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A Phase I Study of MGCD0103 Given Twice Weekly in Patients With Leukemia or Myelodysplastic Syndromes

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: NCT00324194
Recruitment Status : Completed
First Posted : May 10, 2006
Last Update Posted : January 8, 2015
Information provided by:
Mirati Therapeutics Inc.

Brief Summary:
In this study, MGCD0103, a new anticancer drug under investigation, is given twice weekly to patients with leukemia or myelodysplastic syndromes.

Condition or disease Intervention/treatment Phase
Leukemia Myelodysplastic Syndromes Drug: MGCD0103 Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Study of MGCD0103 Given as a Twice Weekly Oral Dose in Patients With Leukemia or Myelodysplastic Syndromes
Study Start Date : February 2005
Actual Primary Completion Date : March 2008
Actual Study Completion Date : August 2008

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: 1
MGCD0103 oral dose 2 times per week.
Drug: MGCD0103
MGCD0103 oral dose given 2 times per week.

Primary Outcome Measures :
  1. Safety and tolerability. [ Time Frame: 1 year (anticipated) ]

Secondary Outcome Measures :
  1. Clinical response. [ Time Frame: 1 year (anticipated) ]
  2. Dose limiting toxicities [ Time Frame: 1 year (anticipated) ]
  3. Pharmacokinetics [ Time Frame: 1 year (anticipated) ]
  4. Pharmacodynamics (histone acetylation, biomarkers) [ Time Frame: 1 year (anticipated) ]

Information from the National Library of Medicine

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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:

  • Patients must have a diagnosis of one of the following:

    • relapsed or refractory AML or ALL that has failed to respond to standard therapy, has progressed despite standard therapy.
    • relapsed or refractory Myelodysplastic Syndromes including CMML (chronic myelomonocytic leukemia) or other myeloproliferative disorders provided there is an MDS component.
    • previously untreated AML or Myelodysplastic Syndromes in patients >60 years of age who refused or are not candidates for induction chemotherapy
    • Patients with relapsed or refractory CML that has failed to respond to imatinib therapy or standard therapy, has progressed despite standard therapy, or for which no standard therapy exists.
  • ECOG performance status of 0, 1, or 2.
  • Age ≥18 years.
  • Laboratory requirements.
  • Patients or their legal representative must be able to read, understand, and sign a written informed consent (approved by the IRB/EC) prior to study entry.

Exclusion Criteria:

  • Patients with other active malignancy except basal cell carcinoma or cervical intraepithelial neoplasia (CIN / cervical in situ).
  • Patients with suspicion of CNS involvement (tests are not required to rule out CNS involvement in the absence of signs or symptoms).
  • Pregnant or lactating women. Women of child-bearing potential must have a negative serum pregnancy test documented within 7 days prior to registration on study.
  • Patients and their partners, if either are of childbearing potential, not using adequate birth control measures throughout the course of the study. Both men and women enrolled on study must agree to use a medically acceptable effective form of contraception during the study and for 90 days following the last dose of study medication. An effective form of contraception is an oral contraceptive or a double barrier method, such as condom with diaphragm.
  • Patients with serious illnesses, medical conditions, or other medical history, including laboratory results, which, in the investigator's opinion, would be likely to interfere with a patient's participation in the study, or with the interpretation of the results.
  • Patients who have been treated with any investigational drug within 30 days prior to study initiation (an investigational drug is one for which there is no approved indication), or who are receiving concurrent treatment with other experimental drugs or anti-cancer therapy.
  • Known hypersensitivity to any of the components of MGCD0103.
  • Known HIV or Hepatitis B or C (tests do not need to be performed to rule out any of these infections).
  • Any psychiatric illness/social situations that would, in the judgment of the investigator, limit compliance with study requirements.

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): NCT00324194

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United States, Florida
Lee Moffitt Cancer Center
Tampa, Florida, United States, 33612
United States, Illinois
University of Chicago Medical Center
Chicago, Illinois, United States, 60637
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
Canada, Quebec
Sir Mortimer Davis Jewish General Hospital
Montreal, Quebec, Canada
Sponsors and Collaborators
Mirati Therapeutics Inc.
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Study Director: Gregory Reid, MSc, MBA MethylGene Inc.

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Responsible Party: Gregory Reid, Chief Medical Officer, MethylGene, Inc. Identifier: NCT00324194    
Other Study ID Numbers: 0103-004
First Posted: May 10, 2006    Key Record Dates
Last Update Posted: January 8, 2015
Last Verified: January 2015
Keywords provided by Mirati Therapeutics Inc.:
Myelodysplastic Syndromes
Phase I
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Histone Deacetylase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action