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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

Primary Purpose

Myelodysplastic Syndromes

Status
Terminated
Phase
Phase 3
Locations
Italy
Study Type
Interventional
Intervention
infusion A: rEPO
B Infusion rEPO combined with vitamins pills
Sponsored by
Fondazione Italiana Sindromi Mielodisplastiche-ETS
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring Myelodysplastic Syndromes, Erythropoietin, Acid 13-Cis-Retinoic, Dihydroxyvitamin D3, low or intermediate-1 IPSS, MDS low risk

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Ospedale SS. Antonio, Biagio e Cesare Arrigo
  • Ospedale degli Infermi
  • Spedali civili
  • Ospedale Santo Spirito
  • Ospedale Maggiore
  • Policlinico dell'Annunziata
  • Ospedale Santa Croce e Carle
  • Ospedale Santa Croce
  • Ospedale San Martino
  • Ospedale civile
  • Ospedale San Gerardo
  • Ospedale Maggiore della Carità
  • Ospedale Civile
  • Istituto clinico Humanitas
  • Ospedale San Giovanni Battista Molinette
  • Ospedale Cardinale Panico
  • Ospedale Sant'Andrea
  • Ospedale San Bortolo

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Infusion A: rEPO

Infusion B combined r-EPO

Arm Description

rEPO for 4 mounths consequently

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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.
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.
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.
To evaluate the quality of life improvement due to therapy.
To evaluate the percentage of leukemic progression.

Full Information

First Posted
December 5, 2008
Last Updated
June 27, 2011
Sponsor
Fondazione Italiana Sindromi Mielodisplastiche-ETS
Collaborators
Centro di Riferimento per l'Epidemiologia e la Prev. Oncologica Piemonte
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1. Study Identification

Unique Protocol Identification Number
NCT00804050
Brief Title
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

2. Study Status

Record Verification Date
June 2011
Overall Recruitment Status
Terminated
Why Stopped
Difficulties of enrollment
Study Start Date
March 2007 (undefined)
Primary Completion Date
March 2009 (Actual)
Study Completion Date
March 2010 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Fondazione Italiana Sindromi Mielodisplastiche-ETS
Collaborators
Centro di Riferimento per l'Epidemiologia e la Prev. Oncologica Piemonte

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes
Keywords
Myelodysplastic Syndromes, Erythropoietin, Acid 13-Cis-Retinoic, Dihydroxyvitamin D3, low or intermediate-1 IPSS, MDS low risk

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
184 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Infusion A: rEPO
Arm Type
Experimental
Arm Description
rEPO for 4 mounths consequently
Arm Title
Infusion B combined r-EPO
Arm Type
Experimental
Arm Description
rEPO in association with acid 13-cis-retinoic acid and Dihydroxyvitamin D3 for 4 mounths consequently
Intervention Type
Drug
Intervention Name(s)
infusion A: rEPO
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
B Infusion rEPO combined with vitamins pills
Intervention Description
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
Primary Outcome Measure Information:
Title
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
Time Frame
After 8 months
Secondary Outcome Measure Information:
Title
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.
Time Frame
8 months
Title
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.
Time Frame
20 mounths
Title
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.
Time Frame
20 mounths
Title
To evaluate the quality of life improvement due to therapy.
Time Frame
8 mounths
Title
To evaluate the percentage of leukemic progression.
Time Frame
20 mounths

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: Myelodisplastic syndrome with excess of blasts (RAEB). IPSS score intermediate-2 or high (appendix). 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. 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dario Ferrero, MD
Organizational Affiliation
University of Torino - Ospedale San Giovanni Battista
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Alessandro Levis, MD
Organizational Affiliation
Ospedale SS. Antonio, Biagio e Cesare Arrigo
Official's Role
Study Director
Facility Information:
Facility Name
Ospedale SS. Antonio, Biagio e Cesare Arrigo
City
Alessandria
Country
Italy
Facility Name
Ospedale degli Infermi
City
Biella
Country
Italy
Facility Name
Spedali civili
City
Brescia
Country
Italy
Facility Name
Ospedale Santo Spirito
City
Casale (AL)
Country
Italy
Facility Name
Ospedale Maggiore
City
Chieri (TO)
Country
Italy
Facility Name
Policlinico dell'Annunziata
City
Cosenza
Country
Italy
Facility Name
Ospedale Santa Croce e Carle
City
Cuneo
Country
Italy
Facility Name
Ospedale Santa Croce
City
Fano (PU)
Country
Italy
Facility Name
Ospedale San Martino
City
Genova
Country
Italy
Facility Name
Ospedale civile
City
Ivrea (TO)
Country
Italy
Facility Name
Ospedale San Gerardo
City
Monza (MI)
Country
Italy
Facility Name
Ospedale Maggiore della Carità
City
Novara
Country
Italy
Facility Name
Ospedale Civile
City
Ovada (AL)
Country
Italy
Facility Name
Istituto clinico Humanitas
City
Rozzano (MI)
Country
Italy
Facility Name
Ospedale San Giovanni Battista Molinette
City
Torino
Country
Italy
Facility Name
Ospedale Cardinale Panico
City
Tricase (LE)
Country
Italy
Facility Name
Ospedale Sant'Andrea
City
Vercelli
Country
Italy
Facility Name
Ospedale San Bortolo
City
Vicenza
Country
Italy

12. IPD Sharing Statement

Learn more about this trial

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

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