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

Sponsor

Johnson & Johnson Pharmaceutical Research & Development, L.L.C. (Industry)

Overall Status

Completed

CT.gov ID

NCT00050154

Collaborator

(none)

Enrollment

Duration (Months)

Study Details

Study Description

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.

Study Design

Study Type:

Interventional

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 :

Jul 1, 2002

Actual Study Completion Date :

May 1, 2006

Outcome Measures

Primary Outcome Measures

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

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. []

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

Investigators

Study Director: Johnson & Johnson Pharmaceutical Research and Development, L.L.C. Clinical Trial, Johnson & Johnson Pharmaceutical Research & Development, L.L.C.