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Safety and Efficacy of Azacitidine, and Thalidomide in Higher Risk MDS (Myelodysplastic Syndrome) (IMDS001)

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: NCT00704704
Recruitment Status : Unknown
Verified June 2008 by Tel-Aviv Sourasky Medical Center.
Recruitment status was:  Not yet recruiting
First Posted : June 25, 2008
Last Update Posted : June 25, 2008
Information provided by:
Tel-Aviv Sourasky Medical Center

Brief Summary:

Study Objectives The aim of the study is to evaluate the safety and efficacy of the combination of 5-Aza-Cytidine + Thalidomide on the course of hrMDS patients.

Primary end point:

• To evaluate the overall response rate (CR+PR) of the combination of 5-Aza-Cytidine + Thalidomide in hrMDS patients (INT-2 and High risk as defined by IPSS).

Secondary end points:

  • To evaluate the safety of the combination of Thalidomide+5-Aza-Cytidine in high risk MDS patients.
  • Hematological improvement rate.
  • Cytogenetic response.
  • Progression free survival (PFS).
  • Quality of life assessment (FACT: MDS and peripheral neuropathy QOL Questionnaires).

Study design:

This is a multicenter, phase II, single arm study designed to evaluate the safety and efficacy of the combination of Thalidomide+5-Aza-Cytidine in high risk MDS patients (INT-2 and High risk defined by IPSS) who are older than 18 years of age. Potential study subjects will sign an informed consent prior to undergoing any study related procedure. Number of patients to be enrolled 50.

Treatment plan: 5-aza-cytidine (75 mg m2/d) will be injected subcutaneously in 5-day cycle every 28 days, for a total of 12 cycles.

Thalidomide will be given at the dose of 50 mg/d, from day 1 until for 6 months together with 5-aza-cytidine .

Treatment period includes 5-aza-cytidine (75 mg m2/d) will be injected subcutaneously in 5-day cycle every 28 days. Total number of 12 cycles or until progression or toxicity. Cycle delay of maximum 2 weeks in case of hematological toxicity grade 3-4 at investigator discretion.

Duration of the follow up period is 6 months. Duration of study The duration of the treatment period is approximately 12 months. This time is required to complete the treatment, and to determine the safety profile and the response rate. The duration of the Follow period will be approximately a half year. The occurrence of PD will determine the duration of progression-free survival of each patient.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome Drug: 5-aza-cytidine and Thalidomide Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Single Arm Study to Determine the Safety and Efficacy of Azacitidine, and Thalidomide in Higher Risk Myelodysplastic Syndrome
Study Start Date : September 2008
Estimated Primary Completion Date : September 2010
Estimated Study Completion Date : April 2011

Resource links provided by the National Library of Medicine

Intervention Details:
  • Drug: 5-aza-cytidine and Thalidomide
    5-AZA-Cytidine 75mg/m2 5 days a month. 12 months. Thalidomide 50mg orally daily for 6 months.
    Other Name: vidaza

Primary Outcome Measures :
  1. overall response rate measured by complete blood counts and bone marrow examinations [ Time Frame: at study entry. after 6 months and after one year ]
  2. overall response rate [ Time Frame: 6 and 12 months ]

Secondary Outcome Measures :
  1. *safety *hematological improvement *cytogenetic response *progression free survival * Quality of life assessment (FACT: MDS and peripheral neuropathy QOL Questionnaires). [ Time Frame: at entry , 6 and 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:

  • Patient is, in the investigator(s) opinion, willing and able to comply with the protocol requirements.
  • Patient has given voluntary written informed consent before performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to their future medical care.
  • Patient is older than 18 years at the time of signing the informed consent.
  • Female patient is either post-menopausal or surgically sterilized or willing to use an acceptable double method of birth control (i.e., a hormonal contraceptive, intrauterine device, diaphragm with spermicide, condom with spermicide, or abstinence) for the duration of the study.
  • Female of childbearing potential must have a negative serum β-human chorionic gonadotropin ( beta sub unit-HCG) pregnancy test both 24 hours prior to beginning of therapy and then at 4 weeks intervals in women with regular menstrual cycles or every 2 weeks in women with irregular menstrual cycles during study treatment for subjects of childbearing potential.
  • Male patient agrees to use an acceptable method for contraception (i.e., condom or abstinence) during study drug therapy (including dose interruption) and for 4 weeks after discontinuation of thalidomide therapy.
  • Patient was diagnosed with myelodysplastic syndrome INT-2 or High risk according IPSS score .Bone marrow aspiration examination including cytogenetics performed up to 12 months before patient signing informed consent.
  • Patient has a Performance Status 0-2 (WHO).
  • Patient has a life-expectancy > 6 months
  • Patient has not known active infectious hepatitis type B or C, or HIV infection.
  • Patient is epopoietin resistant. Erythropoietin should be discontinued 28 days before starting treatment period.
  • Patient has the following laboratory values within 14 days before Baseline (day 1 of the Cycle 1):

    • Platelet count ≥ 25 x 109/L without transfusion support within 7 days before the test.
    • Absolute neutrophil count (ANC) ≥ 0.5 x 109/L without the use of growth factors.
    • Aspartate transaminase (AST): ≤ 2.5 x the upper limit of normal (ULN).
    • Alanine transaminase (ALT): ≤ 2.5 x the ULN.
    • Total bilirubin: ≤ 1.5 x the ULN.
    • Serum creatinine< 2 X the ULN.

Exclusion Criteria:


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): NCT00704704

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Contact: Moshe Mittelman, MD 972-3-6973366 moshemt@TASMC.HEALTH.GOV.IL
Contact: Elizabeth Naparstek, MD 972-524266435

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Hematolpgical department Tel Aviv Medical Center Not yet recruiting
Tel-Aviv, Israel
Contact: Moshe Mittelman, MD    972-3-6973366   
Contact    972-52-4266435   
Principal Investigator: Moshe Mittelman, MD         
Sponsors and Collaborators
Tel-Aviv Sourasky Medical Center

1. Greenberg PL. Risk factors and their relationship to prognosis in myelodysplastic syndromes. Leuk Res. 1998;22 Suppl 1:S3-6. 2. Sanz GF, Sanz MA, Greenberg PL. Prognostic factors and scoring systems in myelodysplastic syndromes. Haematologica. 1998;83:358-368. 3. Hellstrom-Lindberg E, Ahlgren T, Beguin Y, et al. Treatment of anemia in myelodysplastic syndromes with granulocyte colony-stimulating factor plus erythropoietin: results from a randomized phase II study and long-term follow-up of 71 patients. Blood. 1998;92:68-75. 4. Silverman LR, Demakos EP, Peterson BL, et al. Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002;20:2429-2440. 5. Silverman LR, McKenzie DR, Peterson BL, et al. Further analysis of trials with azacitidine in patients with myelodysplastic syndrome: studies 8421, 8921, and 9221 by the Cancer and Leukemia Group B. J Clin Oncol. 2006;24:3895-3903. 6.

Layout table for additonal information Identifier: NCT00704704     History of Changes
Other Study ID Numbers: TASMC-08-MM-TLV-0069-08-CTIL
First Posted: June 25, 2008    Key Record Dates
Last Update Posted: June 25, 2008
Last Verified: June 2008
Keywords provided by Tel-Aviv Sourasky Medical Center:
high risk MDS
response rate
cytogenetic response
QOL questionaries
INT-2 or High risk MDS
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors