European Leukemia Trial Registry
Trial: TEMDS

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Public Title Temsirolimus in Myelodysplastic Syndrome
Scientific Title Treatment of MDS patients with single agent temsirolimus - a pilot study
Short Title TEMDS
Id KN/ELN LN_DEUTSC_2009_327
Trialgroup Deutsche MDS
Type of Trial multicentric, open-label, pilot study
Phase N/A
Disease Myelodysplastic Syndrome(MDS) Low risk and intermedia I
Myelodysplastic Syndrome(MDS) Intermedia II and high risk
Stage of Disease .
Aim
  • Is to evaluate the response of MDS patients to temsirolimus. An overall response rate of 20 % or more according to modified IWG-criteria will be regarded as clinically significant.
  • • To describe the safety and tolerability of temsirolimus in MDS;
  • • to determine the effects of temsirolimus on quality of life in patients with MDS;
  • • to determine the effect of temsirolimus on modulating molecular targets in MDS targets
Inclusion Criteria
  • •Age >=18 years at the time of signing the informed consent form;
  • • Patients able to understand the consequences of participating in this trial and not
  • - having any disorders or other circumstances (i.e. being in ward or imprisoned) which
  • - keeps them from giving written informed consent;
  • • cytologically or histologically established diagnosis of de novo or therapy-related MDS according to the FAB-classification, either previously treated or untreated, presenting with:
  • - Group I (low-risk): Low- or INT-1 risk with and without pretreatment
  • - or
  • - Group II (high-risk): INT-2 or HIGH-risk IPSS with 5-Azacytidine treatment failure
  • CMML patients of dysplastic phenotype (WBC < 13 Gpt/l) may be included in both arms according to IPSS. CMML patients showing proliferative phenotype (WBC >=13 Gpt/l) will be included in the high risk arm;
  • • not eligible for an immediate allogeneic HSCT or conventional chemotherapy
  • • all previous MDS specific therapies (except supportive approaches like transfusions or antibiotics) must have been discontinued at least 4 weeks prior to study enrollment;
  • • ECOG performance status of <= 3 at study entry (see Appendix 01);
  • • laboratory test results within these ranges:
  • Serum creatinine <= 177 ìmo/l (<= 2.0 mg/dL);
  • total bilirubin <= 3 x ULN;
  • AST (SGOT) and ALT (SGPT) <= 3 x ULN;
  • total fasting cholesterol <= 9.1 mmol/l (350 mg/dl);
  • fasting triglyceride level <= 4.5 mmol/l (400 mg/dl);
  • platelets > 25 Gpt/l without transfusion support in patients with LOW- and INT-1 Risk according to IPSS;
  • • signed informed consent
Exclusion Criteria
  • • For Patients with LOW- or INT1-Risk according to IPSS: Thrombocytopenia below 25 Gpt/l (INT2- and HIGH-IPSS patients may be included irrespective of platelet count);
  • • known hypersensitivity to temsirolimus, sirolimus or any components of the infusion solution (dl-alpha-tocopherol, propylene glycol, anhydrous citric acid, polysorbate 80, polyethylene glycol 400, dehydrated alcohol);
  • • known hypersensitivity to macrolid antibiotics (because of structural similarities between this class of antibiotics and study medication);
  • • any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study;
  • • known positive for HIV or any other uncontrolled infection;
  • • presence of any other malignancy being not in complete remission for at least 3 years (previous chemotherapy for other malignancies is not an exclusion criteria);
  • • necessity of therapeutic anticoagulation (excluding low dose ASS);
  • • participation in an other clinical trial within the last 4 weeks
  • • pregnant or breast feeding females (lactating females must agree not to breast feed while on study);
  • • females of childbearing potential (FCBP) except those fulfilling at least one of the following criteria:
  • post-menopausal (12 months of natural amenorrhea or 6 months of amenorrhea with serum FSH > 40 U/ml);
  • post-surgery (6 weeks after bilateral ovarectomy with or without hysterectomy);
  • regular and correct use of contraceptives with a PEARL Index of < 1% (e.g. implants, depot formulations of hormones, oral contraceptives, intra uterine device – IUD);
  • male patients, who do not agree to use a latex condom during sexual contact with females of childbearing potential while participating in the study and for at least 3 months following discontinuation from the study even if he has undergone a successful vasectomy;
  • • patients with a history of chronic drug abuse or another illness which does not allow the patient to assess the nature and/or possible consquences of the study;
  • • patients who are not likely to follow the trial protocol (lack of willingness to cooperate).
Age >= 18 years
Status Closed
Start of Recruitment 15.12.2009
Recruiting Countries Germany
Leader Platzbecker, Prof. Dr. med., Uwe
Scientific Contact Platzbecker, Prof. Dr. med., Uwe (Studienleiter)
Contact Person

principal investigator
Platzbecker, Prof. Dr. med., Uwe
Tel: +49 (0)351 4582583
Fax: +49 (0)351 458-5362
Email: Uwe.Platzbecker@uniklinikum-dresden.de

Study Physician
Wermke, Dr. med., Martin
Tel: +49 (0)351 458 15624
Email: Martin.Wermke@uniklinikum-dresden.de

Centre of Trial Universitätsklinikum Carl Gustav Carus, Dresden
Shortprotocol Shortprotocol
Diagnostics

Zytomorphology
Hämatologisches Labor, Universitätsklinikum Dresden

Cytogenetics
Labor für hämatologische Zytogenetik Göttingen

Sponsors TU Dresden (Dresden University of Technology; Study Alliance Leukemia (SAL) / Studienzentrale)
Supporters Deutsche Krebshilfe e.V.
Wyeth
Other Registers ClinicalTrials.gov NCT01111448 (primary Register)
European Clinical Trials Database - EUDRACT 2009-014768-21
Outcomes
  • Primary endpoint is the overall hematological response rate (combination of CR, PR, marrow-CR and SD with HI) at 4 months using modified IWG-criteria. (Primary Outcome)
  • Toxicity as measured by NCI CTCAE v3.0;
  • • overall survival at 1 year;
  • • progression-free survival at 1 year;
  • • rate of leukemic progression at 1 year;
  • • overall hematological response rate at 1 year using modified IWG-criteria;
  • • quality of life as measured by EORTC-QLQ30.
Interventions
  • • treatment with single agent Temsirolimus 25 mg/day on day 1; 8; 15; 22 of each 28-day cycle as intravenous infusion
created 05.01.2010 Irene Gleske
changed 13.11.2014 Alexandra Lucaciu
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