A Phase 1 interventional study of Placebo and Ruxolitinib in Myelofibrosis, sponsored by Hoffmann-La Roche. Completed at 13 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-05-11.
Sponsored by Hoffmann-La Roche · Phase 1, Interventional, and Treatment
This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy and safety of vismodegib plus (+) ruxolitinib versus placebo + ruxolitinib in participants with intermediate- or high-risk MF. The study will be divided into 2 components. The Phase Ib portion of the study consists of participants receiving open-label vismodegib (150 milligrams [mg] orally [PO] once daily [QD]) + ruxolitinib (PO twice daily [BID]). A safety assessment will be performed after the first 10 participants have been treated for 6 weeks. An analysis for efficacy and safety is planned in the first 10 participants at Week 24. There will be a hold on participant screening and enrollment during this assessment. Another 10 participants may be enrolled, thereafter, to further assess efficacy and safety (at Week 24) before the initiation of the Phase III randomization portion of the study. Similarly, there will be another hold on participant screening and enrollment during this assessment. The participants enrolled in the Phase Ib portion of the study will continue to receive vismodegib (150 mg PO QD) + ruxolitinib (PO BID) for up to 48 weeks, if clinical benefit is observed after 24 weeks. The Phase III randomized, double-blind portion of the study will enroll approximately 84 participants. Participants will be randomly assigned in a 1:1 ratio (double blind) to receive either vismodegib (150 mg PO QD) + ruxolitinib (PO BID) or placebo (PO QD) + ruxolitinib (PO BID) for up to 48 weeks.
419 studies on the registry are indexed under Primary Myelofibrosis; 117 are open to participants now.
This study's enrollment of 10 is below the median of 44 across 347 interventional studies indexed under Primary Myelofibrosis.
Browse Primary Myelofibrosis studies →Hoffmann-La Roche is the lead sponsor of 2,061 studies on the registry; 85 are open to participants now.
Of its 319 completed or terminated interventional studies of FDA-regulated products, 239 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Participants will receive placebo (PO QD) in combination with ruxolitinib (dose will depend on the participant's baseline platelet count) for up to 48 weeks.
Other: Placebo · Drug: Ruxolitinib
Participants will receive vismodegib (150 mg PO QD) in combination with ruxolitinib (dose will depend on the participant's baseline platelet count) for up to 48 weeks.
Drug: Ruxolitinib · Drug: Vismodegib
Placebo will be administered PO QD for up to 48 weeks.
Ruxolitinib will be administered PO BID at a starting dose depending on the participants's baseline platelet count for up to 48 weeks.
Vismodegib will be administered at a dose of 150 mg PO QD for up to 48 weeks.
Also known as: Erivedge
Percentage of Participants who Achieve a Greater Than or Equal to (>=) 35% Reduction in Spleen Volume from Baseline at Week 24
Determined by an Independent Review Committee (IRC) Using International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Revised Response Criteria
Time frame: Week 24
Percentage of Participants with Complete Remission (CR) and Partial Remission (PR) at Week 24, as Determined by an IRC Using IWG-MRT Revised Response Criteria
Time frame: Week 24
Plasma Vismodegib Concentration at Steady State
Time frame: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Unbound Vismodegib Concentration at Steady State
Time frame: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Alpha 1-Acid Glycoprotein Concentration at Steady State
Time frame: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Percentage of Participants who Achieve a >= 35% Reduction in Spleen Volume from Baseline, as Determined by an IRC Using IWG-MRT Revised Response Criteria at Week 48
Time frame: Baseline, Week 48
Percentage of Participants who Achieve a >= 35% Reduction in Spleen Volume from Baseline, as Determined by an Investigator at Weeks 24 and 48
Time frame: Baseline, Weeks 24 and 48
Percentage of Participants with CR and PR, as Determined by an IRC Using IWG-MRT Revised Response Criteria at Week 48
Time frame: Week 48
Percentage of Participants with CR and PR, as Determined by an Investigator at Weeks 24 and 48
Time frame: Weeks 24 and 48
Percentage of Participants with Overall Response Rate (CR, PR, and Clinical Improvement) at Weeks 24 and 48, as Determined by an IRC Using IWG-MRT Revised Response Criteria
Time frame: Weeks 24 and 48
Percentage of Participants with Overall Response Rate (CR, PR, and Clinical Improvement) at Weeks 24 and 48, as Determined by the Investigator Using IWG-MRT Revised Response Criteria
Time frame: Weeks 24 and 48
Percentage of Participants who Achieve Anemia Response at Weeks 24 and 48, as Determined by the Investigator Using IWG-MRT Revised Response Criteria
Time frame: Weeks 24 and 48
Percentage of Participants with Symptom Response (Participants who Achieve a >= 50% Reduction from Baseline in the Myeloproliferative Neoplasm Symptom Assessment Form [MPN-SAF] Total Symptom Score [TSS])
Time frame: Baseline, Weeks 24 and 48
Duration of Response, as Determined by the Investigator and an IRC Using IWG-MRT Revised Response Criteria or Death from Any Cause During the Study
Time frame: Baseline up to 28 days after the last dose of study drug (52 weeks)
Percentage of Participants with Improvement from Baseline in Bone Marrow Fibrosis at Weeks 24 and 48, as Determined by the Investigator Using the European Consensus Grading System
Time frame: Baseline, Weeks 24 and 48
Percentage of Participants with Improvement from Baseline in Bone Marrow Fibrosis at Weeks 24 and 48, as Determined by Independent Pathology Review Using the European Consensus Grading System
Time frame: Baseline, Weeks 24 and 48
Progression-Free Survival
Time frame: Baseline up to the end of the study (up to 1 year after completing 48 weeks of treatment by the last participant)
Percentage of Participants who Achieve a >= 50% Reduction in Fatigue from Baseline to Weeks 24 and 48 as Measured by MPN-SAF TSS
Time frame: Baseline, Weeks 24 and 48
Percentage of Participants who Achieve a >= 50% Reduction in Other Symptom and Impact Item Scores from Baseline to Weeks 24 and 48, as Measured by the MPN-SAF
Time frame: Baseline, Weeks 24 and 48
Percentage of Participants who Achieve a Meaningful Improvement on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 Scale Scores from Baseline to Weeks 24 and 48
Meaningful improvement is defined as a 10-point change.
Time frame: Baseline, Weeks 24 and 48
Overall Survival
Time frame: Baseline up to the end of the study (up to 1 year after completing 48 weeks treatment by the last participant)
Percentage of Participants with Adverse Events (AEs)
Time frame: Baseline up to Month 48
This study is completed, as verified in May 2018. You cannot join it, but the record below documents what was studied.
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Hoffmann-La Roche