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NCT Number: NCT01404741

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

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).

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Key information

Conditions

Myelodysplastic Syndrome Bone Marrow Diseases Chronic Disease Chronic Myelomonocytic Leukemia Disease Attributes Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myelomonocytic, Chronic Myelodysplastic Syndromes Myelodysplastic-Myeloproliferative Diseases Neoplasms Neoplasms by Histologic Type Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

55 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Charité Campus Benjamin Franklin, Berlin, Germany

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About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

allogeneic stem cell transplantation

Procedure

donor available, after 4 cycles 5-azacytidine allogeneic stem cell transplantation after reduced conditioning

5-azacytidine until progress

Procedure

if no donor available 5-azacytidine until progress or toxicities

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Universitätsklinikum Hamburg-Eppendorf

Other

Registry information

Official study 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

Important dates

Study start
2011
Primary completion
2020
Study completion
2021
First posted
Jul 28, 2011
Registry last updated
Oct 27, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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