Myelodysplastic SyndromesPhase 3NCT00804050
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
A Phase 3 study testing infusion A: rEPO and B Infusion rEPO combined with vitamins pills in people with Myelodysplastic Syndromes, run by Fondazione Italiana Sindromi Mielodisplastiche-ETS at 18 sites in Italy.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- infusion A: rEPO and B Infusion rEPO combined with vitamins pills
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 184 participants
- Where
- 18 sitesItaly
- Study dates
- Mar 2007to Mar 2010
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Show all 12Show fewer
- 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.
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
Italy18 sites
Ospedale SS. Antonio, Biagio e Cesare Arrigo
Alessandria
Ospedale degli Infermi
Biella
Spedali civili
Brescia
Ospedale Santo Spirito
Casale (AL)
Ospedale Maggiore
Chieri (TO)
Policlinico dell'Annunziata
Cosenza
Ospedale Santa Croce e Carle
Cuneo
Ospedale Santa Croce
Fano (PU)
Ospedale San Martino
Genova
Ospedale civile
Ivrea (TO)
Ospedale San Gerardo
Monza (MI)
Ospedale Maggiore della Carità
Novara
Ospedale Civile
Ovada (AL)
Istituto clinico Humanitas
Rozzano (MI)
Ospedale San Giovanni Battista Molinette
Torino
Ospedale Cardinale Panico
Tricase (LE)
Ospedale Sant'Andrea
Vercelli
Ospedale San Bortolo
Vicenza
About this study
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
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
- Being tested
- infusion A: rEPO, B Infusion rEPO combined with vitamins pills
- Keywords
- Myelodysplastic Syndromes, Erythropoietin, Acid 13-Cis-Retinoic, Dihydroxyvitamin D3, low or intermediate-1 IPSS, MDS low risk
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
infusion A: rEPODrug
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.
B Infusion rEPO combined with vitamins pillsDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Infusion A: rEPO | Experimental | rEPO for 4 mounths consequently Receives: 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 Receives: Drug: B Infusion rEPO combined with vitamins pills |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 20 mounths
To evaluate the quality of life improvement due to therapy.
Measured: 8 mounths
To evaluate the percentage of leukemic progression.
Measured: 20 mounths
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2008 (estimated)
- Study startMar 2007
- Primary completionMar 2009
- Study completionMar 2010
Why it stopped: Difficulties of enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 184 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione Italiana Sindromi Mielodisplastiche-ETS
- Sponsor type
- University, hospital or other
- Collaborators
- Centro di Riferimento per l'Epidemiologia e la Prev. Oncologica Piemonte
- Investigators
- Dario Ferrero, MD, Study director, University of Torino - Ospedale San Giovanni BattistaAlessandro Levis, MD, Study director, Ospedale SS. Antonio, Biagio e Cesare Arrigo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00804050
- Sponsor's protocol ID
- EPO2006-AISSM04
- Other IDs
- 2006-006482-16
- Status last verified
- Jun 2011
- First submitted
- 5 Dec 2008
- Last updated
- 28 Jun 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Jun 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00804050
- Acronym / sponsor ID
- EPO2006-AISSM04
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Myelodysplastic Syndromes, Erythropoietin, Acid 13-Cis-Retinoic, Dihydroxyvitamin D3, low or intermediate-1 IPSS, MDS low risk
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Difficulties of enrollment
- Enrolment
- 184 participants (estimated)
- Lead sponsor
- Fondazione Italiana Sindromi Mielodisplastiche-ETSUniversity, hospital or other
- Collaborators
- Centro di Riferimento per l'Epidemiologia e la Prev. Oncologica Piemonte
- Study start
- Mar 2007
- Primary completion
- Mar 2009 (actual)
- Study completion
- Mar 2010 (actual)
- First posted
- 8 Dec 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Infusion A: rEPO | Experimental | Drug: infusion A: rEPOrEPO for 4 mounths consequently |
| Infusion B combined r-EPO | Experimental | Drug: B Infusion rEPO combined with vitamins pillsrEPO in association with acid 13-cis-retinoic acid and Dihydroxyvitamin D3 for 4 mounths consequently |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| infusion A: rEPOrEPO 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. | Drug | Infusion A: rEPO |
| B Infusion rEPO combined with vitamins pillsrEPO 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 | Drug | Infusion B combined r-EPO |
Primary outcome
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
5 secondary outcomes
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
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
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
To evaluate the quality of life improvement due to therapy.
Time frame: 8 mounths
To evaluate the percentage of leukemic progression.
Time frame: 20 mounths
Results and safety
The study was stopped early (reason given: Difficulties of enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jun 2011.
Related trials for Myelodysplastic Syndromes
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dario Ferrero, MD study director
University of Torino - Ospedale San Giovanni Battista
Alessandro Levis, MD study director
Ospedale SS. Antonio, Biagio e Cesare Arrigo
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing infusion A: rEPO and B Infusion rEPO combined with vitamins pills in people with Myelodysplastic Syndromes, run by Fondazione Italiana Sindromi Mielodisplastiche-ETS at 18 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 28 Jun 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Difficulties of enrollment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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 (time frame: After 8 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jun 2011.