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5-azacytidine Treatment Versus 5-azacytidine Followed by Allogeneic Stem Cell Transplantation in Elderly Patients With Myelodysplastic Syndrome (MDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01404741
Recruitment Status : Unknown
Verified May 2017 by Universitätsklinikum Hamburg-Eppendorf.
Recruitment status was:  Active, not recruiting
First Posted : July 28, 2011
Last Update Posted : May 31, 2017
Sponsor:
Information provided by (Responsible Party):
Universitätsklinikum Hamburg-Eppendorf

Study Description
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Brief Summary:
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. 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 or disease Intervention/treatment Phase
Myelodysplastic Syndrome Chronic Myelomonocytic Leukemia Procedure: allogeneic stem cell transplantation Procedure: 5-azacytidine until progress Phase 2

Detailed Description:
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).
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 191 participants
Allocation: Non-Randomized
Intervention Model: Factorial Assignment
Masking: None (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
Actual Study Start Date : July 2011
Estimated Primary Completion Date : May 2020
Estimated Study Completion Date : May 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Azacitidine

Arms and Interventions
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Arm Intervention/treatment
Active Comparator: 5-azacytidine treatment until progress
5-azacytidine 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


Outcome Measures
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Primary Outcome Measures :
  1. overall survival [ Time Frame: three years ]
    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 :
  1. response [ Time Frame: three years ]

    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


  2. event-free survival [ Time Frame: three years ]

    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 period


  3. overall survival [ Time Frame: three years ]

    Comparison of overall survival between both arms (230 pat.).

    - evaluation of survival status (alive or death/date of death) in the whole study period


  4. impact of Comorbidity-index on outcome [ Time Frame: three years ]

    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)

  5. Treatment-related mortality [ Time Frame: three years ]

    compare treatment related mortality in both arms (230 pat.):

    - death according to treatment in both arms


  6. Evaluation of toxicity [ Time Frame: three years ]

    the evaluation of toxicity will be performed according to the reporting guidelines as per NCI CTCAE in the whole study period:

    • 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

  7. quality of life [ Time Frame: three years ]
    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


Eligibility Criteria
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Ages Eligible for Study:   55 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01404741


Locations
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Germany
Charité Campus Benjamin Franklin
Berlin, Germany
Uniklinikum Bonn
Bonn, Germany
Universitätsklinikum Dresden
Dresden, Germany, 01307
Universitätsklinikum Düsseldorf
Düsseldorf, Germany
Universitätsklinikum Essen
Essen, Germany, 45122
Universitätsklinikum Essen
Essen, Germany
Klinikum der Johann Wolfgang Goethe-Universität
Frankfurt am Main, Germany
Universitätsklinikum Göttingen
Göttingen, Germany
University Medical Center Hamburg-Eppendorf
Hamburg, Germany
Medizinische Hochschule Hannover
Hannover, Germany
Universität zu Köln
Köln, Germany
Universitätsklinikum Mannheim
Mannheim, Germany
Klinikum rechts der Isar
München, Germany
Universitätsklinikum Münster
Münster, Germany
Klinikum Nürnberg
Nürnberg, Germany
Medizinische Universitätsklinik II
Tübingen, Germany
Universitätsklinikum Ulm
Ulm, Germany
Sponsors and Collaborators
Universitätsklinikum Hamburg-Eppendorf
Investigators
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Principal Investigator: Nicolaus Kroeger, Prof. University Medical Centre Hamburg-Eppendorf, Stem-Cell-Transplantation
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Universitätsklinikum Hamburg-Eppendorf
ClinicalTrials.gov Identifier: NCT01404741    
Other Study ID Numbers: VidazaAlloStudy
First Posted: July 28, 2011    Key Record Dates
Last Update Posted: May 31, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Universitätsklinikum Hamburg-Eppendorf:
MDS
CMML
allogeneic stem cell transplantation
5-azacytidine
Additional relevant MeSH terms:
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Preleukemia
Leukemia, Myelomonocytic, Chronic
Leukemia, Myelomonocytic, Juvenile
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Leukemia, Myeloid
 Leukemia
Neoplasms by Histologic Type
Myelodysplastic-Myeloproliferative Diseases
Chronic Disease
Disease Attributes
Azacitidine
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors