A Phase II, Multi-center, Open Label, Randomized Study of Ribavirin and Hedgehog Inhibitor With or Without Decitabine in Acute Myeloid Leukemia (AML)
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Enrollment
- 23
- Locations
- 1
- Primary Endpoint
- Efficacy will be measured by overall response rate (ORR).
Study Overview
Brief Summary
This is a research study of ribavirin which will be given in combination with vismodegib and/or decitabine. The purpose of this study is to see if patients respond to treatment when ribavirin is given with vismodegib alone or in combination with decitabine.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Ribavirin, vismodegib
Ribavirin 1400mg BID, vismodegib 150mg QD
Intervention: Ribavirin (Drug)
Ribavirin, vismodegib
Ribavirin 1400mg BID, vismodegib 150mg QD
Intervention: Decitabine (Drug)
Ribavirin, vismodegib, decitabine
Decitabine 20mg/m2 IV QD days -7 to -3 for cycle 1. Ribavirin 1400mg BID and vismodegib 150mg QD starting on day 1. On subsequent cycles, decitabine will be administered on days 1 to 5.
Intervention: Ribavirin (Drug)
Ribavirin, vismodegib, decitabine
Decitabine 20mg/m2 IV QD days -7 to -3 for cycle 1. Ribavirin 1400mg BID and vismodegib 150mg QD starting on day 1. On subsequent cycles, decitabine will be administered on days 1 to 5.
Intervention: Vismodegib (Drug)
Ribavirin, vismodegib, decitabine
Decitabine 20mg/m2 IV QD days -7 to -3 for cycle 1. Ribavirin 1400mg BID and vismodegib 150mg QD starting on day 1. On subsequent cycles, decitabine will be administered on days 1 to 5.
Intervention: Decitabine (Drug)
Outcomes
Primary Outcomes
Efficacy will be measured by overall response rate (ORR).
Time Frame: Measured up to 2 years after the last subject has enrolled in the study.
Secondary Outcomes
- Time to response(Measured up to 2 years after the last subject has enrolled in the study.)
- Duration of response(Measured up to 2 years after the last subject has enrolled in the study.)
- One year survival(Measured up to 2 years after the last subject has enrolled in the study.)
- Overall survival(Measured up to 3 years after the last subject has enrolled in the study.)
- Hematologic improvement defined by the number of individual, positively affected cell lines (erythroid, neutrophil and platelet cells) per patient.(Measured up to 2 years after the last subject has enrolled in the study.)
- Number of participants with Adverse Events as a Measure of Safety and Tolerability(Measured up to 2 years after the last subject has enrolled in the study.)
- Changes in eIF4E expression, localization, and signalling pathways (measured by immuno-histochemical analysis, PCR or western blot) and correlating with each patient's overall response.(Measured up to 2 years after the last subject has enrolled in the study.)
Investigators
Sarit Assouline
Hematologist-oncologist
Jewish General Hospital