This study has been terminated.
| Sponsored by: | Schering-Plough |
| Information provided by: | Schering-Plough |
| ClinicalTrials.gov Identifier: | NCT00109538 |
PurposeThe purpose of this study is to assess the benefit of lonafarnib (versus placebo) in patients with myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). Benefit will be measured by achievement of platelet transfusion independence for at least 8-consecutive weeks, and without simultaneous worsening of hemoglobin and/or need for red blood cell (RBC) transfusion. Additional endpoints will be hematologic response (which includes complete remission, partial remission, hematologic improvement), number of RBC transfusions, bleeding events, infections and safety.
| Condition | Intervention | Phase |
| Myelodysplastic Syndromes Leukemia, Myelomonocytic, Chronic Myelodysplasia Myelomonocytic | Drug: Lonafarnib Other: Placebo | Phase III |
| Genetics Home Reference related topics: | Bone Marrow Diseases |
| MedlinePlus related topics: | Blood Transfusion and Donation Leukemia, Adult Acute Leukemia, Adult Chronic |
| ChemIDplus related topics: | Lonafarnib |
U.S. FDA Resources
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Double Blind (Subject, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
| Official Title: | 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 |
Further study details as provided by Schering-Plough:
Primary Outcome Measures:
- 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. [ Time Frame: Any 8-consecutive week period after randomization ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Hematologic response rate (CR, PR, HI), number of RBC transfusion events during a 4-week period, active bleeding events (number and severity), number of CTCAE Grade 3 and 4 infections and days of acute intervention, and safety. [ Time Frame: Any 8-consecutive week period after randomization ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 200 |
| Study Start Date: | May 2005 |
| Estimated Study Completion Date: | January 2012 |
| Estimated Primary Completion Date: | January 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
| Lonafarnib: Experimental Lonafarnib 200 mg twice daily, oral, continuously | Drug: Lonafarnib 200 mg twice daily (BID, ie, approximately 12 hours apart with food), oral, continuously, or until unacceptable toxicity or transformation to AML, or disease progression, or other discontinuation criteria |
| Placebo: Placebo Comparator Placebo, BID, oral | Other: Placebo BID, oral, continuously, or until unacceptable toxicity or transformation to AML, or disease progression, or other discontinuation criteria |
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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.
Contacts and LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT00109538
Sponsors and Collaborators
Investigators
 | Show 46 Study Locations |
Sponsors and Collaborators
| Schering-Plough |
Investigators
| Study Director: | Antoine Yver, MD, MSc | Schering-Plough |