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Treatment of MDS Patients With Single Agent Temsirolimus - a Pilot Study

Phase 0
18 Years
Open (Enrolling)
Myelodysplastic Syndrome

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Trial Information

Treatment of MDS Patients With Single Agent Temsirolimus - a Pilot Study

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

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

- 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

- 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

Type of Study:


Study Design:

Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall hematological response rate using modified IWG criteria (combination of CR, PR, marrow-CR and SD with HI).

Outcome Time Frame:

at 4 months

Safety Issue:


Principal Investigator

Uwe Platzbecker, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Medizinische Klinik I, Universitätsklinikum Carl-Gustav-Carus, Dresden, Germany


Germany: Federal Institute for Drugs and Medical Devices

Study ID:




Start Date:

April 2010

Completion Date:

Related Keywords:

  • Myelodysplastic Syndrome
  • Myelodysplastic Syndromes of all IPSS subgroups
  • Myelodysplastic Syndromes
  • Preleukemia