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

NCT00804050 | Terminated Phase 3 DMC

• 2 other identifiers: EPO2006-AISSM042006-006482-16
• Study Type: interventional
• Target: 184
• Locations: 1 country
• Sites: 18

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

Conditions

Myelodysplastic Syndromes

Keywords

Myelodysplastic Syndromes; Erythropoietin; Acid 13-Cis-Retinoic; Dihydroxyvitamin D3; low or intermediate-1 IPSS; MDS low risk

Outcome Measures

Primary Outcomes (1)

• 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

  After 8 months

Secondary Outcomes (5)

• 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.

  8 months

• 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.

  20 mounths

• 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.

  20 mounths

• To evaluate the quality of life improvement due to therapy.

  8 mounths

• To evaluate the percentage of leukemic progression.

  20 mounths

Study Arms (2)

Infusion A: rEPO

EXPERIMENTAL

rEPO for 4 mounths consequently

Drug: infusion A: rEPO

Infusion B combined r-EPO

EXPERIMENTAL

rEPO in association with acid 13-cis-retinoic acid and Dihydroxyvitamin D3 for 4 mounths consequently

Drug: B Infusion rEPO combined with vitamins pills

Interventions

infusion A: rEPO DRUG

rEPO 40.000 unit/week for 4 mounths, then patients who have obtained an erythroid response, will continue for another 4 months with therapy rEPO. Patients who have not obtained an erythroid response, will increase the dose rEPO to 80,000 units per week for an additional 4 months.

Infusion A: rEPO

B Infusion rEPO combined with vitamins pills DRUG

rEPO 40.000 unit/week plus acid 13 cis-retinoic (20 mg/die) plus Dihydroxyvitamin D3 (1 μg os/die), for 4 mounths. Patients who have obtained an erythroid response will continue for another 4 months with the same therapy. Patients who have not obtained an erythroid will increase the dose of rEPO to 80,000 units per week for additional 4 months.Patients also will continue with the same doses of acid 13-cis-retinoic acid and Dihydroxyvitamin D3

Infusion B combined r-EPO

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age higher than 18;
• 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).
• Low or intermediate-1 IPSS (appendix).
• Hb \< 11g/dl.
• rEPO serum level \< 500mU/L.
• Women in menopause from at least one year.
• Informed consent

You may not qualify if:

• Myelodisplastic syndrome with excess of blasts (RAEB).
• IPSS score intermediate-2 or high (appendix).
• Renal failure with creatininemia value greater than 3 times the normal limit.
• 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.
• Presence of second tumor or other serious pathology with life expectancy lower than one year.
• Presence of neurologic or psychiatric pathologies that make the patient unreliable in the acquisition of drugs.
• Allergy/intolerance known to use drugs.
• Pregnant women.
• Women of childbearing age or in menopause from less than one year.
• Age \< 18 years old.
• HIV positive.

Sponsors & Collaborators

• Fondazione Italiana Sindromi Mielodisplastiche-ETS: lead
• Centro di Riferimento per l'Epidemiologia e la Prev. Oncologica Piemonte: collaborator

Study Sites (18)

Ospedale SS. Antonio, Biagio e Cesare Arrigo

Alessandria, Italy

Location

Ospedale degli Infermi

Biella, Italy

Location

Spedali civili

Brescia, Italy

Location

Ospedale Santo Spirito

Casale (AL), Italy

Location

Ospedale Maggiore

Chieri (TO), Italy

Location

Policlinico dell'Annunziata

Cosenza, Italy

Location

Ospedale Santa Croce e Carle

Cuneo, Italy

Location

Ospedale Santa Croce

Fano (PU), Italy

Location

Ospedale San Martino

Genova, Italy

Location

Ospedale civile

Ivrea (TO), Italy

Location

Ospedale San Gerardo

Monza (MI), Italy

Location

Ospedale Maggiore della Carità

Novara, Italy

Location

Ospedale Civile

Ovada (AL), Italy

Location

Istituto clinico Humanitas

Rozzano (MI), Italy

Location

Ospedale San Giovanni Battista Molinette

Torino, Italy

Location

Ospedale Cardinale Panico

Tricase (LE), Italy

Location

Ospedale Sant'Andrea

Vercelli, Italy

Location

Ospedale San Bortolo

Vicenza, Italy

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Dario Ferrero, MD

  University of Torino - Ospedale San Giovanni Battista

  STUDY DIRECTOR

• Alessandro Levis, MD

  Ospedale SS. Antonio, Biagio e Cesare Arrigo

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: December 5, 2008
• First Posted: December 8, 2008
• Study Start: March 1, 2007
• Primary Completion: March 1, 2009
• Study Completion: March 1, 2010
• Last Updated: June 28, 2011

Record last verified: 2011-06

Locations

IT (18)