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NCT03389802 · OUTCOME

Incidence of Anti-APX005M Antibodies

Phase I Study of APX005M in Pediatric Central Nervous System Tumors · Source last updated 2026-08-13

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

Measure: NUMBER
Unit: percentage of patients with positive ADA
Interval / dispersion: 95% Confidence Interval
Time frame: Prior to dosing on courses 1, 2, 3, 4, 7, 10, 14, 18, 22, 26, 30, and 34 and at the end of therapy (up to end of course 36)

What was measured

Serial blood samples for anti-drug-antibodies (ADA) were to be collected prior to dosing on courses 1, 2, 3, and 4, then every third course (courses 7 and 10), and then every 4 courses (courses 14, 18, 22, 26, 30, 34) until the end of therapy visit and collected into serum tubes. Percentages of patients with positive anti-drug antibodies with a 95% Blyth-Still-Casella confidence interval are reported by time point.

Analysis population: Ineligible patients were excluded from these analyses.

Groups in this outcome

Stratum 1, Dose Level 1

Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.1 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

Stratum 1, Dose Level 2

Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

Stratum 1, Dose Level 3

Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

Stratum 2, Dose Level 2

Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

Stratum 2, Dose Level 3

Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

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
Stratum 1, Dose Level 1	3
Stratum 1, Dose Level 2	3
Stratum 1, Dose Level 3	12
Stratum 2, Dose Level 2	6
Stratum 2, Dose Level 3	5

Reported measurements

Prior to Course 1

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	2
Stratum 1, Dose Level 2	3
Stratum 1, Dose Level 3	12
Stratum 2, Dose Level 2	6
Stratum 2, Dose Level 3	4

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 1	0	Not reported	0	77.64	Not reported
Stratum 1, Dose Level 2	0	Not reported	0	63.16	Not reported
Stratum 1, Dose Level 3	8.33	Not reported	0.43	34.89	Not reported
Stratum 2, Dose Level 2	0	Not reported	0	40.19	Not reported
Stratum 2, Dose Level 3	0	Not reported	0	52.71	Not reported

Prior to Course 2

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	3
Stratum 1, Dose Level 2	3
Stratum 1, Dose Level 3	11
Stratum 2, Dose Level 2	6
Stratum 2, Dose Level 3	2

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 1	33.33	Not reported	1.70	86.46	Not reported
Stratum 1, Dose Level 2	0	Not reported	0	63.16	Not reported
Stratum 1, Dose Level 3	0	Not reported	0	24.95	Not reported
Stratum 2, Dose Level 2	16.67	Not reported	0.85	59.81	Not reported
Stratum 2, Dose Level 3	0	Not reported	0	77.64	Not reported

Prior to Course 3

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	1
Stratum 1, Dose Level 2	3
Stratum 1, Dose Level 3	4
Stratum 2, Dose Level 2	4
Stratum 2, Dose Level 3	2

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 1	100	Not reported	50.0	100.0	Not reported
Stratum 1, Dose Level 2	0	Not reported	0	63.16	Not reported
Stratum 1, Dose Level 3	50.0	Not reported	9.76	90.24	Not reported
Stratum 2, Dose Level 2	50.0	Not reported	9.76	90.24	Not reported
Stratum 2, Dose Level 3	0	Not reported	0	77.64	Not reported

Prior to Course 4

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	1
Stratum 1, Dose Level 2	1
Stratum 1, Dose Level 3	4
Stratum 2, Dose Level 2	4
Stratum 2, Dose Level 3	1

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 1	100	Not reported	50.0	100.0	Not reported
Stratum 1, Dose Level 2	100	Not reported	50.0	100.0	Not reported
Stratum 1, Dose Level 3	50.0	Not reported	9.76	90.24	Not reported
Stratum 2, Dose Level 2	75	Not reported	24.86	98.73	Not reported
Stratum 2, Dose Level 3	0	Not reported	0	95.0	Not reported

Prior to Course 7

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	0
Stratum 1, Dose Level 2	1
Stratum 1, Dose Level 3	2
Stratum 2, Dose Level 2	1
Stratum 2, Dose Level 3	1

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 2	100	Not reported	50.0	100.0	Not reported
Stratum 1, Dose Level 3	50	Not reported	2.52	97.47	Not reported
Stratum 2, Dose Level 2	100	Not reported	50.0	100.0	Not reported
Stratum 2, Dose Level 3	0	Not reported	0	95.0	Not reported

Prior to Course 10

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	0
Stratum 1, Dose Level 2	0
Stratum 1, Dose Level 3	2
Stratum 2, Dose Level 2	1
Stratum 2, Dose Level 3	0

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 3	50	Not reported	2.52	97.47	Not reported
Stratum 2, Dose Level 2	100	Not reported	50.0	100.0	Not reported

Prior to Course 14

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	0
Stratum 1, Dose Level 2	0
Stratum 1, Dose Level 3	1
Stratum 2, Dose Level 2	1
Stratum 2, Dose Level 3	0

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 3	100	Not reported	50.0	100.0	Not reported
Stratum 2, Dose Level 2	100	Not reported	50.0	100.0	Not reported

Prior to Course 18

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	0
Stratum 1, Dose Level 2	0
Stratum 1, Dose Level 3	1
Stratum 2, Dose Level 2	0
Stratum 2, Dose Level 3	0

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 3	100	Not reported	50.0	100.0	Not reported

Prior to Course 22

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	0
Stratum 1, Dose Level 2	0
Stratum 1, Dose Level 3	1
Stratum 2, Dose Level 2	0
Stratum 2, Dose Level 3	0

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 3	0	Not reported	0	95.0	Not reported

Prior to Course 26

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	0
Stratum 1, Dose Level 2	0
Stratum 1, Dose Level 3	1
Stratum 2, Dose Level 2	0
Stratum 2, Dose Level 3	0

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 3	0	Not reported	0	95.0	Not reported

Prior to Course 30

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	0
Stratum 1, Dose Level 2	0
Stratum 1, Dose Level 3	0
Stratum 2, Dose Level 2	0
Stratum 2, Dose Level 3	0

Prior to Course 34

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	0
Stratum 1, Dose Level 2	0
Stratum 1, Dose Level 3	0
Stratum 2, Dose Level 2	0
Stratum 2, Dose Level 3	0

End of Treatment

Analysis denominator · Participants

Group	Source count
Stratum 1, Dose Level 1	2
Stratum 1, Dose Level 2	0
Stratum 1, Dose Level 3	6
Stratum 2, Dose Level 2	3
Stratum 2, Dose Level 3	4

Values in percentage of patients with positive ADA · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Stratum 1, Dose Level 1	100	Not reported	22.36	100	Not reported
Stratum 1, Dose Level 3	33.33	Not reported	6.29	72.87	Not reported
Stratum 2, Dose Level 2	66.67	Not reported	13.54	98.30	Not reported
Stratum 2, Dose Level 3	0	Not reported	0	52.71	Not reported

Complete source fields

classes

categories
measurements
group Id
OG000
lower Limit
0
upper Limit
77.64
value
0
group Id
OG001
lower Limit
0
upper Limit
63.16
value
0
group Id
OG002
lower Limit
0.43
upper Limit
34.89
value
8.33
group Id
OG003
lower Limit
0
upper Limit
40.19
value
0
group Id
OG004
lower Limit
0
upper Limit
52.71
value
0
denoms
counts
group Id
OG000
value
2
group Id
OG001
value
3
group Id
OG002
value
12
group Id
OG003
value
6
group Id
OG004
value
4
units
Participants
title
Prior to Course 1
categories
measurements
group Id
OG000
lower Limit
1.70
upper Limit
86.46
value
33.33
group Id
OG001
lower Limit
0
upper Limit
63.16
value
0
group Id
OG002
lower Limit
0
upper Limit
24.95
value
0
group Id
OG003
lower Limit
0.85
upper Limit
59.81
value
16.67
group Id
OG004
lower Limit
0
upper Limit
77.64
value
0
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
11
group Id
OG003
value
6
group Id
OG004
value
2
units
Participants
title
Prior to Course 2
categories
measurements
group Id
OG000
lower Limit
50.0
upper Limit
100.0
value
100
group Id
OG001
lower Limit
0
upper Limit
63.16
value
0
group Id
OG002
lower Limit
9.76
upper Limit
90.24
value
50.0
group Id
OG003
lower Limit
9.76
upper Limit
90.24
value
50.0
group Id
OG004
lower Limit
0
upper Limit
77.64
value
0
denoms
counts
group Id
OG000
value
1
group Id
OG001
value
3
group Id
OG002
value
4
group Id
OG003
value
4
group Id
OG004
value
2
units
Participants
title
Prior to Course 3
categories
measurements
group Id
OG000
lower Limit
50.0
upper Limit
100.0
value
100
group Id
OG001
lower Limit
50.0
upper Limit
100.0
value
100
group Id
OG002
lower Limit
9.76
upper Limit
90.24
value
50.0
group Id
OG003
lower Limit
24.86
upper Limit
98.73
value
75
group Id
OG004
lower Limit
0
upper Limit
95.0
value
0
denoms
counts
group Id
OG000
value
1
group Id
OG001
value
1
group Id
OG002
value
4
group Id
OG003
value
4
group Id
OG004
value
1
units
Participants
title
Prior to Course 4
categories
measurements
group Id
OG001
lower Limit
50.0
upper Limit
100.0
value
100
group Id
OG002
lower Limit
2.52
upper Limit
97.47
value
50
group Id
OG003
lower Limit
50.0
upper Limit
100.0
value
100
group Id
OG004
lower Limit
0
upper Limit
95.0
value
0
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
1
group Id
OG002
value
2
group Id
OG003
value
1
group Id
OG004
value
1
units
Participants
title
Prior to Course 7
categories
measurements
group Id
OG002
lower Limit
2.52
upper Limit
97.47
value
50
group Id
OG003
lower Limit
50.0
upper Limit
100.0
value
100
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
2
group Id
OG003
value
1
group Id
OG004
value
0
units
Participants
title
Prior to Course 10
categories
measurements
group Id
OG002
lower Limit
50.0
upper Limit
100.0
value
100
group Id
OG003
lower Limit
50.0
upper Limit
100.0
value
100
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
1
group Id
OG003
value
1
group Id
OG004
value
0
units
Participants
title
Prior to Course 14
categories
measurements
group Id
OG002
lower Limit
50.0
upper Limit
100.0
value
100
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
1
group Id
OG003
value
0
group Id
OG004
value
0
units
Participants
title
Prior to Course 18
categories
measurements
group Id
OG002
lower Limit
0
upper Limit
95.0
value
0
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
1
group Id
OG003
value
0
group Id
OG004
value
0
units
Participants
title
Prior to Course 22
categories
measurements
group Id
OG002
lower Limit
0
upper Limit
95.0
value
0
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
1
group Id
OG003
value
0
group Id
OG004
value
0
units
Participants
title
Prior to Course 26
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
0
group Id
OG003
value
0
group Id
OG004
value
0
units
Participants
title
Prior to Course 30
denoms
counts
group Id
OG000
value
0
group Id
OG001
value
0
group Id
OG002
value
0
group Id
OG003
value
0
group Id
OG004
value
0
units
Participants
title
Prior to Course 34

denoms

counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
12
group Id
OG003
value
6
group Id
OG004
value
5
units
Participants

description

Serial blood samples for anti-drug-antibodies (ADA) were to be collected prior to dosing on courses 1, 2, 3, and 4, then every third course (courses 7 and 10), and then every 4 courses (courses 14, 18, 22, 26, 30, 34) until the end of therapy visit and collected into serum tubes. Percentages of patients with positive anti-drug antibodies with a 95% Blyth-Still-Casella confidence interval are reported by time point.

dispersion Type

95% Confidence Interval

groups

description
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.1 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG000
title
Stratum 1, Dose Level 1
description
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG001
title
Stratum 1, Dose Level 2
description
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG002
title
Stratum 1, Dose Level 3
description
Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG003
title
Stratum 2, Dose Level 2
description
Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG004
title
Stratum 2, Dose Level 3

param Type

NUMBER

population Description

Ineligible patients were excluded from these analyses.

reporting Status

POSTED

time Frame

Prior to dosing on courses 1, 2, 3, 4, 7, 10, 14, 18, 22, 26, 30, and 34 and at the end of therapy (up to end of course 36)

title

Incidence of Anti-APX005M Antibodies

type

OTHER_PRE_SPECIFIED

unit Of Measure

percentage of patients with positive ADA

Download exact source JSON → · Snapshot ctgov-results-25a395029948b04d52b1253f

Preserved source evidence · Not medical advice