A Study to Evaluate the Efficacy and Safety of Vismodegib in Combination With Ruxolitinib for the Treatment of Intermediate- or High-Risk Myelofibrosis (MF)

Part of paid clinical trials in Fort Myers, Florida.

Sponsor
Hoffmann-La Roche
Study ID
NCT02593760
Phase
PHASE1
Status
Completed

Conditions

  • Myelofibrosis

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Placebo — OTHER
    Placebo will be administered PO QD for up to 48 weeks.
  • Ruxolitinib — DRUG
    Ruxolitinib will be administered PO BID at a starting dose depending on the participants's baseline platelet count for up to 48 weeks.
  • Vismodegib — DRUG
    Vismodegib will be administered at a dose of 150 mg PO QD for up to 48 weeks.

Study Details

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.

Key Dates

Start date
Jan 25, 2016
Status verified
May 2018
Primary completion
Mar 29, 2017
Completion
Jul 12, 2017

Study Design

Enrollment
10 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Active Comparator: Placebo + Ruxolitinib
    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.
  • Experimental: Vismodegib + 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.

Primary Outcome Measure

Percentage of Participants who Achieve a Greater Than or Equal to (>=) 35% Reduction in Spleen Volume from Baseline at Week 24 [ Time Frame: Week 24 ]

Locations (6)

FacilityCityStateZIPSite coordinators
Florida Cancer Specialists-Broadway, Fort MyersFort MyersFlorida33908-
Florida Cancer Specialist, North RegionSt. PetersburgFlorida33705-
Florida Cancer SpecialistsWest Palm BeachFlorida33401-
Oncology Hematology Care IncCincinnatiOhio45242-
Sarah Cannon Research InstituteNashvilleTennessee37203-
Uni of Texas - Md Anderson Cancer Center; Dept of LeukemiaHoustonTexas77030-

Find similar trials in Fort Myers, FL

By research site
Florida Cancer Specialists-Broadway, Fort Myers· Fort Myers, FLFlorida Cancer Specialist, North Region· St. Petersburg, FLFlorida Cancer Specialists· West Palm Beach, FLOncology Hematology Care Inc· Cincinnati, OHSarah Cannon Research Institute· Nashville, TNUni of Texas - Md Anderson Cancer Center; Dept of Leukemia· Houston, TX

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