Clinical trial NCT00804050: Multicentric Study Comparison Between Erythropoietin and Erythropoietin Associated to Differentiating Therapy With Acid 13-cis-retinoic and Dihydroxyvitamin D3 in Myelodysplastic Syndromes Without Excess of Blasts
Official title: Comparison Between Erythropoietin and Erythropoietin Associated to Differentiating Therapy With Acid 13-cis-retinoic and Dihydroxyvitamin D3 in Myelodysplastic Syndromes Without Excess of Blasts
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Myelodysplastic Syndromes
Keywords: Myelodysplastic Syndromes
Erythropoietin
Acid 13-Cis-Retinoic
Dihydroxyvitamin D3
low or intermediate-1 IPSS
MDS low risk
Lead sponsor: Fondazione Italiana Sindromi Mielodisplastiche-ETS
Sponsor class: OTHER
Collaborators: name: Centro di Riferimento per l'Epidemiologia e la Prev. Oncologica Piemonte
First posted: 2008-12-08
Enrollment: 184
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a prospective, randomized multicenter phase III clinical trial designed to evaluate the safety and activity of comparison between Erythropoietin and Erythropoietin Associated to Differentiating Therapy With Acid 13-Cis-Retinoic and Dihydroxyvitamin D3 in Myelodysplastic Syndromes Without Excess of Blasts


Eligibility criteria: Inclusion Criteria:

1. Age higher than 18;
2. Confirmed diagnosis by osseous biopsy and bone marrow cyto-morphologic counts of blast cells, of Myelodysplastic syndrome without excess of blasts: "refractary anemia", "refractary anemia with rings sideroblasts", "refractary citopenya with multilineage dysplasia", " refractary citopenya with multilineage dysplasia and rings sideroblasts" or "5q-syndrome" without excess of blasts based on WHO classification (appendix).
3. Low or intermediate-1 IPSS (appendix).
4. Hb \< 11g/dl.
5. rEPO serum level \< 500mU/L.
6. Women in menopause from at least one year.
7. Informed consent

Exclusion Criteria:

1. Myelodisplastic syndrome with excess of blasts (RAEB).
2. IPSS score intermediate-2 or high (appendix).
3. Forecasted allogeneic bone marrow transplant within 1 year after diagnosis(patients younger than 60 years, transfusion dependents or with serious leuko/thrombocytopenia and HLA compatible family donor).Considering the time needed to perform this procedure, the indication of a transplant from non-consanguineous donor has no contraindication to the inclusion in this protocol of the response to rEPO therapy ± differentiating therapy.
4. Renal failure with creatininemia value greater than 3 times the normal limit.
5. Chronic hepatophaty with bilirubinemia value greater than 3 times the normal limit and/or AST or ALT or ALP values greater than 5 times the normal limit.
6. Presence of second tumor or other serious pathology with life expectancy lower than one year.
7. Presence of neurologic or psychiatric pathologies that make the patient unreliable in the acquisition of drugs.
8. Allergy/intolerance known to use drugs.
9. Pregnant women.
10. Women of childbearing age or in menopause from less than one year.
11. Age \< 18 years old.
12. HIV positive.

Primary outcomes:
measure: study of comparison between standard theraphy rEPO (40.000unit/week) and association therapy between rEPO (40.000unit/week)plus Acid 13-Cis-Retinoic and Dihydroxyvitamin D3
timeFrame: After 8 months
Secondary outcomes:
measure: TO evaluate if,the patients without eritroyd response at the end of the 4° month of therapy, the increase of dose of rEPO to 80.000 U / week allows to get an increase of valued response at the end of the 8° month.
timeFrame: 8 months
measure: To evaluate if there is a difference of duration in the eritroyd response with standard therapy rEPO in comparison with association therapy rEPO plus Acid 13-Cis-Retinoic and Dihydroxyvitamin D3.
timeFrame: 20 mounths
measure: To evaluate the existing relationships among eritroyd response and clinical-biological parameters at baseline of anemia, subclass of MDS, silky dosing of the EPO etc.
timeFrame: 20 mounths
measure: To evaluate the quality of life improvement due to therapy.
timeFrame: 8 mounths
measure: To evaluate the percentage of leukemic progression.
timeFrame: 20 mounths
Other outcomes:
Locations:
city: Alessandria
country: Italy
facility: Ospedale SS. Antonio, Biagio e Cesare Arrigo
city: Biella
country: Italy
facility: Ospedale degli Infermi
city: Brescia
country: Italy
facility: Spedali civili
city: Casale (AL)
country: Italy
facility: Ospedale Santo Spirito
city: Chieri (TO)
country: Italy
facility: Ospedale Maggiore
city: Cosenza
country: Italy
facility: Policlinico dell'Annunziata
city: Cuneo
country: Italy
facility: Ospedale Santa Croce e Carle
city: Fano (PU)
country: Italy
facility: Ospedale Santa Croce
city: Genova
country: Italy
facility: Ospedale San Martino
city: Ivrea (TO)
country: Italy
facility: Ospedale civile
city: Monza (MI)
country: Italy
facility: Ospedale San Gerardo
city: Novara
country: Italy
facility: Ospedale Maggiore della Carità
city: Ovada (AL)
country: Italy
facility: Ospedale Civile
city: Rozzano (MI)
country: Italy
facility: Istituto clinico Humanitas
city: Torino
country: Italy
facility: Ospedale San Giovanni Battista Molinette
city: Tricase (LE)
country: Italy
facility: Ospedale Cardinale Panico
city: Vercelli
country: Italy
facility: Ospedale Sant'Andrea
city: Vicenza
country: Italy
facility: Ospedale San Bortolo
Study officials:
affiliation: University of Torino - Ospedale San Giovanni Battista
name: Dario Ferrero, MD
role: STUDY_DIRECTOR
affiliation: Ospedale SS. Antonio, Biagio e Cesare Arrigo
name: Alessandro Levis, MD
role: STUDY_DIRECTOR
References: