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Myelodysplastic SyndromePhase 2NCT01404741

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

A Phase 2 study testing allogeneic stem cell transplantation and 5-azacytidine until progress in people with Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia, run by Universitätsklinikum Hamburg-Eppendorf at 17 sites in Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 55 to 70 years
What is being tested?
allogeneic stem cell transplantation and 5-azacytidine until progress
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
191 participants
Where
17 sitesGermany
Study dates
Jul 2011to Jul 2021
Phase
Phase 2
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 55 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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 %)

Who cannot join

  • 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
Show all 13Show fewer
  • 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

Trial locations and contacts

17 sites in 1 country.

17 sites in 1 country

Germany17 sites

  • Charité Campus Benjamin Franklin

    Berlin

  • Uniklinikum Bonn

    Bonn

  • Universität zu Köln

    Cologne

  • Universitätsklinikum Dresden

    Dresden, 01307

  • Universitätsklinikum Düsseldorf

    Düsseldorf

  • Universitätsklinikum Essen

    Essen, 45122

  • Universitätsklinikum Essen

    Essen

  • Klinikum der Johann Wolfgang Goethe-Universität

    Frankfurt am Main

  • Universitätsklinikum Göttingen

    Göttingen

  • University Medical Center Hamburg-Eppendorf

    Hamburg

  • Medizinische Hochschule Hannover

    Hanover

  • Universitätsklinikum Mannheim

    Mannheim

  • Klinikum rechts der Isar

    München

  • Universitätsklinikum Münster

    Münster

  • Klinikum Nürnberg

    Nuremberg

  • Medizinische Universitätsklinik II

    Tübingen

  • Universitätsklinikum Ulm

    Ulm

Other trials in Germany

About this study

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

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).

Read the 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).

Being tested
allogeneic stem cell transplantation, 5-azacytidine until progress
Keywords
MDS, CMML, allogeneic stem cell transplantation, 5-azacytidine
Medical subject headings
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • allogeneic stem cell transplantationProcedure or surgery

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

  • 5-azacytidine until progressProcedure or surgery

    if no donor available 5-azacytidine until progress or toxicities

Study groups in NCT01404741
Study groupRoleWhat they receive
5-azacytidine treatment until progressActive comparator5-azacytidine until progress
Receives: Procedure: 5-azacytidine until progress
allogeneic stem cell transplantationExperimentalafter 4 cycles 5-azacytidine and if donor available: allogeneic stem cell transplantation after reduced intensity conditioning
Receives: Procedure: allogeneic stem cell transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. overall survival

    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

    Measured: three years

Secondary outcomes (7)

  1. response

    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

    Measured: three years

  2. event-free survival

    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

    Measured: three years

  3. overall survival

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

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

    Measured: three years

  4. impact of Comorbidity-index on outcome

    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)

    Measured: three years

  5. Treatment-related mortality

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

    • death according to treatment in both arms

    Measured: three years

  6. Evaluation of toxicity

    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

    Measured: three years

  7. quality of life

    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

    Measured: three years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Jul 2011 (estimated)
  2. Study startJul 2011
  3. Primary completionMay 2020
  4. Study completionJul 2021

Resources

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Factorial
Masking
None (open label)
Enrolment
191 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01404741
Sponsor's protocol ID
VidazaAlloStudy
Status last verified
Oct 2023
First submitted
30 Jun 2011
Last updated
27 Oct 2023
Results posted
No
Data monitoring committee
No
Individual data sharing
Undecided

Publications

  • Kroger N, Sockel K, Wolschke C, Bethge W, Schlenk RF, Wolf D, Stadler M, Kobbe G, Wulf G, Bug G, Schafer-Eckart K, Scheid C, Nolte F, Kronke J, Stelljes M, Beelen D, Heinzelmann M, Haase D, Buchner H, Bleckert G, Giagounidis A, Platzbecker U. Comparison Between 5-Azacytidine Treatment and Allogeneic Stem-Cell Transplantation in Elderly Patients With Advanced MDS According to Donor Availability (VidazaAllo Study). J Clin Oncol. 2021 Oct 20;39(30):3318-3327. doi: 10.1200/JCO.20.02724. Epub 2021 Jul 20.

    PubMed 34283629

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing allogeneic stem cell transplantation and 5-azacytidine until progress in people with Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia, run by Universitätsklinikum Hamburg-Eppendorf at 17 sites in Germany.

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 13 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: finished, as of the sponsor's last update on 27 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 17 sites in Germany. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

overall survival (time frame: three years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01404741, which also lists 1 linked publication.

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 27 Oct 2023.

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