell counts.

4 response assessment

The response to the treatment is evaluated after the third cycle, using specific criteria to measure improvement.

The duration of the response is also measured, from the date of initial improvement to any signs of relapse or progression.

5 safety and tolerability evaluation

Throughout the study, the safety and tolerability of the treatment are closely monitored.

This includes regular assessments to ensure that any side effects are manageable and to compare the oral treatment to previous intravenous forms.

6 long-term monitoring

The study includes long-term monitoring to track progression-free survival and overall survival from the start of the study to the end or last follow-up.

Additional exploratory analyses may be conducted to identify factors associated with survival and response to treatment.

Who Can Join the Study?

The patient must have a condition called Myelodysplastic Syndrome as per the WHO 2022 classification.
The patient must have adequate kidney function, which means their creatinine level (a waste product in the blood) should be less than 1.5 times the normal limit, and their creatinine clearance (a measure of kidney function) should be at least 40 mL/min.
The patient must have adequate liver function, meaning their total bilirubin and transaminases (liver enzymes) should be less than 1.5 times the normal limit.
The patient should not be resistant to platelet transfusions.
If the patient has diabetes, it should be well-controlled with an HbA1c level (a measure of blood sugar control) of 7.5% or lower before joining the study.
Females of childbearing potential must have two negative pregnancy tests before starting the study and agree to monthly pregnancy tests during and after the study. They must use highly effective birth control methods if sexually active.
Male participants must agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while in the study and for at least 24 weeks after stopping the study treatment.
The patient must be at least 18 years old.
The patient must have low-risk Myelodysplastic Syndrome according to a specific classification and meet certain criteria related to previous treatments.
The patient must need regular blood transfusions, specifically at least 3 red blood cell transfusions within a 16-week period and at least 2 separate transfusion episodes during this time.
The patient must not be eligible for another clinical trial.
The patient must understand and voluntarily sign an informed consent form.
The patient must be able to follow the study’s visit schedule and requirements.
The patient must have a performance status of 0-2, which is a measure of their general well-being and ability to perform daily activities.

Who Cannot Join the Study?

Patients who have not failed treatment with Erythropoiesis Stimulating Agents or are not ineligible for Luspatercept. Erythropoiesis Stimulating Agents are medicines that help the body make more red blood cells. Luspatercept is a medication used to treat certain blood disorders.
Patients who do not have low-risk Myelodysplastic Syndromes. This is a group of disorders caused by poorly formed or dysfunctional blood cells.
Patients who are not within the specified age range for the study.
Patients who are not part of the specified clinical trial groups.
Patients who are not considered part of a vulnerable population.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Institut Gustave Roussy	Villejuif	France

Other Sites

Site Name	City	Country	Status
Centre Hospitalier Universitaire De Nice	Nice	France
Hopital Beaujon	Clichy	France

Trial status

Country	Status	Recruitment Start
France	Recruiting	04.07.2025

Trial locations

Investigated drugs:

Arsenic Trioxide

Arsenic (ATO) is being studied to see how safe and effective it is for people with low-risk Myelodysplastic Syndromes who have not responded well to other treatments. This trial is looking at how the body handles the medication and what side effects might occur. The goal is to find out the best dose that can be given without causing too many side effects and to see if it helps improve blood cell production after 12 weeks of treatment.

Myelodysplastic Syndromes – Myelodysplastic Syndromes (MDS) are a group of disorders caused by poorly formed or dysfunctional blood cells. In MDS, the bone marrow does not produce enough healthy blood cells, leading to symptoms like fatigue, infections, and easy bruising. The condition is characterized by ineffective hematopoiesis, which means the bone marrow fails to produce adequate numbers of blood cells. Over time, MDS can lead to severe anemia, increased risk of bleeding, and susceptibility to infections. Some cases of MDS may progress to acute myeloid leukemia, a more aggressive form of blood cancer. The disease is often classified based on the risk of progression and the severity of symptoms.

Trial ID:

2024-515311-22-00

Protocol code:

ATOMYELO

NCT ID:

NCT06670222

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on Oral Arsenic Trioxide for Patients with Low-Risk Myelodysplastic Syndromes Not Responding to Erythropoiesis Stimulating Agents and Luspatercept

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?