A Phase 1/2 interventional study of Ad-RTS-hIL-12 and Oral Veledimex - Arm 1 (Pediatric Brain Tumor) in Pediatric Brain Tumor and Diffuse Intrinsic Pontine Glioma, sponsored by Alaunos Therapeutics. Terminated at 3 sites in United States. Open to participants aged 0 Years to 21 Years. Per ClinicalTrials.gov, last updated 2025-08-12.
Sponsored by Alaunos Therapeutics · Phase 1/2, Interventional, and Treatment
Among 88 studies in Diffuse Intrinsic Pontine Glioma with a site in United States — 24 open to participants now, 34 under way, none of them phase 3. Diffuse Intrinsic Pontine Glioma · United States.
Among the 88 studies in Diffuse Intrinsic Pontine Glioma with a site in United States, this one is the 72nd largest by enrollment of the 82 interventional ones, is one of 4 that started in 2017, is one of 17 in phase 1/2, and was the 35th registered.
119 studies on the registry are indexed under Diffuse Intrinsic Pontine Glioma; 39 are open to participants now.
This study's enrollment of 6 is below the median of 30 across 109 interventional studies indexed under Diffuse Intrinsic Pontine Glioma.
Browse Diffuse Intrinsic Pontine Glioma studies →24 of the 88 studies in Diffuse Intrinsic Pontine Glioma with a site in United States are open now, in 9 countries. Each count lists the studies.
Of the 29 Diffuse Intrinsic Pontine Glioma studies recruiting now, 3 (10%) set no upper age limit and 26 (90%) stop at 75 or younger; 28 take children under 18; 2 take healthy volunteers.
This study: 0 Years to 21 Years · all sexes · no healthy volunteers.
The full table on the hub →88 studies; the count open now beside each. This study's row is marked.
First in 2002; this study’s year is marked. 4 started in 2017.
Median enrollment 30 across the 82 interventional studies, 100 across the 5 observational. Of the 47 that completed or were terminated, 17 (36%) have results posted.
18 of 88 have an industry lead sponsor. The 10 most frequent:
Alaunos Therapeutics is the lead sponsor of 37 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 5 (100%) have results posted.
Alaunos Therapeutics has 1 study filed under Diffuse Intrinsic Pontine Glioma — fewer than ten, so there is no comparison to draw.
9 of the studies in Diffuse Intrinsic Pontine Glioma with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05762419Columbia UniversityFUS Etoposide for DMG | 2026-12 |
| NCT04655404Nationwide Children's HospitalA Pilot Study of Larotrectinib for Newly-Diagnosed High-Grade Glioma With NTRK Fusion | 2026-12-01 |
| NCT04870944Children's Oncology GroupCBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma | 2026-12-31 |
| NCT04099797Baylor College of MedicineC7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B) | 2027-02 |
| NCT01837862Julie KrystalA Phase I Study of Mebendazole for the Treatment of Pediatric Gliomas | 2027-03 |
Among the 88 studies in Diffuse Intrinsic Pontine Glioma with a site in United States, from their registry updates:
This research study involves an investigational product: Ad-RTS-hIL-12 given with veledimex for production of human IL-12. IL-12 is a protein that can improve the body's natural response to disease by enhancing the ability of the immune system to kill tumor cells and may interfere with blood flow to the tumor.
The main purpose of this study is to evaluate the safety and tolerability of a single tumor injection of Ad-RTS-hIL-12 given with oral veledimex in the pediatric population.
Eligible patients will be stratified to one of two arms, according to clinical indication for tumor resection. Pediatric patients who are scheduled for craniotomy and tumor resection will receive one dose of veledimex before the resection procedure. Ad-RTS-hIL-12 will be administered by free-hand injection. Patients will continue on oral veledimex for 14 days. This arm has been completed and is currently closed to enrollment.
Pediatric patients with diffuse intrinsic pontine glioma (DIPG) will receive Ad-RTS-hIL-12 by stereotactic injection and then will continue on oral veledimex for 14 days.
The study is divided into three periods: the screening period, the treatment period and the follow-up period.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Arm 1: Evidence of recurrent or progressive supratentorial tumor, which has shown a > 25% increase in bi dimensional measurements by MRI or is with significant neuro deterioration that is not otherwise explained with no known curative therapy, not in direct continuity with the ventricular system (e.g., there is physical separation between the tumor and ventricle, the tumor does not open directly into the ventricular system).
Arm 2: Clinical presentation of DIPG and compatible MRI with approximately 2/3 of the pons included and without evidence of dissemination. Subjects should be ≥ 2 weeks and ≤ 10 weeks post standard focal radiotherapy (ie, dose of 5400 to 5960 cGy and maximum dexamethasone of 1 mg/m2/day)
At the time of registration, subjects must have recovered from the toxic effects of previous treatments, as determined by the treating physician.
Have age-appropriate functional performance:
Have adequate bone marrow reserves and liver and kidney function, as assessed by the following laboratory requirements:
Exclusion Criteria:
Radiotherapy treatment prior to the first veledimex dose:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
PD-1, PD-L1 and CTLA-4 are proteins that act as brakes on the immune system. Checkpoint inhibitors, such as pembrolizumab or nivolumab, block them so the immune system can attack a cancer.
PD-L1 can also be measured in a tumour sample. Some scores count stained tumour cells only (TPS); others also count certain immune cells (CPS). The study names the score and the minimum it uses.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
PD-1, PD-L1 and checkpoint inhibitors
PD-1, PD-L1 and CTLA-4 are proteins that act as brakes on the immune system. Checkpoint inhibitors, such as pembrolizumab or nivolumab, block them so the immune system can attack a cancer.
PD-L1 can also be measured in a tumour sample. Some scores count stained tumour cells only (TPS); others also count certain immune cells (CPS). The study names the score and the minimum it uses.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Intratumoral Ad-RTS-hIL-12 freehand injection after tumor resection and oral veledimex (activator ligand) in pediatric patients with brain tumors.
Biological: Ad-RTS-hIL-12 · Drug: Oral Veledimex - Arm 1 (Pediatric Brain Tumor)
Intratumoral Ad-RTS-hIL-12 stereotactic injection and oral veledimex (activator ligand) in pediatric patients with DIPG.
Biological: Ad-RTS-hIL-12 · Drug: Oral Veledimex - Arm 2 (DIPG)
2.0 x 10\^11 viral particles (vp) per injection, one intratumoral injection of Ad-RTS-hIL-12
1 dose level (10mg/day) 15 oral daily doses of veledimex
2 dose levels (10mg/day, 20mg/day) 14 oral daily doses of veledimex
The Safety and Tolerability of Intratumoral Ad-RTS-hIL-12 and Veledimex as Measured by Dose Limiting Toxicities and Compliance.
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
Time frame: From Day 0 through Day 56
To Measure the Veledimex in Blood and Brain Tumor by Using the LC-MS Method
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.
Time frame: From Day 0 through Day 15 of veledimex dosing
Overall Survival (OS)
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.
Time frame: 2 Years
Measure Immune Response of Ad-RTS-hIL-12 and Veledimex by a Quantitative Multiplex Immunoassay for Determination of IL-12 and IFNg Levels
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.
Time frame: Day 0, Day 3, Day 7, Day 14, and Day 28
Subjects With Ad-RTS-hIL-12 and Veledimex Related Adverse Events Will be Assessed for Safety by CTCAE v5.0
Subjects with Ad-RTS-hIL-12 and veledimex related adverse events will be assessed for safety by CTCAE v5.0
Time frame: 2 Years
Best Overall Response by iRANO Criteria
Best Overall Response was assessed using the immunotherapy Response Assessment for Neuro-Oncology (iRANO) criteria.
Time frame: 2 Years
Duration of Response (DOR)
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.
Time frame: From the time of first response (CR or PR) until progression or death
One Arm 2 subject was screened and enrolled but consent was withdrawn prior to Day 0.
| Milestone | Arm 1 - Closed | Arm 2 - Closed |
|---|---|---|
| Started | 3 | 3 |
| Treated with ad-rts-hil-12 injection | 3 | 2 |
| Treated with veledimex | 3 | 1 |
| Did not receive veledimex treatment | 0 | 2 |
| Received veledimex treatment | 3 | 1 |
| Completed | 3 | 1 |
| Not completed | 0 | 2 |
| Withdrew: Adverse event | 0 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 |
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
| Participants | Arm 1 - Closed | Arm 2 - Closed |
|---|---|---|
| DLTs | 0 | 1 |
| AE leading to death | 0 | 0 |
| AEs leading to study discontinuation | 0 | 0 |
| AEs leading to study treatment discontinuation | 0 | 1 |
| AE leading to leading to study treatment dose reduction | 0 | 0 |
| Adverse events leading to study treatment dose interruption | 1 | 1 |
| Treatment emergent adverse events | 3 | 2 |
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.
| ng/mL | Arm 1 - Closed | Arm 2 - Closed |
|---|---|---|
| Brain tumor results (Day 0) | 0.172 (0.1 to 0.211) | NA (NA to NA) |
| Day 0 time of resection - veledimex plasma PK | 1.40 (1.00 to 1.86) | NA (NA to NA) |
| Day 1 predose- veledimex plasma PK | 1.02 (1 to 1.07) | NA (NA to NA) |
| Day 1 2 hr postdose- veledimex plasma PK | 11.64 (8.61 to 13.8) | NA (NA to NA) |
| Day 1 4 hr postdose- veledimex plasma PK | 5.66 (3.31 to 9.85) | NA (NA to NA) |
| Day 1 6 hr postdose- veledimex plasma PK | 5.64 (2.91 to 11.1) | NA (NA to NA) |
| Day 2 - veledimex plasma PK | 2.09 (1.4 to 2.92) | NA (NA to NA) |
| Day 14 predose- veledimex plasma PK | 4.39 (2.61 to 6.15) | NA (NA to NA) |
| Day 14 2 hr postdose - veledimex plasma PK | 36.83 (14.3 to 66.9) | NA (NA to NA) |
| Day 14 4 hr postdose - veledimex plasma PK | 19.80 (9.19 to 30.9) | NA (NA to NA) |
| Day 14 6 hr postdose - veledimex plasma PK | 7.63 (7.41 to 8.05) | NA (NA to NA) |
| Day 15 24 hr postdose - veledimex plasma PK | 4.35 (3.13 to 5.03) | NA (NA to NA) |
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.
| participants | Arm 1 - Closed | Arm 2 - Closed |
|---|---|---|
| Deceased | 2 | 1 |
| Alive | 0 | 1 |
| Unknown | 1 | 0 |
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.
| pg/mL | Arm 1 - Closed | Arm 2 - Closed |
|---|---|---|
| Serum IFN-gamma - Day 0 | NA ± NA | NA ± NA |
| Serum IFN-gamma - Day 3 | 4.4 ± 0 | 11.4 ± 0 |
| Serum IFN-gamma - Day 7 | 6.4 ± 0 | NA ± NA |
| Serum IFN-gamma - Day 14 | NA ± NA | NA ± NA |
| Serum IFN-gamma - Day 28 | NA ± NA | NA ± NA |
| Serum IL-12 - Day 0 | 12 ± 0 | NA ± NA |
| 1.6Serum IL-12 - Day 3 | 11.1 ± 2.1 | NA ± NA |
| Serum IL-12 - Day 7 | 6.6 ± 4.7 | NA ± NA |
| Serum IL-12 - Day 14 | NA ± NA | NA ± NA |
| Serum IL-12 - Day 28 | 1.6 ± 0 | NA ± NA |
Subjects with Ad-RTS-hIL-12 and veledimex related adverse events will be assessed for safety by CTCAE v5.0
| participants | Arm 1 - Closed | Arm 2 - Closed |
|---|---|---|
| WHITE BLOOD CELL COUNT DECREASED (INVESTIGATIONS) | 2 | 2 |
| LYMPHOCYTE COUNT DECREASED (INVESTIGATIONS) | 2 | 2 |
| VOMITING (GASTROINTESTINAL DISORDERS) | 2 | 1 |
| HEADACHE (NERVOUS SYSTEM DISORDERS) | 2 | 1 |
| PYREXIA (GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS) | 1 | 2 |
| ANAEMIA (BLOOD AND LYMPHATIC SYSTEM DISORDERS) | 2 | 0 |
| APHASIA (NERVOUS SYSTEM DISORDERS) | 1 | 1 |
| FATIGUE (GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS) | 1 | 1 |
| HYPONATRAEMIA (METABOLISM AND NUTRITION DISORDERS) | 2 | 0 |
| NAUSEA (GASTROINTESTINAL DISORDERS) | 1 | 1 |
| PLATELET COUNT DECREASED (INVESTIGATIONS) | 2 | 0 |
| TACHYCARDIA (CARDIAC DISORDERS) | 0 | 2 |
Best Overall Response was assessed using the immunotherapy Response Assessment for Neuro-Oncology (iRANO) criteria.
| participants | Arm 1 - Closed | Arm 2 - Closed |
|---|---|---|
| Best Tumor Response SD | 0 | 2 |
| Best Tumor Response PD | 3 | 0 |
| Not Evaluable | 0 | 1 |
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.
No measurements were reported for this outcome.
Collected over 2 years. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Arm 1 | 2/3 (66.7%) | 1/3 (33.3%) | 3/3 (100%) |
| Arm 2 | 1/2 (50%) | 1/2 (50%) | 2/2 (100%) |
| Event | Arm 1 | Arm 2 |
|---|---|---|
| HydrocephalusNervous system disorders | 0/3 | 1/2 |
| SeizureNervous system disorders | 1/3 | 0/2 |
| Event | Arm 1 | Arm 2 |
|---|---|---|
| HYPOCALCAEMIAMetabolism and nutrition disorders | 2/3 | 2/2 |
| LYMPHOCYTE COUNT DECREASEDInvestigations | 2/3 | 2/2 |
| WHITE BLOOD CELL COUNT DECREASEDInvestigations | 2/3 | 2/2 |
| ANAEMIABlood and lymphatic system disorders | 3/3 | 1/2 |
| PYREXIAGeneral disorders | 1/3 | 2/2 |
| HYPERGLYCAEMIAMetabolism and nutrition disorders | 0/3 | 2/2 |
| TACHYCARDIACardiac disorders | 0/3 | 2/2 |
| VOMITINGGastrointestinal disorders | 2/3 | 2/2 |
| HEADACHENervous system disorders | 2/3 | 1/2 |
| HYPOALBUMINAEMIAMetabolism and nutrition disorders | 2/3 | 1/2 |
The baseline population includes every subject who received at least one dose of any study drug recorded in the Data Management (DM) database excluding screen failures. One patient in arm 2 withdrew consent prior to dosing and is considered a screen failure and one patient in Arm 2 only received Ad-RTS-hIL-12 (veledimex was not administered due to a serious adverse event.
| Age, Continuous(years) | Arm 1 - Closed | Arm 2 - Closed | Total |
|---|---|---|---|
| Mean | 16.1 (12.1 to 21.9) | 5.8 (5.7 to 5.9) | 12.1 (5.7 to 21.9) |
| Sex: Female, Male(Participants) | Arm 1 - Closed | Arm 2 - Closed | Total |
|---|---|---|---|
| Female | 2 | 1 | 3 |
| Male | 1 | 1 | 2 |
| Race (NIH/OMB)(Participants) | Arm 1 - Closed | Arm 2 - Closed | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 2 | 1 | 3 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 1 | 2 |
| Region of Enrollment(participants) | Arm 1 - Closed | Arm 2 - Closed | Total |
|---|---|---|---|
| United States | 3 | 2 | 5 |
| WHO High Grade at Baseline(Participants) | Arm 1 - Closed | Arm 2 - Closed | Total |
|---|---|---|---|
| Count of participants | 3 | 2 | 5 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Undecided
Ran from Sep 2017 to primary completion in Sep 2021, 47 months.
Results were first posted in Aug 2025, 3 years 11 months after primary completion.
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Diffuse Intrinsic Pontine Glioma →