Ribavirin and Hedgehog Inhibitor With or Without Decitabine in AML

Phase 2

Completed

• Conditions: Acute Myeloid Leukemia
• Interventions: Drug: Ribavirin; Drug: Vismodegib; Drug: Decitabine

• Registration Number: NCT02073838
• Lead Sponsor: Sarit Assouline

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-02-21
• Study First Submitted QC: 2014-02-25
• Study First Posted: 2014-02-27
• Study Start: 2015-05
• Primary Completion: 2022-11
• Study Completion: 2022-11
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-28
• Last Update Posted: 2023-09-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Ribavirin, vismodegib	Ribavirin	Ribavirin 1400mg BID, vismodegib 150mg QD
Ribavirin, vismodegib, decitabine	Vismodegib	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.
Ribavirin, vismodegib, decitabine	Ribavirin	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.
Ribavirin, vismodegib	Decitabine	Ribavirin 1400mg BID, vismodegib 150mg QD
Ribavirin, vismodegib, decitabine	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.

Primary Outcome Measures

Name	Time	Method
Efficacy will be measured by overall response rate (ORR).	Measured up to 2 years after the last subject has enrolled in the study.

Secondary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Jewish General Hospital; 🇨🇦 Montreal, Quebec, Canada