Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

RecruitingRare disease

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

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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. Step 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. Step 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 trial?

14 criteria

  • 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 trial?

5 criteria

  • 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.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

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.

What is already known about the treatment

Arsenic (ATO) – This medication is administered orally and is currently being studied in clinical trials for its safety and effectiveness in treating low-risk Myelodysplastic Syndromes that do not respond to Erythropoiesis Stimulating Agents and Luspatercept, or for patients who are not eligible for these treatments. The main therapeutic indication is to improve blood cell production in these specific syndromes. At the molecular level, arsenic works by interfering with cellular processes that lead to the death of abnormal cells, thereby potentially improving blood cell counts. It is classified pharmacologically as an antineoplastic agent.

Investigated diseases

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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-515311-22-00Protocol codeATOMYELOEstimated enrolment24 patientsSponsorGroupe Francophone Des Myelodysplasies

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).