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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


Phase 3
18 Years
N/A
Not Enrolling
Both
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic, Myelodysplasia, Myelomonocytic

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Trial Information

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


Inclusion Criteria:



- 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.

Exclusion Criteria:

- 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
lymphocyte infusion.

- 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.

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Outcome Measure:

Proportion of subjects who achieved platelet transfusion independence for any 8-consecutive week period after randomization without worsening of RBC transfusion requirements or hemoglobin (untransfused) during the same 8-consecutive-week period.

Outcome Time Frame:

Any 8-consecutive week period after randomization

Safety Issue:

No

Authority:

United States: Food and Drug Administration

Study ID:

P02978

NCT ID:

NCT00109538

Start Date:

May 2005

Completion Date:

August 2008

Related Keywords:

  • Myelodysplastic Syndromes
  • Leukemia, Myelomonocytic, Chronic
  • Myelodysplasia
  • Myelomonocytic
  • Lonafarnib
  • Leukemia
  • Leukemia, Myelomonocytic, Chronic
  • Myelodysplastic Syndromes
  • Preleukemia
  • Leukemia, Myelomonocytic, Acute

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