Skip to content
Triangle logo TriangleGet started
← Full study record

NCT03330197 · OUTCOME

The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance.

A Study of Ad-RTS-hIL-12 + Veledimex in Pediatric Subjects With Brain Tumors Including DIPG · Source last updated 2025-08-12

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

Measure
COUNT_OF_PARTICIPANTS
Unit
Participants
Interval / dispersion
Not reported
Time frame
From Day 0 through Day 56

What was measured

Dose Limiting Toxicities are defined as events that occur during the first 28 days (Day 0 - Day 28) that meets one of the following criteria: * Any local reaction that requires operative intervention and is felt to be attributable to study drug * Any local reaction that has life-threatening consequences requiring urgent intervention or results in death and is felt to be attributable to study drug * Any Grade 3 or higher non-hematologic adverse event that is at least possibly related to study drug and lasts ≥ 3 days * Nausea and vomiting will not be considered a DLT unless at least Grade 3 and refractory to antiemetics * Grade 3 or higher thrombocytopenia (\< 50,000/mm3) at least possibly related to study drug * Any Grade 4 hematologic toxicity (except thrombocytopenia) that is at least possibly related to study drug and lasts ≥ 5 days * Transient neurological changes are expected in Arm 2 and will not be considered a DLT unless they last \> 10 days

Analysis population: The Full Analysis Set (FAS) or the OSP includes every subject who received at least one dose of any study drug recorded in the Data Management (DM) database excluding screen failures. • The data analysis for Adverse Events or other Safety variables will be performed on the OSP.

Groups in this outcome

Arm 1 - Closed

Intratumoral Ad-RTS-hIL-12 freehand injection after tumor resection and oral veledimex (activator ligand) in pediatric patients with brain tumors. Ad-RTS-hIL-12: 2.0 x 10\^11 viral particles (vp) per injection, one intratumoral injection of Ad-RTS-hIL-12 Oral Veledimex - Arm 1 (Pediatric Brain Tumor): * Planned: 1 dose level (10mg/day) 15 oral daily doses of veledimex * Actual: All patients received 1 dose level (10 mg/day veledimex)

Arm 2 - Closed

Intratumoral Ad-RTS-hIL-12 stereotactic injection and oral veledimex (activator ligand) in pediatric patients with DIPG. Ad-RTS-hIL-12: 2.0 x 10\^11 viral particles (vp) per injection, one intratumoral injection of Ad-RTS-hIL-12 Oral Veledimex - Arm 2 (DIPG): * Planned:2 dose levels (10mg/day, 20mg/day) * Actual: 1 patient received 5 mg/day veledimex. The other patient did not receive veledimex due to SAE.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Arm 1 - Closed3
Arm 2 - Closed2

Reported measurements

DLTs
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Arm 1 - Closed0Not reportedNot reportedNot reportedNot reported
Arm 2 - Closed1Not reportedNot reportedNot reportedNot reported
AE leading to death
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Arm 1 - Closed0Not reportedNot reportedNot reportedNot reported
Arm 2 - Closed0Not reportedNot reportedNot reportedNot reported
AEs leading to study discontinuation
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Arm 1 - Closed0Not reportedNot reportedNot reportedNot reported
Arm 2 - Closed0Not reportedNot reportedNot reportedNot reported
AEs leading to study treatment discontinuation
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Arm 1 - Closed0Not reportedNot reportedNot reportedNot reported
Arm 2 - Closed1Not reportedNot reportedNot reportedNot reported
AE leading to leading to study treatment dose reduction
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Arm 1 - Closed0Not reportedNot reportedNot reportedNot reported
Arm 2 - Closed0Not reportedNot reportedNot reportedNot reported
Adverse events leading to study treatment dose interruption
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Arm 1 - Closed1Not reportedNot reportedNot reportedNot reported
Arm 2 - Closed1Not reportedNot reportedNot reportedNot reported
Treatment emergent adverse events
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Arm 1 - Closed3Not reportedNot reportedNot reportedNot reported
Arm 2 - Closed2Not reportedNot reportedNot reportedNot reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        0
      2. group Id
        OG001
        value
        1
    title
    DLTs
  2. categories
    1. measurements
      1. group Id
        OG000
        value
        0
      2. group Id
        OG001
        value
        0
    title
    AE leading to death
  3. categories
    1. measurements
      1. group Id
        OG000
        value
        0
      2. group Id
        OG001
        value
        0
    title
    AEs leading to study discontinuation
  4. categories
    1. measurements
      1. group Id
        OG000
        value
        0
      2. group Id
        OG001
        value
        1
    title
    AEs leading to study treatment discontinuation
  5. categories
    1. measurements
      1. group Id
        OG000
        value
        0
      2. group Id
        OG001
        value
        0
    title
    AE leading to leading to study treatment dose reduction
  6. categories
    1. measurements
      1. group Id
        OG000
        value
        1
      2. group Id
        OG001
        value
        1
    title
    Adverse events leading to study treatment dose interruption
  7. categories
    1. measurements
      1. group Id
        OG000
        value
        3
      2. group Id
        OG001
        value
        2
    title
    Treatment emergent adverse events
denoms
  1. counts
    1. group Id
      OG000
      value
      3
    2. group Id
      OG001
      value
      2
    units
    Participants
description
Dose Limiting Toxicities are defined as events that occur during the first 28 days (Day 0 - Day 28) that meets one of the following criteria: * Any local reaction that requires operative intervention and is felt to be attributable to study drug * Any local reaction that has life-threatening consequences requiring urgent intervention or results in death and is felt to be attributable to study drug * Any Grade 3 or higher non-hematologic adverse event that is at least possibly related to study drug and lasts ≥ 3 days * Nausea and vomiting will not be considered a DLT unless at least Grade 3 and refractory to antiemetics * Grade 3 or higher thrombocytopenia (\< 50,000/mm3) at least possibly related to study drug * Any Grade 4 hematologic toxicity (except thrombocytopenia) that is at least possibly related to study drug and lasts ≥ 5 days * Transient neurological changes are expected in Arm 2 and will not be considered a DLT unless they last \> 10 days
groups
  1. description
    Intratumoral Ad-RTS-hIL-12 freehand injection after tumor resection and oral veledimex (activator ligand) in pediatric patients with brain tumors. Ad-RTS-hIL-12: 2.0 x 10\^11 viral particles (vp) per injection, one intratumoral injection of Ad-RTS-hIL-12 Oral Veledimex - Arm 1 (Pediatric Brain Tumor): * Planned: 1 dose level (10mg/day) 15 oral daily doses of veledimex * Actual: All patients received 1 dose level (10 mg/day veledimex)
    id
    OG000
    title
    Arm 1 - Closed
  2. description
    Intratumoral Ad-RTS-hIL-12 stereotactic injection and oral veledimex (activator ligand) in pediatric patients with DIPG. Ad-RTS-hIL-12: 2.0 x 10\^11 viral particles (vp) per injection, one intratumoral injection of Ad-RTS-hIL-12 Oral Veledimex - Arm 2 (DIPG): * Planned:2 dose levels (10mg/day, 20mg/day) * Actual: 1 patient received 5 mg/day veledimex. The other patient did not receive veledimex due to SAE.
    id
    OG001
    title
    Arm 2 - Closed
param Type
COUNT_OF_PARTICIPANTS
population Description
The Full Analysis Set (FAS) or the OSP includes every subject who received at least one dose of any study drug recorded in the Data Management (DM) database excluding screen failures. • The data analysis for Adverse Events or other Safety variables will be performed on the OSP.
reporting Status
POSTED
time Frame
From Day 0 through Day 56
title
The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance.
type
PRIMARY
unit Of Measure
Participants

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

Preserved source evidence · Not medical advice