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Study of Tipifarnib in Patients With High-Risk Myelodysplastic Syndrome (MDS)

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: NCT00050154
Recruitment Status : Completed
First Posted : November 25, 2002
Last Update Posted : April 27, 2010
Information provided by:
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

Brief Summary:
The purpose of this study is to characterize the hematological response rate, as well as other parameters of efficacy and safety induced by tipifarnib in patients with high-risk myelodysplastic syndrome (MDS). Tipifarnib belongs to a class of drugs called Farnesyl Transferase Inhibitors (FTI). It blocks proteins that make cancer cells grow.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome Drug: ZARNESTRA, tipifarnib, R115777 Phase 2

Detailed Description:

Treatment with tipifarnib will be given during one or more periods of time called cycle(s). Each cycle will be 28 days long and patients will take tipifarnib for the first 21 days of each cycle. No medication will be taken during the last 7 days of each cycle. On day 1 and 15 of each cycle, patients will be asked about any side effects that have occurred since their last visit. Blood will drawn for routine testing to evaluate any possible effects of tipifarnib on white blood cells or on specific elements, that can be measured in the blood. The study doctor will decide if any bone marrow aspirates or biopsies should be taken. Tipifarnib will be given until the patient's disease worsens or they develop unacceptable side effects or until they withdraw consent to receive tipifarnib. When tipifarnib treatment is ended or if the patient leaves the study early, they will be asked to come in for a final visit. The study doctor will decide if any blood draws, bone marrow aspirates or biopsies need to be taken.

Tipifarnib is 300 mg administered orally as three 100 mg tablets twice daily for the first 21 days in each 28-day cycle. Tipifarnib will be administered until the patient discontinues treatment due to disease progression or unacceptable toxicity.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 82 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Phase 2 Study to Evaluate the Efficacy and Safety of the Farnesyl-Transferase Inhibitor ZARNESTRA(tm) (R115777) in Subjects With High-Risk Myelodysplastic Syndrome (MDS)
Study Start Date : July 2002
Actual Study Completion Date : May 2006

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. The purpose of this research study is to determine if tipifarnib leads to a complete response for in patients with high-risk Myelodysplastic Syndrome (MDS).

Secondary Outcome Measures :
  1. Efficacy will be assessed by evaluation of bone marrow and hematologic laboratory tests. Safety will be assessed by evaluation of adverse events and clinical laboratory tests.

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:

  • Pathological evidence of MDS
  • Not more than 1 prior cytotoxic treatment for MDS
  • Able to take oral study drug
  • Able to understand and provide signed informed consent

Exclusion Criteria:

  • Refractory anemia (RA) or RA with excess of blasts (RAEB) or patients with RAEB with < or = 10% marrow blasts
  • Treatment-related MDS, if treated with chemotherapy less than 3 years ago
  • Not adequately recovered from any treatment-related non-hematological toxicity
  • Refractory to platelet transfusion
  • Candidates for hematopoietic stem cell transplantation
  • Previous therapy with a farnesyl transferase inhibitor
  • Prior extensive radiation therapy

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): NCT00050154

Sponsors and Collaborators
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
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Study Director: Johnson & Johnson Pharmaceutical Research and Development, L.L.C. Clinical Trial Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Layout table for additonal information Identifier: NCT00050154    
Obsolete Identifiers: NCT00722787
Other Study ID Numbers: CR004027
First Posted: November 25, 2002    Key Record Dates
Last Update Posted: April 27, 2010
Last Verified: April 2010
Keywords provided by Johnson & Johnson Pharmaceutical Research & Development, L.L.C.:
Myelodysplastic syndromes
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antineoplastic Agents