Informations générales (source: ClinicalTrials.gov)
Phase I Study With Dose-escalation and Expansion Evaluating the Safety and Efficacy of Oral Arsenic (ATO) in Low-risk Myelodysplastic Syndromes Failing Erythropoiesis Stimulating Agents and Luspatercept (or Ineligible for the Latter)
Interventional
Phase 1
Groupe Francophone des Myelodysplasies (Voir sur ClinicalTrials)
juillet 2025
juillet 2027
01 août 2025
Phase I study with dose-escalation and expansion evaluating the safety and efficacy of
oral Arsenic (ATO) in low-risk Myelodysplastic Syndromes having failed to Erythropoiesis
Stimulating Agents and Luspatercept (or ineligible for the latter).
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| CLCC INSTITUT GUSTAVE ROUSSY | Jean-Baptiste MICOL | 17/04/2026 12:45:06 | Contacter | ||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| CHU de Nice - Hôpital l'Archet - Service d'hématologie clinique - 06200 - Nice - France | Thomas CLUZEAU, MD/PHD | Contact (sur clinicalTrials) | |||
| Hôpital Saint Louis - Service Hématologie séniors - 75010 - Paris - France | Pierre FENAUX, MD/PHD | Contact (sur clinicalTrials) | |||
Critères
Tous
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.
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.
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