Trial · NCT03330197
A Study of Ad-RTS-hIL-12 + Veledimex in Pediatric Subjects With Brain Tumors Including DIPG
NCT03330197, Phase 1, Phase 2, Terminated, Alaunos Therapeutics; results first posted 2025-08-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Ad-RTS-hIL-12 + Veledimex in Pediatric Subjects With Brain Tumors Including DIPG. Trials, record NCT03330197. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03330197
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Ad-RTS-hIL-12 + Veledimex in Pediatric Subjects With Brain Tumors Including DIPG
- NCT number
- NCT03330197
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- Alaunos Therapeutics
- Conditions
- Pediatric Brain Tumor, Diffuse Intrinsic Pontine Glioma
- Interventions
- Ad-RTS-hIL-12, Oral Veledimex - Arm 1 (Pediatric Brain Tumor), Oral Veledimex - Arm 2 (DIPG)
- First posted
- 2017-11-06
- Last updated
- 2025-08-12
- Results first posted
- 2025-08-12
- Limitations and caveats
Given the limited number of subjects enrolled and early closure of the study, complete assessment of safety and tolerability could not be determined. There was a wide range of adverse events reported and limited survival among the subjects enrolled.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | From Day 0 through Day 56 | 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 |
| Secondary | To Measure the Veledimex in Blood and Brain Tumor by Using the LC-MS Method | From Day 0 through Day 15 of veledimex dosing | Veledimex brain concentration. Veledimex plasma concentration was below the limit of quantitation for most patients. Given the limited number of subjects enrolled and early closure of the study, complete assessment of veledimex in blood and brain tumor could not be determined. |
| Secondary | Overall Survival (OS) | 2 Years | OS is defined as the duration of time from the first dose of the study drug to the date of death. Subjects were followed for up to 2 years. |
| Secondary | Measure Immune Response of Ad-RTS-hIL-12 and Veledimex by a Quantitative Multiplex Immunoassay for Determination of IL-12 and IFNg Levels | Day 0, Day 3, Day 7, Day 14, and Day 28 | Samples for serum cytokine analysis were collected on Days 0, 3, 7, 14, and 28. For the results, participants who had samples analyzed were included in the "number analyzed" counts; however, values that were below the limit of quantitation (BLQ) were excluded from the calculation of mean and standard deviation. |
| Secondary | Subjects With Ad-RTS-hIL-12 and Veledimex Related Adverse Events Will be Assessed for Safety by CTCAE v5.0 | 2 Years | Subjects with Ad-RTS-hIL-12 and veledimex related adverse events will be assessed for safety by CTCAE v5.0 |
| Secondary | Best Overall Response by iRANO Criteria | 2 Years | Best Overall Response was assessed using the immunotherapy Response Assessment for Neuro-Oncology (iRANO) criteria. |
| Secondary | Duration of Response (DOR) | From the time of first response (CR or PR) until progression or death | Duration of Response (DOR) was defined as the time from the first documentation of a Complete Response (CR) or Partial Response (PR) to the first documentation of Progressive Disease (PD) or death due to any cause, whichever occurred first. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | From Day 0 through Day 56 | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | To Measure the Veledimex in Blood and Brain Tumor by Using the LC-MS Method | From Day 0 through Day 15 of veledimex dosing | Patients with available brain tumor results. Arm 2 patient result was below the level of quantitation (1.8 ng/mL). Arm 1 BLQ was 0.1 ng/mL. One patients was less than BLQ.~Arm 2 patient plasma concentrations were not reported due to bioanalytical non-reproducibility. NRR (Non-Reportable Results) is the actual result reported from the central lab which was due to assay reagent expiration. | ng/mL | MEAN |
| SECONDARY | Overall Survival (OS) | 2 Years | The Safety Population was defined as all subjects who received at least 1 dose of study drug. | participants | NUMBER |
| SECONDARY | Measure Immune Response of Ad-RTS-hIL-12 and Veledimex by a Quantitative Multiplex Immunoassay for Determination of IL-12 and IFNg Levels | Day 0, Day 3, Day 7, Day 14, and Day 28 | The Biomarker Evaluation Population (BEP) includes all subjects who received Ad-RTS-hIL-12 + at least one dose of their cohort-specific dose of veledimex who have adequate biomarker sample(s) at screening (baseline) and at least one non-missing follow-up biomarker assessment, excluding the disqualified assessments. All biomarker related data presentation will be based on the BEP unless specified otherwise. Note: In Arm 2, only 1 subject met this criteria. | pg/mL | MEAN |
| SECONDARY | Subjects With Ad-RTS-hIL-12 and Veledimex Related Adverse Events Will be Assessed for Safety by CTCAE v5.0 | 2 Years | 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. | participants | NUMBER |
| SECONDARY | Best Overall Response by iRANO Criteria | 2 Years | The Efficacy Evaluable population is defined as all subjects who received at least 1 dose of study drug and had at least one post-treatment tumor assessment. | participants | NUMBER |
| SECONDARY | Duration of Response (DOR) | From the time of first response (CR or PR) until progression or death | The analysis population for this measure included subjects from the Efficacy Evaluable population who achieved a Complete Response (CR) or Partial Response (PR). |
Source ClinicalTrials.gov
Results by group94
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 1 - Closed | DLTs | 0 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 2 - Closed | DLTs | 1 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 1 - Closed | AE leading to death | 0 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 2 - Closed | AE leading to death | 0 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 1 - Closed | AEs leading to study discontinuation | 0 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 2 - Closed | AEs leading to study discontinuation | 0 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 1 - Closed | AEs leading to study treatment discontinuation | 0 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 2 - Closed | AEs leading to study treatment discontinuation | 1 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 1 - Closed | AE leading to leading to study treatment dose reduction | 0 | Participants | |
| The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance. | Arm 2 - Closed | AE leading to leading to study treatment dose reduction | 0 | Participants |
1 to 10 of 94Source ClinicalTrials.gov
See all 94 results by groupParticipant flow14
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arm 1 - Closed | 3 | |
| Overall Study | STARTED | Arm 2 - Closed | 3 | |
| Overall Study | Treated With Ad-RTS-hIL-12 Injection | Arm 1 - Closed | 3 | |
| Overall Study | Treated With Ad-RTS-hIL-12 Injection | Arm 2 - Closed | 2 | |
| Overall Study | Treated With Veledimex | Arm 1 - Closed | 3 | |
| Overall Study | Treated With Veledimex | Arm 2 - Closed | 1 | |
| Overall Study | Did Not Receive Veledimex Treatment | Arm 1 - Closed | 0 | |
| Overall Study | Did Not Receive Veledimex Treatment | Arm 2 - Closed | 2 | |
| Overall Study | Received Veledimex Treatment | Arm 1 - Closed | 3 | |
| Overall Study | Received Veledimex Treatment | Arm 2 - Closed | 1 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 participant flowAdverse events116
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | HYPOCALCAEMIA | Metabolism and nutrition disorders | Arm 1 | 2 | 3 | 2 |
| other | HYPOCALCAEMIA | Metabolism and nutrition disorders | Arm 2 | 2 | 2 | 4 |
| other | LYMPHOCYTE COUNT DECREASED | Investigations | Arm 1 | 2 | 3 | 4 |
| other | LYMPHOCYTE COUNT DECREASED | Investigations | Arm 2 | 2 | 2 | 5 |
| other | WHITE BLOOD CELL COUNT DECREASED | Investigations | Arm 1 | 2 | 3 | 3 |
| other | WHITE BLOOD CELL COUNT DECREASED | Investigations | Arm 2 | 2 | 2 | 3 |
| other | ANAEMIA | Blood and lymphatic system disorders | Arm 1 | 3 | 3 | 5 |
| other | ANAEMIA | Blood and lymphatic system disorders | Arm 2 | 1 | 2 | 1 |
| other | PYREXIA | General disorders | Arm 1 | 1 | 3 | 1 |
| other | PYREXIA | General disorders | Arm 2 | 2 | 2 | 4 |
1 to 10 of 116Source ClinicalTrials.gov
See all 116 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arm 1 | 2 | 3 |
| Total, all-cause mortality | Arm 2 | 1 | 2 |
| Total, other adverse events | Arm 1 | 3 | 3 |
| Total, other adverse events | Arm 2 | 2 | 2 |
| Total, serious adverse events | Arm 1 | 1 | 3 |
| Total, serious adverse events | Arm 2 | 1 | 2 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Arm 1 vs Arm 2: deaths from any cause | Trial comparisons |
| Arm 1 vs Arm 2: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03330197
GET https://conductscience.org/api/trials/v1/NCT03330197/results?page=2