A Pivotal Randomized Study of Lonafarnib Versus Placebo in the Treatment of Subjects With Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) Who Are Platelet Transfusion Dependent With or Without Anemia
- Confirmed MDS (RA, RARS, RAEB, RAEB-T) or CMML according to FAB classification.
- Platelet transfusion dependence (requiring 1 to 8 platelet transfusion events every 4
week period (Day 84 to Day 57, Day 56 to Day 29, and Day 28 to Day 1) over an
8-week retrospective and 4-week prospective screening period).
- The individual number of platelet transfusion events during the three 4-weekly
periods (Day 84 to Day -57; Day -56 to Day 29; Day -28 to Day -1) must not differ
by greater more than 2 from the average number of platelet transfusion events during
the 12 week screening period.
- If the subject is RBC transfusion dependent, the number of RBC transfusion events
during the three 4-weekly periods (Days -84 to -57; Day -56 to Day 29 and Day -28 to
Day -1) must not differ by more than 2 from the average number of RBC transfusion
events during this 12 week screening period.
ECOG PS 0-2.
- Subjects with chemotherapy/radiotherapy-associated secondary MDS.
- <12 Weeks (prior to Day-1 Randomization) from any investigational drug use, any
chemotherapy, radiotherapy, immunotherapy and any other treatment or MDS/CMML other
than best supportive care.
- Hx of bone-marrow or peripheral stem-cell transplantation or treatment with donor
- Hx of AML.
- Known hx of immune thrombocytopenic purpura.
- Marked baseline prolongation of QTc interval, CTCAE Grade >=1.
- Use of ketokonazole within 72 hours prior to study drug administration.