Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score
Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS) · Source last updated 2022-05-11
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
Measure
NUMBER
Unit
participants
Interval / dispersion
Not reported
Time frame
By Week 120
What was measured
Tumor Volume Score (TVS) is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. The overall number of responses and the number of participants with and without disease progression was assessed.
Analysis population: The overall number of responses and the number of participants with and without disease progression was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only were not analyzed.
Groups in this outcome
Pexidartinib in Part 1 and Part 2
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
Placebo Part 1, Pexidartinib Part 2
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
All Pexidartinib Treated
All participants who received pexidartinib in Part 1 and Part 2 as well as those who received pexidartinib in Part 2 only.
Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.
Analysis denominator · Participants
Group
Source count
Pexidartinib in Part 1 and Part 2
61
Placebo Part 1, Pexidartinib Part 2
30
All Pexidartinib Treated
91
Reported measurements
Number of responders
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
34
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
18
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
52
Not reported
Not reported
Not reported
Not reported
Week 12 (Day 84); Without disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
33
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
18
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
51
Not reported
Not reported
Not reported
Not reported
Week 12 (Day 84); With disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
0
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
0
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
0
Not reported
Not reported
Not reported
Not reported
Week 24 (Day 168); Without disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
32
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
18
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
50
Not reported
Not reported
Not reported
Not reported
Week 24 (Day 168); With disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
0
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
0
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
0
Not reported
Not reported
Not reported
Not reported
Week 48 (Day 336); Without disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
22
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
13
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
35
Not reported
Not reported
Not reported
Not reported
Week 48 (Day 336); With disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
3
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
1
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
4
Not reported
Not reported
Not reported
Not reported
Week 72 (Day 504); Without disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
13
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
3
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
16
Not reported
Not reported
Not reported
Not reported
Week 72 (Day 504); With disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
3
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
1
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
4
Not reported
Not reported
Not reported
Not reported
Week 96 (Day 672); Without disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
3
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
1
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
4
Not reported
Not reported
Not reported
Not reported
Week 96 (Day 672); With disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
3
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
1
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
4
Not reported
Not reported
Not reported
Not reported
Week 120 (Day 840); Without disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
1
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
0
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
1
Not reported
Not reported
Not reported
Not reported
Week 120 (Day 840); With disease progression
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Pexidartinib in Part 1 and Part 2
3
Not reported
Not reported
Not reported
Not reported
Placebo Part 1, Pexidartinib Part 2
1
Not reported
Not reported
Not reported
Not reported
All Pexidartinib Treated
4
Not reported
Not reported
Not reported
Not reported
Complete source fields
classes
categories
measurements
group Id
OG000
value
34
group Id
OG001
value
18
group Id
OG002
value
52
title
Number of responders
categories
measurements
group Id
OG000
value
33
group Id
OG001
value
18
group Id
OG002
value
51
title
Week 12 (Day 84); Without disease progression
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
0
title
Week 12 (Day 84); With disease progression
categories
measurements
group Id
OG000
value
32
group Id
OG001
value
18
group Id
OG002
value
50
title
Week 24 (Day 168); Without disease progression
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
0
title
Week 24 (Day 168); With disease progression
categories
measurements
group Id
OG000
value
22
group Id
OG001
value
13
group Id
OG002
value
35
title
Week 48 (Day 336); Without disease progression
categories
measurements
group Id
OG000
value
3
group Id
OG001
value
1
group Id
OG002
value
4
title
Week 48 (Day 336); With disease progression
categories
measurements
group Id
OG000
value
13
group Id
OG001
value
3
group Id
OG002
value
16
title
Week 72 (Day 504); Without disease progression
categories
measurements
group Id
OG000
value
3
group Id
OG001
value
1
group Id
OG002
value
4
title
Week 72 (Day 504); With disease progression
categories
measurements
group Id
OG000
value
3
group Id
OG001
value
1
group Id
OG002
value
4
title
Week 96 (Day 672); Without disease progression
categories
measurements
group Id
OG000
value
3
group Id
OG001
value
1
group Id
OG002
value
4
title
Week 96 (Day 672); With disease progression
categories
measurements
group Id
OG000
value
1
group Id
OG001
value
0
group Id
OG002
value
1
title
Week 120 (Day 840); Without disease progression
denoms
counts
group Id
OG000
value
61
group Id
OG001
value
30
group Id
OG002
value
91
units
Participants
description
Tumor Volume Score (TVS) is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. The overall number of responses and the number of participants with and without disease progression was assessed.
groups
description
Participants received treatment of Pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Pexidartinib: Each capsule contains 200 mg of pexidartinib for oral administration
id
OG000
title
Pexidartinib in Part 1 and Part 2
description
Participants received treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks in Part 1 and also received Pexidartinib in Part 2 at their prescribed dose.
Placebo: Placebo capsule matching Pexidartinib capsule for oral administration Pexidartinib: Each capsule contains 200 mg of Pexidartinib for oral administration.
id
OG001
title
Placebo Part 1, Pexidartinib Part 2
description
All participants who received pexidartinib in Part 1 and Part 2 as well as those who received pexidartinib in Part 2 only.
id
OG002
title
All Pexidartinib Treated
param Type
NUMBER
population Description
The overall number of responses and the number of participants with and without disease progression was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Pexidartinib Part 2 only; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only were not analyzed.
reporting Status
POSTED
time Frame
By Week 120
title
Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score
Triangle