Study of Association of Arsenic Trioxide (ATO) and Ascorbic Acid in Myelodysplastic Syndromes
Information source: Fondazione Italiana Sindromi Mielodisplastiche Onlus
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Myelodysplastic Syndromes
Intervention: ATO + Ascorbic acid (Drug)
Phase: Phase 2
Sponsored by: Fondazione Italiana Sindromi Mielodisplastiche Onlus
Official(s) and/or principal investigator(s):
Alessandro Levis, MD, Study Director, Affiliation: S.O.C. di Ematologia, Azienda Ospedaliera SS Antonio e Biagio, Alessandria. Via Venezia 18 - 15100 - Alessandria
This is a prospective, multicenter phase II trial designed to evaluate the safety and
activity of the combination of association of arsenic trioxide (ATO) and ascorbic acid in
patients with myelodysplastic syndromes
Official title: Phase II Multicenter Study of Association of Arsenic Trioxide (ATO) and Ascorbic Acid in Myelodysplastic Syndromes
Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: TO evaluate the erythroid response rate (major), according to the International Working Group (IWG) criteria (42) after four months of treatment with the association of ATO and ascorbic acid.
Secondary outcome: To evaluate platelets and granulocyte response according to the International Working Group (IWG) criteria (42) after four months of treatment with the association of ATO and ascorbic acid
Minimum age: 18 Years.
Maximum age: 80 Years.
1. Patients affected by myelodysplastic syndromes, entering in one of the following
1. Myelodysplastic syndromes independent of WHO diagnostic classification (43) and
IPSS prognostic score (2), when present at least one of the following
- 3q26 chromosome rearrangement.
- High EVI-1 transcript levels.
2. Myelodysplastic syndromes without excess of blasts (non-RAEB patients) at low or
intermediate-1 score risk according to the IPSS (2), as a second line treatment
option, after a failure to the first line treatment with erythropoietin +/-
G-CSF, immunosuppressive therapy, or other initial treatment modality.
3. Non RAEB patients at intermediate-2 or high risk score or RAEB patients at any
prognostic score, who are non candidate to treatment with conventional
2. Presence of one ore more cytopenias characterised by one ore more of the following
- Transfusions dependence.
- Hb< 11 gr/dl
- Platelet count < 50x109/L
- Absolute neutrophil count < .5x109/L.
3. ECOG Performance status ≤ 2.
4. Aged from 18 to 80.
5. Life expectancy > 4 months.
6. Creatinine level < 1. 5 mg/dl.
7. Liver function tests, including ASL-ALT-alkaline phosphatase lower than 3xULN
8. No previous treatment with chemotherapy, growth factors, cytokines or other
experimental treatment within 4 weeks of starting treatment.
9. No history of clinically significant cardiac disease, including congestive heart
10. Cytogenetic evaluation available.
11. Sending of both peripheral blood and bone marrow sample to the central laboratory for
EVI-1 rearrangement evaluation.
12. Written Informed consent.
1. Patients affected by myelodysplastic syndromes entering in categories other than
those foreseen by inclusion criteria point 1.
2. Absence of cytopenia defined as the contemporarily presence of all the following
conditions: a) no transfusion need; b) Hb > 11 gr/dl; c) platelet count > 50x109/L;
d) absolute neutrophil count > .5x109/L.
3. All patients that might be candidate to allogenic stem cell transplantation.
4. Patients that might be candidate to a first line immunosuppressive therapy.
5. ECOG Performance status > 2.
6. Age lower than 18 or higher then 80.
7. Life expectancy < 4 months.
8. Creatinine level > 1. 5 mg/dl.
9. Liver function tests, including ASL-ALT-alkaline phosphatase higher than 3xULN
10. Treatment with chemotherapy, growth factors, cytokines or other experimental
treatment within 4 weeks of starting treatment.
11. Clinically significant cardiac disease, including congestive heart failure, rhythm
abnormalities, QT time > 460m/s, or need of anti-arrhythmic drugs.
12. Concurrent co-morbid medical condition which might exclude administration of therapy,
as judged by individual investigator.
13. Absence of cytogenetic evaluation.
14. Participation at same time in another study in which investigational drugs are used.
15. Absence of written Informed consent.
Locations and Contacts
Ospedale SS Antonio, Biagio e Cesare Arrigo, Alessandria, Italy
Ospedale Cardinal Massaia, Asti, Italy
Spedali Civili, Brescia, Italy
Ospedale Maggiore, Chieri, Italy
Ospedale civico di Chivasso, Chivasso (TO), Italy
Ospedale Santa Croce e Carle, Cuneo, Italy
AOS San Gerardo de' Tintori, Monza, Italy
Università Avogadro Divisione di Ematologia, Novara, Italy
Ospedale San Luigi Gonzaga Divisione di Ematologia, Orbassano (TO), Italy
Azienda Ospedaliera Perugia, Perugia, Italy
Ospedale San Giovanbni Battista-Molinette, Torino, Italy
Ospedale San Giovanni Battista -Molinette, Torino, Italy
Ospedale San Bortolo, Vicenza, Italy
Starting date: September 2005
Last updated: June 27, 2011