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

Johnson & Johnson (J&J)

Status and phase

Completed

Phase 2

Conditions

Myelodysplastic Syndrome

Treatments

Drug: ZARNESTRA, tipifarnib, R115777

Study type

Interventional

Funder types

Industry

Identifiers

NCT00050154

CR004027

Details and patient eligibility

About

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.

Full 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.

Enrollment

82 patients

Sex

All

Ages

18+ years old

Volunteers

No 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