PLX3397 Phase 3 Study for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS) (ENLIVEN)
This is a Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug PLX3397 in the treatment of tumour of pigmented villonodular synovitis or giant cell tumor of the tendon sheath in subjects, for whom surgical removal of the tumour would cause more harm than good. The main purpose of this study is to gather information about the investigational drug PLX3397, which may help to treat these tumours.
The study consists of two parts. In Part 1, eligible study participants will be assigned to receive either PLX3397 or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumour to the treatment which will be independently assessed by central readers blinded to the treatment assignment. Those subjects, whether assigned to PLX3397 or matching placebo, who have completed Part 1 (i.e, complete 24 weeks of dosing and the Week 25 assessments) will be eligible to advance to Part 2, a long-term treatment phase in which all subjects will receive open-label PLX3397
|Pigmented Villonodular Synovitis Giant Cell Tumors of the Tendon Sheath Tenosynovial Giant Cell Tumour||Drug: PLX3397 capsule Drug: Placebo capsule matching PLX3397 capsule||Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
|Official Title:||A Double-blind, Randomized, Placebo-controlled Phase 3 Study of Orally Administered PLX3397 in Subjects With Pigmented Villonodular Synovitis or Giant Cell Tumor of the Tendon Sheath|
- Percentage of participants achieving complete or partial response [ Time Frame: to the end of the trial, assessed up to an estimated total of 36 months ]The percentage of participants who achieved a complete response (CR) or partial response (PR) at the Week 25 visit based on centrally read MRI scans and RECIST 1.1.
- Mean composite score for patient reported outcomes [ Time Frame: to the end of the trial, assessed up to an estimated total of 36 months ]Evaluate composite patient-reported outcomes (PROs), including the Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) item, Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Scale, and Worst Stiffness NRS item, at Week 25
- Number of responders based on Tumor Volume Score [ Time Frame: to the end of the trial, assessed up to an estimated total of 36 months ]Response based on Tumor Volume Score (TVS)
- Mean range of motion [ Time Frame: to the end of the trial, assessed up to an estimated total of 36 months ]
- Mean duration of response. [ Time Frame: to the end of the trial, assessed up to an estimated total of 36 months ]
|Study Start Date:||March 2015|
|Estimated Study Completion Date:||March 2018|
|Estimated Primary Completion Date:||March 2018 (Final data collection date for primary outcome measure)|
1000 mg per day for 2 weeks (dose split over morning and evening), then 800 mg per day for 22 weeks (dose split over morning and evening).
Drug: PLX3397 capsule
Each capsule of PLX3397 will contain 200 mg of PLX3397 HCl
Placebo Comparator: Placebo
1000 mg matching placebo per day for 2 weeks (dose split over morning and evening), then up to 800 mg placebo per day for 22 weeks (dose split over morning and evening).
Drug: Placebo capsule matching PLX3397 capsule
Placebo capsule matching PLX3397 capsule
Please refer to this study by its ClinicalTrials.gov identifier: NCT02371369
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