Comparison between 5 – azacytidine treatment and 5 – azacytidine followed by allogeneic stem cell transplantation in elderly patients with MDS according to donor availability

Overview
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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).

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).

SEEK ID: https://cgnbonn.fdm.digital-medicine.org/projects/12

Public web page: Not specified

Organisms: No Organisms specified

NFDI4Health PIs: Cristoph Scheid

Trial Project start date: 29th Oct 2011

Trial Project end date: 30th Nov 2016

Extended Metadata (Nfdi4Health MDS 3.3)

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Id of the project(*) : 29546

Resource classification(*)

Type of the resource : Study

Title(s)/name(s) of the Project

Title/name : Comparison between 5 – azacytidine treatment and 5 – azacytidine followed by allogeneic stem cell transplantation in elderly patients with MDS according to donor availability
Language of the title/name : English

Acronym(s) of the Project

Acronym : VidazaAlloStudy
Language of the acronym : English

Description(s) of the Project

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).
Language of the description : English

Keyword(s) describing the Project

Keyword(*) : MDS

Keyword(*) : CMML

Keyword(*) : allogeneic stem cell transplantation

Keyword(*) : 5-azacytidine

Web page of the project : https://clinicaltrials.gov/study/NCT01404741

Contributor(s) of the Project

Is it a personal or organisational contribution?(*) : Personal
Details about the contributing organisation(s)/institution(s)/group(s)
Contributor type(*) : Not specified
Funding identifier(s) : Not specified
Name of the organisation/institution/group(*) : Not specified
Details about the contributing person(s)
Contributor type(*) : Principal investigator
Given name(*) : Cristoph
Family name(s)(*) : Scheid
Digital identifier(s)
Identifier(*) : 0009-0007-6539-226X
Scheme(*) : ORCID
Email address : christoph.scheid@uk-koeln.de
Phone number : Not specified
Organisation(s) associated with the contributor
Name(*) : Klinik I für innere Medizin, University Hospital Cologne
Address : Kerpener Straße 62 50937 Cologne, Germany
Web page : https://innere1.uk-koeln.de/
Digital identifier(s)
Identifier(*) : 05mxhda18
Scheme(*) : ROR

Is it a personal or organisational contribution?(*) : Personal
Details about the contributing organisation(s)/institution(s)/group(s)
Contributor type(*) : Not specified
Funding identifier(s) : Not specified
Name of the organisation/institution/group(*) : Not specified
Details about the contributing person(s)
Contributor type(*) : Contact
Given name(*) : Andreas
Family name(s)(*) : Zueiter
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified
Email address : Not specified
Phone number : Not specified
Organisation(s) associated with the contributor
Name(*) : Klinik I für innere Medizin, University Hospital Cologne
Address : Kerpener Straße 62 50937 Cologne, Germany
Web page : https://innere1.uk-koeln.de
Digital identifier(s)
Identifier(*) : 05mxhda18
Scheme(*) : ROR
Identifier(*) : Not specified
Scheme(*) : Not specified
Name(*) : Not specified
Address : Not specified
Web page : Not specified
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified

Is it a personal or organisational contribution?(*) : Organisational
Details about the contributing organisation(s)/institution(s)/group(s)
Contributor type(*) : Sponsor (primary)
Funding identifier(s) : Not specified
Name of the organisation/institution/group(*) : Universitätsklinikum Hamburg-Eppendorf
Details about the contributing person(s)
Contributor type(*) : Not specified
Given name(*) : Not specified
Family name(s)(*) : Not specified
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified
Email address : Not specified
Phone number : Not specified
Organisation(s) associated with the contributor
Name(*) : Universitätsklinikum Hamburg-Eppendorf
Address : Martinistrasse 52 20246 Hamburg, Germany
Web page : https://www.uke.de/
Digital identifier(s)
Identifier(*) : 01zgy1s35
Scheme(*) : ROR
Identifier(*) : Not specified
Scheme(*) : Not specified
Name(*) : Not specified
Address : Not specified
Web page : Not specified
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified

Alternative identifiers

Type of the registry(*) : NCT (ClinicalTrials.gov)
Identifier(*) : NCT01404741

Characteristics of the Project

Is it an interventional or non-interventional Project? : Interventional
Specification of the type of the Project
Interventional Project type : Factorial
Non-interventional Project type : []
Primary health condition(s) or disease(s) considered in the Project
Primary health condition or disease name(*) : Myelodysplastic syndrome (disorder)
Terminology/classification(*) : SNOMED CT
Code of the health condition or disease : 109995007
Primary health condition or disease name(*) : Chronic myelomonocytic leukemia (disorder)
Terminology/classification(*) : SNOMED CT
Code of the health condition or disease : 127225006
Groups of diseases or conditions(*)
Which groups of diseases or conditions were the data collected on? : Neoplasms (II)
On which diseases or conditions were the data collected? : []
Collects mortality data? : Not specified
Administrative information about the Project
Status of the ethics committee approval : Not specified
Overall Project status : Completed: Recruitment, data collection, and data quality management completed normally
Participants enrolled by invitation? : Not specified
Start date : 29 October 2011
End date : 30 November 2016
Mono- or multicentric? : Multicentric
Number of centers : 17
One or more data providers? : Not specified
Number of data providers : Not specified
Primary subject(*) : Person
Eligibility criteria for Project participants
Eligibility criteria: Minimum age
Minimum eligible age(*) : 55
Unit of age(*) : Years
Eligibility criteria: Maximum age
Maximum eligible age(*) : 70
Unit of age(*) : Years
Eligible gender : Not applicable
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
Population of the Project(*)
Coverage : National
Country(ies) : Germany
Region(s) and/or city(ies) : Berlin, Bonn, Dresden, Düsseldorf, Essen, Frankfurt am Main, Göttingen, Hamburg, Hannover, Cologne, Mannheim, Munich, Nurenberg, Tübingen, Ulm
Interventions of the Project
Name of the intervention(*) : 5-azacytidine until progress
Type of the intervention : Drug (including placebo)
Additional information about the intervention : Not specified
Name(s) of the arm(s) associated with the given intervention : Active Comparator: 5-azacytidine treatment until progress 5-azacytidine until progress
Name of the intervention(*) : allogeneic stem cell transplantation
Type of the intervention : Genetic (including gene transfer, stem cell and recombinant DNA)
Additional information about the intervention : Not specified
Name(s) of the arm(s) associated with the given intervention : Experimental: allogeneic stem cell transplantation after 4 cycles 5-azacytidine and if donor available: allogeneic stem cell transplantation after reduced intensity conditioning
Exposures of the Project
Name of the exposure : Not specified
Type of the exposure : Not specified
Additional information about the exposure : Not specified
Name(s) of the group(s) associated with the given exposure : Not specified
Outcome measures in the Project
Name of the outcome measure(*) : overall survival
Description of the outcome measure : 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
Type of the outcome measure : Primary
Time point(s) of assessment : Not specified
Name of the outcome measure(*) : response
Description of the outcome measure : 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
Type of the outcome measure : Secondary
Time point(s) of assessment : Not specified
Name of the outcome measure(*) : event-free survival
Description of the outcome measure : 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
Type of the outcome measure : Secondary
Time point(s) of assessment : Not specified
Name of the outcome measure(*) : overall survival
Description of the outcome measure : Comparison of overall survival between both arms (230 pat.). - evaluation of survival status (alive or death/date of death) in the whole study period
Type of the outcome measure : Secondary
Time point(s) of assessment : Not specified
Name of the outcome measure(*) : impact of Comorbidity-index on outcome
Description of the outcome measure : 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)
Type of the outcome measure : Secondary
Time point(s) of assessment : Not specified
Name of the outcome measure(*) : Treatment-related mortality
Description of the outcome measure : compare treatment related mortality in both arms (230 pat.): - death according to treatment in both arms
Type of the outcome measure : Secondary
Time point(s) of assessment : Not specified
Name of the outcome measure(*) : Evaluation of toxicity
Description of the outcome measure : 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
Type of the outcome measure : Secondary
Time point(s) of assessment : Not specified
Name of the outcome measure(*) : quality of life
Description of the outcome measure : 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
Type of the outcome measure : Secondary
Time point(s) of assessment : Not specified
Additional information about the Project : Not specified
Additional assessments/measurements in the Project : []
Data sharing strategy of the Project(*)
Is it planned to share the data?(*) : Undecided, it is not yet known if data will be made available
Supporting documents available in addition to the data : []
When and for how long will the data be available? : Not specified
Criteria for data access : Not specified
Additional information about data sharing : Not specified
DataSHIELD/Opal infrastructure available? : Not specified
Link to data request application : Not specified
Web page with additional information about data sharing : Not specified
Record linkage possible? : Not specified
Non-interventional aspects of the Project
Temporal design : []
Target follow-up duration of the Project
Target follow-up duration(*) : Not specified
Unit of time(*) : Not specified
Number of follow-ups conducted : Not specified
Biosamples retained in a biorepository : []
Specific types of retained biosamples : Not specified
Interventional aspects of the Project
Numerical phase : Phase-2
Masking of intervention(s) assignment
Masking implemented? : false
Who is masked? : []
Additional information about masking : Not specified
Type of allocation of participants to an arm : Nonrandomized
Off-label use of a drug product : Not specified

Comparison between 5 – azacytidine treatment and 5 – azacytidine followed by allogeneic stem cell transplantation in elderly patients with MDS according to donor availability

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