5-azacytidine Treatment Versus 5-azacytidine Followed by Allogeneic Stem Cell Transplantation in Elderly Patients With Myelodysplastic Syndrome (MDS)
This study is currently recruiting participants.
Verified July 2011 by Universitätsklinikum Hamburg-Eppendorf
Sponsor:
Universitätsklinikum Hamburg-Eppendorf
Information provided by:
Universitätsklinikum Hamburg-Eppendorf
ClinicalTrials.gov Identifier:
NCT01404741
First received: June 30, 2011
Last updated: July 27, 2011
Last verified: July 2011
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Purpose
5-azacytidine treatment prolongs survival in patients with myelodysplastic syndrome (MDS), but does not cure the disease. Allogeneic stem cell transplantation is a curative treatment option but is associated with a high risk treatment-related morbidity and mortality. Dose-reduced conditioning prior transplantation allows also treatment of elderly patients with MDS. In the current trial allogeneic stem cell transplantation will be compared to 5-azacytidine only treatment according to donor availability in elderly patients with MDS (55-70 years).
| Condition | Intervention | Phase |
|---|---|---|
|
Myelodysplastic Syndrome Chronic Myelomonocytic Leukemia |
Procedure: allogeneic stem cell transplantation Procedure: 5-azacytidine until progress |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Comparison Between 5-azacytidine Treatment and 5-azacytidine Followed by Allogeneic Stem Cell Transplantation in Elderly Patients With Advanced MDS According to Donor Availability |
Resource links provided by NLM:
Further study details as provided by Universitätsklinikum Hamburg-Eppendorf:
Primary Outcome Measures:
- overall survival [ Time Frame: three years ] [ Designated as safety issue: No ]compare to overall survival of patients who receive after 4 cycles of 5-azacytidine either allogeneic stem cell transplantation or continuous 5-azacytidine if no compatible donor is available overall 230 patients
Secondary Outcome Measures:
- response [ Time Frame: three years ] [ Designated as safety issue: No ]
Comparison of response according to International Working Group Response Criteria between both arms:
- Examinations of bone marrow (count of blasts) and peripheral blood (hematological improvement)after schedule of study assessments (after cycle 4 in both arms, after cycle 8 and after months 12-24-36 in the 5-azacytidine treatment and on day 100, day 180, months 12-24-36 after allogeneic stem cell transplantation
- event-free survival [ Time Frame: three years ] [ Designated as safety issue: No ]
comparison of event free survival in both arms (230 pat.):
- evaluation of survival status (relapse, date of relapse, alive or death) in the whole study periode
- overall survival [ Time Frame: three years ] [ Designated as safety issue: No ]
Comparison of overall survival between both arms (230 pat.).
- evaluation of survival status (alive or death/date of death) in the whole study periode
- impact of Comorbidity-index on outcome [ Time Frame: three years ] [ Designated as safety issue: No ]
impact of comorbidity-index on outcome after study entry and prior to allogeneic stem cell transplantation (according definitions and weighted scores of comorbidities by Sorror et al):
- physical examination
- laboratory values(creatinine,Alt, AST, bilirubin, etc.)
- apparative diagnostics (echo,lufu,ECG)
- Treatment-related mortality [ Time Frame: three years ] [ Designated as safety issue: No ]
compare treatment related mortality in both arms (230 pat.):
- death according to treatment in both arms
- Evaluation of toxicity [ Time Frame: three years ] [ Designated as safety issue: Yes ]
the evaluation of toxicity will be performed according to the reporting guidelines as per NCI CTCAE in the whole study periode:
- adverse events grade 3 and 4
- cytopenia grade 3 and 4 only be reported as AE which are judged by the investigator as clinically relevant
- quality of life [ Time Frame: three years ] [ Designated as safety issue: No ]Comparison of quality of life between both arms with the quality of life core questionnaire QLQ-C30 and the treatment specific high-dose chemotherapy module QLQ HD-C29 to assess the quality of life of cancer patient. The questionnaire has to be answered after the fourth cycle, 6 months, 1 year, 2 years and 3 years after both treatment arms
| Estimated Enrollment: | 230 |
| Study Start Date: | June 2011 |
| Estimated Study Completion Date: | June 2017 |
| Estimated Primary Completion Date: | June 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Active Comparator: 5-azacytidine treatment until progress |
Procedure: 5-azacytidine until progress
if no donor available 5-azacytidine until progress or toxicities
|
|
Experimental: allogeneic stem cell transplantation
after 4 cycles 5-azacytidine and if donor available: allogeneic stem cell transplantation after reduced intensity conditioning
|
Procedure: allogeneic stem cell transplantation
donor available, after 4 cycles 5-azacytidine allogeneic stem cell transplantation after reduced conditioning
|
Eligibility| Ages Eligible for Study: | 55 Years to 70 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Patients with proven de novo or therapy-related MDS / CMML (WBC <13 GPT/l)according to FAB and risk profile according to IPSS: intermediate II- risk or high-risk or intermediate I with high-risk cytogenetic (according to IPSS, taking into account that IPSS, however, was not validated for t- MDS), patients with secondary AML (according to WHO) and blasts ≤ 30 % (= RAEB-t according to FAB)
- Previously untreated or maximal 1 cycle of 5-azacytidine (Vidaza®)
- Male or Female; Age 55 - 70 years
- Understand and voluntarily sign an informed consent form
- ECOG performance status of ≤ 2 at study entry
- Adequate renal and liver function: creatinine and bilirubin < 3 x the upper limit of normal
- Sufficient cardiac function (ejection fraction > 30 %)
Exclusion Criteria:
- Blasts > 30 % in bone marrow at time of diagnosis
- Central nervous involvement
- Severe irreversible renal, hepatic, pulmonary or cardiac disease, such as
- Total bilirubin, SGPT or SGOT ≥ 3 times upper the normal level
- Left ventricular ejection fraction < 30 %
- Creatinine clearance < 30 ml/min
- DLCO < 35 % and/or receiving supplementary continuous oxygen
- Pregnant or breastfeeding female subject
- Patients with a life-expectancy of less than six months because of another debilitating disease
- Serious psychiatric or psychological disorders
- Uncontrolled invasive fungal infection at time of registration
- Known positive for HIV or acute infectious hepatitis, type A, B or C
- Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment until the end of the study
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01404741
Contacts
| Contact: Nicolaus Kroeger, Prof. | +49-40-7410-55864 | nkroeger@uke.uni-hamburg.de |
| Contact: Marion Heinzelmann | +49-40-7410-54188 | mheinzel@uke.uni-hamburg.de |
Locations
| Germany | |
| Charité Campus Benjamin Franklin | Not yet recruiting |
| Berlin, Germany | |
| Contact: Lutz Uharek, Prof. Dr. | |
| Principal Investigator: Lutz Uharek, Prof. Dr. | |
| Uniklinikum Bonn | Not yet recruiting |
| Bonn, Germany | |
| Contact: Marie v. Lilienfeld-Toal, PD Dr. | |
| Principal Investigator: Marie v.Lilienfeld-Toal, PD Dr. | |
| Universitätsklinikum Dresden | Recruiting |
| Dresden, Germany, 01307 | |
| Contact: Uwe Platzbecker, PD Dr. | |
| Principal Investigator: Uwe Platzbecker, PD Dr. | |
| Universitätsklinikum Düsseldorf | Not yet recruiting |
| Düsseldorf, Germany | |
| Contact: Guido Kobbe, PD Dr. | |
| Principal Investigator: Guido Kobbe, PD Dr. | |
| Universitätsklinikum Essen | Not yet recruiting |
| Essen, Germany, 45122 | |
| Contact: Dietrich W. Beelen, Prof. Dr. | |
| Principal Investigator: Dietrich W. Beelen, Prof. Dr. | |
| Universitätsklinikum Essen | Not yet recruiting |
| Essen, Germany | |
| Contact: Richard Noppeney, Dr. | |
| Principal Investigator: Richard Noppeney, Dr. | |
| Klinikum der Johann Wolfgang Goethe-Universität | Not yet recruiting |
| Frankfurt am Main, Germany | |
| Contact: Gesine Bug, Dr. | |
| Principal Investigator: Gesine Bug, Dr. | |
| Universitätsklinikum Göttingen | Not yet recruiting |
| Göttingen, Germany | |
| Contact: Gerald Wulf, Prof. Dr. | |
| Principal Investigator: Gerald Wulf, Prof. Dr. | |
| University Medical Center Hamburg-Eppendorf | Recruiting |
| Hamburg, Germany | |
| Contact: Nicolaus Kroeger, Prof. +49-40-7410-55864 nkroeger@uke.uni-hamburg.de | |
| Contact: Marion Heinzelmann +49-40-7410-54188 mheinzel@uke.uni-hamburg.de | |
| Principal Investigator: Nicolaus Kroeger, Prof. | |
| Medizinische Hochschule Hannover | Not yet recruiting |
| Hannover, Germany | |
| Contact: Michael Stadler, Dr.Dr. | |
| Principal Investigator: Michael Stadler, Dr.Dr. | |
| Universität zu Köln | Not yet recruiting |
| Köln, Germany | |
| Contact: Christof Scheid, PD Dr. | |
| Principal Investigator: Christof Scheid, PD Dr. | |
| Universitätsklinikum Mannheim | Not yet recruiting |
| Mannheim, Germany | |
| Contact: Stefan A. Klein, PD Dr. | |
| Principal Investigator: Stefan A. Klein, PD Dr. | |
| Klinikum rechts der Isar | Not yet recruiting |
| München, Germany | |
| Contact: Helge Menzel, Dr | |
| Principal Investigator: Helge Menzel, Dr. | |
| Universitätsklinikum Münster | Not yet recruiting |
| Münster, Germany | |
| Contact: Matthias Stelljes, PD Dr. | |
| Principal Investigator: Matthias Stelljes, PD Dr. | |
| Klinikum Nürnberg | Not yet recruiting |
| Nürnberg, Germany | |
| Contact: Kerstin Schäfer-Eckart, Dr. | |
| Principal Investigator: Kerstin Schäfer-Eckart, Dr. | |
| Medizinische Universitätsklinik II | Not yet recruiting |
| Tübingen, Germany | |
| Contact: Wolfgang Bethge, PD Dr. | |
| Principal Investigator: Wolfgang Betghe, PD Dr. | |
| Universitätsklinikum Ulm | Not yet recruiting |
| Ulm, Germany | |
| Contact: Richard F. Schlenk, PD Dr. | |
| Principal Investigator: Richard F. Schlenk, PD Dr. | |
Sponsors and Collaborators
Universitätsklinikum Hamburg-Eppendorf
Investigators
| Principal Investigator: | Nicolaus Kroeger, Prof. | University Medical Centre Hamburg-Eppendorf, Stem-Cell-Transplantation |
More Information
No publications provided
| Responsible Party: | Prof. N. Kroeger, University Medical Center Hamburg-Eppendorf |
| ClinicalTrials.gov Identifier: | NCT01404741 History of Changes |
| Other Study ID Numbers: | VidazaAlloStudy |
| Study First Received: | June 30, 2011 |
| Last Updated: | July 27, 2011 |
| Health Authority: | Germany: Federal Institute for Drugs and Medical Devices Germany: Paul-Ehrlich-Institut |
Keywords provided by Universitätsklinikum Hamburg-Eppendorf:
|
MDS CMML allogeneic stem cell transplantation 5-azacytidine |
Additional relevant MeSH terms:
|
Leukemia Leukemia, Myelomonocytic, Chronic Myelodysplastic Syndromes Preleukemia Leukemia, Myelomonocytic, Acute Neoplasms by Histologic Type Neoplasms Leukemia, Myeloid Myelodysplastic-Myeloproliferative Diseases Bone Marrow Diseases |
Hematologic Diseases Precancerous Conditions Azacitidine Antimetabolites, Antineoplastic Antimetabolites Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Antineoplastic Agents Therapeutic Uses Enzyme Inhibitors |
ClinicalTrials.gov processed this record on May 22, 2013