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

Phase 1

Active, not recruiting

• Conditions: Myelodysplastic Syndrome (MDS); MedDRA version: 20.0 Level: PT Classification code 10028533 Term: Myelodysplastic syndrome System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2010-018467-42-DE
• Lead Sponsor: niversity Medical Center Hamburg-Eppendorf

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-10-25
• Last Update Posted: 2019-11-25

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: Not specified
• Target Recruitment: 110

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®)
•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 bilirubine < 3 x the upper limit of normal
•Sufficient cardiac function (ejection fraction > 30 %)

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 76
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 34

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To compare the overall survival at three years of patients who receive after 4 cycles of 5-azacytidine (Vidaza®) either allogeneic stem cell transplantation or continuous 5-azacytidine (Vidaza®) if no compatible donor is available.;<br> Secondary Objective: Comparison of overall survival” and event-free survival” between treatment arm Allogeneic stem cell transplantation after 5 azacytidine induction” and treatment arm Continuous 5 azacytidine alone” over the whole study period.<br> To assess the response rate, the event-free survival at three years, toxicity and treatment-related mortality, the impact of the comorbidity-index (HCT-CI) on outcome and the quality of life in both treatment arms. Further to analyse prognostic factors which influence survival in both arms.<br> ;Primary end point(s): Overall survival at three years between both arms.;Timepoint(s) of evaluation of this end point: After three years