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NCT03389802 · OUTCOME

Tumor Mutation Burden Based on Whole Exome Sequencing of Tumor Tissue

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
MEAN
Unit
mutations per megabase
Interval / dispersion
Standard Deviation
Time frame
Day 0 of treatment

What was measured

Tumor mutation burden was defined as the number of somatic mutations per megabase. Average tumor mutation burden was reported with a standard deviation. When only one patient was analyzed, the standard deviation was reported as 0 rather than undefined.

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

GroupSource count
Stratum 1, Dose Level 13
Stratum 1, Dose Level 23
Stratum 1, Dose Level 311
Stratum 2, Dose Level 21
Stratum 2, Dose Level 32

Reported measurements

Source class 1
Values in mutations per megabase · Interval/dispersion: Standard Deviation
GroupValueSpreadLowerUpperComment
Stratum 1, Dose Level 12.621.03Not reportedNot reportedNot reported
Stratum 1, Dose Level 23.822.79Not reportedNot reportedNot reported
Stratum 1, Dose Level 33.952.34Not reportedNot reportedNot reported
Stratum 2, Dose Level 20.970.0Not reportedNot reportedNot reported
Stratum 2, Dose Level 34.835.94Not reportedNot reportedNot reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        spread
        1.03
        value
        2.62
      2. group Id
        OG001
        spread
        2.79
        value
        3.82
      3. group Id
        OG002
        spread
        2.34
        value
        3.95
      4. group Id
        OG003
        spread
        0.0
        value
        0.97
      5. group Id
        OG004
        spread
        5.94
        value
        4.83
denoms
  1. counts
    1. group Id
      OG000
      value
      3
    2. group Id
      OG001
      value
      3
    3. group Id
      OG002
      value
      11
    4. group Id
      OG003
      value
      1
    5. group Id
      OG004
      value
      2
    units
    Participants
description
Tumor mutation burden was defined as the number of somatic mutations per megabase. Average tumor mutation burden was reported with a standard deviation. When only one patient was analyzed, the standard deviation was reported as 0 rather than undefined.
dispersion Type
Standard Deviation
groups
  1. 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
  2. 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
  3. 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
  4. 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
  5. 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
MEAN
reporting Status
POSTED
time Frame
Day 0 of treatment
title
Tumor Mutation Burden Based on Whole Exome Sequencing of Tumor Tissue
type
OTHER_PRE_SPECIFIED
unit Of Measure
mutations per megabase

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

Preserved source evidence · Not medical advice