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Temsirolimus in Myelodysplastic Syndrome (MDS) (TEMDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01111448
Recruitment Status : Terminated (Unfavourable Risk-Benefit-Ratio)
First Posted : April 27, 2010
Last Update Posted : February 13, 2015
Wyeth is now a wholly owned subsidiary of Pfizer
Information provided by (Responsible Party):
Technische Universität Dresden

Brief Summary:
The goal of this Pilot-study is to evaluate the response of unselected MDS patients to temsirolimus a drug approved for the treatment of renal cell cancer. It is planned to give temsirolimus at a weekly dose of 25 mg as intravenous infusion for a maximum duration of 12 months. Regular bone marrow biopsies are planned for controlling MDS response.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome Drug: Temsirolimus Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment of MDS Patients With Single Agent Temsirolimus - a Pilot Study
Study Start Date : April 2010
Actual Primary Completion Date : June 2013
Actual Study Completion Date : June 2014

Arm Intervention/treatment
Experimental: Temsirolimus
25 mg/day 1; 8; 15; 22 of each 28-day cycle
Drug: Temsirolimus
25 mg/day 1; 8; 15; 22 of each 28-day cycle as intravenous infusion over 30 min

Primary Outcome Measures :
  1. Overall hematological response rate using modified IWG criteria (combination of CR, PR, marrow-CR and SD with HI). [ Time Frame: at 4 months ]

Secondary Outcome Measures :
  1. Toxicity as measured by NCI CTCAE v3.0 [ Time Frame: 4 and 12 months ]
  2. Overall survival [ Time Frame: 1 year ]
  3. Progression-free-survival [ Time Frame: 1 year ]
  4. Rate of leukemic progression [ Time Frame: 1 year ]
  5. Overall hematological response rate using modified IWG-criteria [ Time Frame: 1 year ]
  6. Quality of life as measured by EORTC-QLQ30 [ Time Frame: 4 months, 12 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 features according to IPSS and requiring at least 4 units of red blood cells within the last 8 weeks prior to screening visit or presenting with neutropenia (<1 Gpt/l neutrophils) or
    • Group II (high-risk): INT-2 or HIGH-risk IPSS refractory or intolerant to 5-Azacytidine.

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;
  • laboratory test results within these ranges:

    • Serum creatinine <= 177 µmo/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);
    • sexual abstinence;
    • partner, who had vasectomy (confirmed by two negative analyses of semen);
  • 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 consequences of the study;
  • patients who are not likely to follow the trial protocol (lack of willingness to cooperate).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01111448

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Klinikum Chemnitz Klinik für Innere Medizin III
Chemnitz, Germany, 09113
Universitätsklinikum C. G. Carus der TU Dresden
Dresden, Germany, 01307
Universitätsklinikum Düsseldorf Klinik für Hämatologie, Onkologie und Klin. Immunologie
Düsseldorf, Germany, 40225
Universitätsmedizin Göttingen Georg-August-Universität Abteilung Hämatologie und Onkologie
Goettingen, Germany, 37075
Universitätsklinikum Leipzig AöR
Leipzig, Germany, 04103
Forschungsgesellschaft mbH
Leipzig, Germany, 04289
Klinikum Mannheim GmbH III. Medizinische Universitätsklinik -SP Hämatologie/Onkologie
Mannheim, Germany, 68167
Sponsors and Collaborators
Technische Universität Dresden
Wyeth is now a wholly owned subsidiary of Pfizer
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Principal Investigator: Uwe Platzbecker, MD, PhD Medizinische Klinik I, Universitätsklinikum Carl-Gustav-Carus, Dresden, Germany

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Responsible Party: Technische Universität Dresden Identifier: NCT01111448    
Other Study ID Numbers: TUD-TEMDS1-042
First Posted: April 27, 2010    Key Record Dates
Last Update Posted: February 13, 2015
Last Verified: February 2015
Keywords provided by Technische Universität Dresden:
Myelodysplastic Syndromes of all IPSS subgroups
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents