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

NCT ID: NCT01404741

Last Updated: 2023-10-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 191 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-07-31
• Study Completion Date: 2021-07-31

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).

Conditions

Myelodysplastic Syndrome; Chronic Myelomonocytic Leukemia

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: FACTORIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

5-azacytidine treatment until progress

5-azacytidine until progress

Group Type: ACTIVE_COMPARATOR

5-azacytidine until progress

Intervention Type: PROCEDURE

if no donor available 5-azacytidine until progress or toxicities

allogeneic stem cell transplantation

after 4 cycles 5-azacytidine and if donor available: allogeneic stem cell transplantation after reduced intensity conditioning

Group Type: EXPERIMENTAL

allogeneic stem cell transplantation

Intervention Type: PROCEDURE

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

Interventions

allogeneic stem cell transplantation

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

Intervention Type: PROCEDURE

5-azacytidine until progress

if no donor available 5-azacytidine until progress or toxicities

Intervention Type: PROCEDURE

Eligibility 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

• Minimum Eligible Age: 55 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Universitätsklinikum Hamburg-Eppendorf

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Nicolaus Kroeger, Prof.

Role: PRINCIPAL_INVESTIGATOR

University Medical Centre Hamburg-Eppendorf, Stem-Cell-Transplantation

Locations

Charité Campus Benjamin Franklin

Berlin, , Germany

Uniklinikum Bonn

Bonn, , Germany

Universität zu Köln

Cologne, , Germany

Universitätsklinikum Dresden

Dresden, , Germany

Universitätsklinikum Düsseldorf

Düsseldorf, , Germany

Universitätsklinikum Essen

Essen, , Germany

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

Hanover, , Germany

Universitätsklinikum Mannheim

Mannheim, , Germany

Klinikum rechts der Isar

München, , Germany

Universitätsklinikum Münster

Münster, , Germany

Klinikum Nürnberg

Nuremberg, , Germany

Medizinische Universitätsklinik II

Tübingen, , Germany

Universitätsklinikum Ulm

Ulm, , Germany

Countries

Germany

References

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.

Reference Type: DERIVED

PMID: 34283629 (View on PubMed)

Other Identifiers

VidazaAlloStudy

Identifier Type: -

Identifier Source: org_study_id