NCT02989857 · OUTCOME

Progression Free Survival (PFS) as Determined by the Independent Radiology Committee (IRC)

Study of AG-120 in Previously Treated Advanced Cholangiocarcinoma With IDH1 Mutations (ClarIDHy) · Source last updated 2024-08-20

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure: MEDIAN
Unit: months
Interval / dispersion: 95% Confidence Interval
Time frame: From the date of randomization to the date of first documentation of disease progression or death due to any cause (Up to approximately 2 years)

What was measured

PFS is defined as the time from date of randomization to the date of first documented disease progression as assessed by the IRC using Response Evaluation Criteria in Solid Tumors \[RECIST\] v1.1, or date of death due to any cause, whichever occurred first. Disease progression was defined as greater than or equal to (≥)20 percent (%) increase in sum of the diameter of target lesions, taking as reference the smallest sum diameter recorded since the treatment started. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 millimeters (mm) or the appearance of 1 or more new lesions.

Analysis population: ITT set included all participants who were randomized, with the treatment group designated according to the randomization. Number analyzed is the number of participants with data available for analyses at the specified time point. Two participants were excluded from the analysis as they were randomized in the study after the data cutoff date (31 January 2019) for the analysis of PFS.

Groups in this outcome

AG-120

Participants received AG-120 500 mg, tablet, orally, QD in each 28-day treatment cycle, until occurrence of disease progression, unacceptable toxicity, confirmed pregnancy, death, subject withdrawal, lost to follow-up, or the sponsor ended the study for up to approximately 45 months.

Placebo

Participants received AG-120 matched placebo, orally, QD in each 28-day treatment cycle, until occurrence of disease progression, unacceptable toxicity, confirmed pregnancy, death, subject withdrawal, lost to follow-up or the sponsor ended the study for up to approximately 7 months. Participants who experienced disease progression and received placebo were allowed to cross over and receive AG-120.

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
AG-120	124
Placebo	61

Reported measurements

Source class 1

Values in months · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
AG-120	2.7	Not reported	1.6	4.2	Not reported
Placebo	1.4	Not reported	1.4	1.6	Not reported

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

ci Lower Limit
0.25
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
0.54
estimate Comment
Hazard ratio was calculated from stratified Cox regression model with placebo as the denominator, with two-sided 95% confidence interval.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
<0.0001
p Value Comment
P-value was calculated from the one-sided stratified log-rank test.
param Type
Hazard Ratio (HR)
param Value
0.37
statistical Method
Log Rank

Complete source fields

analyses

ci Lower Limit
0.25
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
0.54
estimate Comment
Hazard ratio was calculated from stratified Cox regression model with placebo as the denominator, with two-sided 95% confidence interval.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
<0.0001
p Value Comment
P-value was calculated from the one-sided stratified log-rank test.
param Type
Hazard Ratio (HR)
param Value
0.37
statistical Method
Log Rank

classes

categories
measurements
group Id
OG000
lower Limit
1.6
upper Limit
4.2
value
2.7
group Id
OG001
lower Limit
1.4
upper Limit
1.6
value
1.4

denoms

counts
group Id
OG000
value
124
group Id
OG001
value
61
units
Participants

description

dispersion Type

95% Confidence Interval

groups

description
Participants received AG-120 500 mg, tablet, orally, QD in each 28-day treatment cycle, until occurrence of disease progression, unacceptable toxicity, confirmed pregnancy, death, subject withdrawal, lost to follow-up, or the sponsor ended the study for up to approximately 45 months.
id
OG000
title
AG-120
description
Participants received AG-120 matched placebo, orally, QD in each 28-day treatment cycle, until occurrence of disease progression, unacceptable toxicity, confirmed pregnancy, death, subject withdrawal, lost to follow-up or the sponsor ended the study for up to approximately 7 months. Participants who experienced disease progression and received placebo were allowed to cross over and receive AG-120.
id
OG001
title
Placebo

param Type

MEDIAN

population Description

ITT set included all participants who were randomized, with the treatment group designated according to the randomization. Number analyzed is the number of participants with data available for analyses at the specified time point. Two participants were excluded from the analysis as they were randomized in the study after the data cutoff date (31 January 2019) for the analysis of PFS.

reporting Status

POSTED

time Frame

From the date of randomization to the date of first documentation of disease progression or death due to any cause (Up to approximately 2 years)

title

Progression Free Survival (PFS) as Determined by the Independent Radiology Committee (IRC)

type

PRIMARY

unit Of Measure

months

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

Preserved source evidence · Not medical advice