NCT02371369 · OUTCOME

Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49

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: MEAN
Unit: units on a scale
Interval / dispersion: Standard Deviation
Time frame: Baseline, Week 25, and Week 49

What was measured

The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).

Analysis population: Worst stiffness was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Placebo Part 1, Pexidartinib Part 2; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only or Pexidartinib Part 2 only were not analyzed.

Groups in this outcome

Pexidartinib 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 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 only received Pexidartinib in Part 2.

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 Part 1 and Part 2	61
Placebo Part 1, Pexidartinib Part 2	30
All Pexidartinib Treated	91

Reported measurements

Baseline

Analysis denominator · Participants

Group	Source count
Pexidartinib Part 1 and Part 2	59
Placebo Part 1, Pexidartinib Part 2	26
All Pexidartinib Treated	85

Values in units on a scale · Interval/dispersion: Standard Deviation
Group	Value	Spread	Lower	Upper	Comment
Pexidartinib Part 1 and Part 2	5.6	1.7	Not reported	Not reported	Not reported
Placebo Part 1, Pexidartinib Part 2	5.7	2.3	Not reported	Not reported	Not reported
All Pexidartinib Treated	5.6	1.9	Not reported	Not reported	Not reported

Week 25

Analysis denominator · Participants

Group	Source count
Pexidartinib Part 1 and Part 2	33
Placebo Part 1, Pexidartinib Part 2	18
All Pexidartinib Treated	51

Values in units on a scale · Interval/dispersion: Standard Deviation
Group	Value	Spread	Lower	Upper	Comment
Pexidartinib Part 1 and Part 2	-2.7	2.2	Not reported	Not reported	Not reported
Placebo Part 1, Pexidartinib Part 2	-3.0	3.1	Not reported	Not reported	Not reported
All Pexidartinib Treated	-2.8	2.5	Not reported	Not reported	Not reported

Week 49

Analysis denominator · Participants

Group	Source count
Pexidartinib Part 1 and Part 2	22
Placebo Part 1, Pexidartinib Part 2	10
All Pexidartinib Treated	32

Values in units on a scale · Interval/dispersion: Standard Deviation
Group	Value	Spread	Lower	Upper	Comment
Pexidartinib Part 1 and Part 2	-3.5	1.9	Not reported	Not reported	Not reported
Placebo Part 1, Pexidartinib Part 2	-2.2	2.8	Not reported	Not reported	Not reported
All Pexidartinib Treated	-3.1	2.3	Not reported	Not reported	Not reported

Complete source fields

classes

categories
measurements
group Id
OG000
spread
1.7
value
5.6
group Id
OG001
spread
2.3
value
5.7
group Id
OG002
spread
1.9
value
5.6
denoms
counts
group Id
OG000
value
59
group Id
OG001
value
26
group Id
OG002
value
85
units
Participants
title
Baseline
categories
measurements
group Id
OG000
spread
2.2
value
-2.7
group Id
OG001
spread
3.1
value
-3.0
group Id
OG002
spread
2.5
value
-2.8
denoms
counts
group Id
OG000
value
33
group Id
OG001
value
18
group Id
OG002
value
51
units
Participants
title
Week 25
categories
measurements
group Id
OG000
spread
1.9
value
-3.5
group Id
OG001
spread
2.8
value
-2.2
group Id
OG002
spread
2.3
value
-3.1
denoms
counts
group Id
OG000
value
22
group Id
OG001
value
10
group Id
OG002
value
32
units
Participants
title
Week 49

denoms

counts
group Id
OG000
value
61
group Id
OG001
value
30
group Id
OG002
value
91
units
Participants

description

dispersion Type

Standard Deviation

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 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 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 only received Pexidartinib in Part 2.
id
OG002
title
All Pexidartinib Treated

param Type

MEAN

population Description

Worst stiffness was assessed in the ITT population, specifically among the Pexidartinib Part 1, Pexidartinib Part 2; Placebo Part 1, Pexidartinib Part 2; and All Pexidartinib Treated participants. Participants who received Placebo Part 1 only or Pexidartinib Part 2 only were not analyzed.

reporting Status

POSTED

time Frame

Baseline, Week 25, and Week 49

title

Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49

type

OTHER_PRE_SPECIFIED

unit Of Measure

units on a scale

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice