Could a Centuries-Old poison become a new weapon against a blood disorder?
NCT ID NCT06670222
First seen Aug 06, 2026 · Last updated Sep 11, 2026 · Updated 3 times
Summary
This early-stage trial is testing whether an oral form of arsenic trioxide can safely help people with low-risk myelodysplastic syndromes (MDS) who have not improved with standard treatments. The study will first find the safest dose, then check how well it works after several cycles. Participants are adults with low-risk MDS who have failed or are not eligible for other therapies.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- oral arsenic trioxide (ATO)
- What this could lead to
- If it works, oral arsenic could offer a new treatment option for people with low-risk myelodysplastic syndromes who have not responded to standard therapies.
- What could go wrong
- This is an early-phase trial with a small number of participants, so the benefits are uncertain. Arsenic can be toxic, and the study is primarily checking safety and the right dose.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 24 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2025
- Expected to finish
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Jul 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion criteria: Patients must meet all the following criteria to participate in the study: 1. Myelodysplastic syndrome according to WHO (World Health Organization) 2022 classification 2. Age ≥ 18 years 3. Patient with low-risk Myelodysplastic Syndromes according to Revised International Prognostic Scoring System (IPSS-R) classification (very low, low, intermediate): * non-sideroblastic who failed to achieved a response or who subsequently relapse after Erythropoiesis Stimulating Agents (ESA) (at Epoetin alfa 60000UI or equivalent over at least 12 weeks) without disease progression or ineligible to ESA (defined by Erythopoietine (EPO) \> 500UI/L) * sideroblastic who failed to achieved a response or who subsequently relapse after ESA (at Epoetin alfa 60000UI or equivalent over at least 12 weeks) or ineligible for ESA (defined by EPO \>500UI/L) and who failed to achieved a response or who subsequently relapse after Luspatercept * del (5q) who failed to achieved a response or who subsequently relapse after ESA (at Epoetin alfa 60000IU or equivalent over at least 12 weeks) and who failed to achieved a response or who subsequently relapse after Lenalidomide 4. Transfusion dependence (at least 3 RBC (Red Blood Cell) within a 16-week period and at least 2 transfusion episodes during this period) 5. Patient not eligible for another clinical trial 6. Adequate renal function defined by creatinine level less than 1.5 times the upper limit of normal and creatinine clearance ≥ 40mL/min (according to MDRD (Modification of Diet in Renal Disease) formula) 7. Adequate liver function defined by total bilirubin and transaminases less than 1.5 times the upper limit of normal 8. Patient not refractory to platelet transfusions 9. Written consent 10. Patient must understand and voluntarily sign informed consent form 11. Patient must be able to adhere to the visit schedule as outlined in the study and follow protocol requirements 12. Performance status 0-2 at the time of screening 13. A FCBP (female of childbearing potential) for this study was defined as a sexually mature woman who: (1) had not undergone a hysterectomy or bilateral oophorectomy; or (2) had not been naturally postmenopausal (amenorrhea following cancer therapy did not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months). A FCBP participating in the study must: * Have had 2 negative pregnancy tests as verified by the investigator prior to starting IP (unless the screening pregnancy test was done within 72 hours of Cycle 1 Day 1). She must have had agreed to ongoing a monthly pregnancy testing during the course of the study and after end of treatment. * If sexually active, agreed to have used, and been able to comply with, highly effective contraception\*\* without interruption, 5 weeks prior to starting treatment, during treatment (including dose interruptions), and for 24 weeks after discontinuation of treatment. * Highly effective contraception was defined in this protocol as the following (information also appeared in the Informed Consent Form): Hormonal contraception (eg, birth control pills, injection, implant, transdermal patch, vaginal ring), intrauterine device, tubal ligation (tying your tubes), or a partner with a vasectomy. 14. Male subjects must: Have agreed to use a condom, defined as a male latex condom or nonlatex condom NOT made out of natural (animal) membrane (eg, polyurethane), during sexual contact with a pregnant female or a FCBP while participating in the study, during dose interruptions, and for at least 24 weeks following treatment discontinuation, even if he had undergone a successful vasectomy. Exclusion criteria: Any patient meeting one of the following criteria cannot be included in the study: 1. Severe infection or any uncontrolled severe condition 2. Uncontrolled hypertension 3. Significant cardiac disease - NYHA (New York Heart Association) Class III or IV or having suffered a myocardial infarction in the last 6 months 4. QTcF (Fridericia's corrected QT interval) \> 460ms 5. Use of investigational agents within 30 days or any anticancer therapy (including IMiD (Immunomodulatory treatments)) within 2 weeks before the study entry with the exception of hydroxyurea. The patient must have recovered at least a grade 1 from all acute toxicity from any previous therapy. However, patients may have received Lenalidomide, hypomethylating agent, or anti-lymphocytic serum (ALS) (but not within 4 weeks before the study entry and, for ALS, within 16 weeks before the study entry). 6. Use of EPO within 4 weeks before the study entry 7. Active cancer or cancer during the year prior to trial entry other than basal cell carcinoma, or carcinoma in situ of the cervix or breast 8. Patient already enrolled in another therapeutic trial of an investigational drug 9. Known Human Immunodeficiency Virus infection or active hepatitis B or C 10. Women who are or could become pregnant or who are currently breastfeeding 11. Any medical or psychiatric contraindication that would prevent the patient from understanding and signing the informed consent form 12. Patient eligible for allogeneic stem cell transplantation 13. No affiliation to a health insurance system
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
3 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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CHU de Nice - Hôpital l'Archet - Service d'hématologie clinique
RECRUITINGNice, 06200, France
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Hôpital Saint Louis - Service Hématologie séniors
RECRUITINGParis, 75010, France
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Institut Gustave Roussy - Service d'hématologie
RECRUITINGVillejuif, 94805, France
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