A Phase 1 interventional study of Carboxylesterase-expressing Allogeneic Neural Stem Cells and Irinotecan in Recurrent Anaplastic Astrocytoma, Recurrent Anaplastic Oligoastrocytoma and Recurrent Anaplastic Oligodendroglioma, sponsored by City of Hope Medical Center. Active, not recruiting at 1 site in United States. Open to participants aged 18 Years to 69 Years. Per ClinicalTrials.gov, last updated 2026-05-11.
Sponsored by City of Hope Medical Center · Phase 1, Interventional, and Treatment
This phase I trial studies the side effects and best dose of carboxylesterase-expressing allogeneic neural stem cells when given together with irinotecan hydrochloride in treating patients with high-grade gliomas that have come back. Placing genetically modified neural stem cells into brain tumor cells may make the tumor more sensitive to irinotecan hydrochloride. Irinotecan hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving carboxylesterase-expressing allogeneic neural stem cells and irinotecan hydrochloride may be a better treatment for high-grade gliomas.
PRIMARY OBJECTIVE:
I. To define the recommended phase II doses (RP2D) of intracranially administered carboxylesterase-expressing allogeneic neural stem cells (hCE1m6-NSCs) in combination with intravenous irinotecan in patients with recurrent high grade glioma.
SECONDARY OBJECTIVES:
I. To describe the relationship between hCE1m6-NSC dose and SN-38 (SN-38) concentrations in brain interstitium.
II. To characterize the relationship between intracerebral and systemic concentrations of irinotecan (irinotecan hydrochloride) and SN-38.
III. To investigate the biologic activity of hCE1m6 NSCs by comparing SN-38 concentrations in the brain after treatment with hCE1m6-NSCs and irinotecan versus irinotecan alone.
IV. To assess for possible development of adenovirally transduced neural stem cell (NSC) immunogenicity after first exposure and with repeat doses of NSCs.
V. To describe the clinical benefit (defined as stable disease, partial response, or complete response) in patients who receive treatment with repeat cycles of NSCs and irinotecan.
VI. To determine, at time of autopsy, the fate of the NSCs.
OUTLINE: This is a dose-escalation study of carboxylesterase-expressing allogeneic neural stem cells.
Patients receive carboxylesterase-expressing allogeneic neural stem cells intracranially over 1.5-4.5 hours on days 1 and 15 (day 1 only for patients at dose level 1) and irinotecan hydrochloride intravenously (IV) over 90 minutes on days 3 and 17. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 days, 3 and 6 months, and then annually thereafter for a minimum of 15 years.
2,132 studies on the registry are indexed under Glioblastoma; 464 are open to participants now.
This study's enrollment of 18 is below the median of 36 across 1,753 interventional studies indexed under Glioblastoma.
Browse Glioblastoma studies →City of Hope Medical Center is the lead sponsor of 677 studies on the registry; 181 are open to participants now.
Of its 31 completed or terminated interventional studies of FDA-regulated products, 12 (39%) have results posted.
Compared within Glioblastoma, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Astrocytoma. The counts are of registry records indexed under each term, refreshed daily. How we classify →
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.
Exclusion Criteria:
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.
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.
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.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
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.
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.
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.
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.
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.
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.
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.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
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.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
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.
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.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
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.
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.
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.
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.
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.
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.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
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.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients receive carboxylesterase-expressing allogeneic neural stem cells intracranially over 1.5-4.5 hours on days 1 and 15 (day 1 only for patients at dose level 1) and irinotecan hydrochloride IV over 90 minutes on days 3 and 17. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Biological: Carboxylesterase-expressing Allogeneic Neural Stem Cells · Drug: Irinotecan · Drug: Irinotecan Hydrochloride · Other: Laboratory Biomarker Analysis · Other: Pharmacological Study
Given intracranially
Also known as: CE-secreting Allogeneic NSCs, hCE1m6-NSC
Given IV
Given IV
Also known as: Campto, Camptosar, Camptothecin 11, Camptothecin-11, CPT 11, CPT-11, Irinomedac, U-101440E, Irinotecan Hydrochloride Trihydrate, Irinotecan Monohydrochloride Trihydrate
Correlative studies
Correlative studies
Number of Participants Experiencing a Dose-limiting Toxicity (DLT)
Toxicities will be graded using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. A DLT is defined as an adverse event that is related to the administration of NSCs and/or irinotecan, occurs during the first treatment cycle and meets any of the following: 1. Received less than 80% of study treatments except due to CNS toxicity 2. Grade 4 thrombocytopenia or anemia or neutropenia lasting \> 7 days 3. Febrile neutropenia with ANC \< 0.5 x10\^9/L 4. Grade 3 central nervous system (CNS) disorder lasting \> 7 days not attributed to tumor or surgery and not present at baseline 5. Second occurrence of grade 3 CNS disorder not attributed to tumor or surgery and not present at baseline 6. Any grade 4 CNS disorder not attributed to tumor or surgery and not present at baseline 7. Grade 3 toxicity despite therapy lasting \> 7 days 8. Grade 3 toxicity resulting in study agent discontinuation 9. Grade 4 toxicity, except grade 4 diarrhea responding to therapy within 3 days
Time frame: 28 days post first dose of NSC treatment on day 1, cycle 1
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to NSCs
Grade 3 or higher toxicity profile as assessed by the NCI CTCAE version version 4.0. Toxicities reported are possibly, probably or definitely related to NSCs.
Time frame: Followed 30 days post treatment for all toxicities (min=33,max 142 days), up to 5 years for gene therapy toxicities
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Grade 3 or higher toxicity profile as assessed by the NCI CTCAE version version 4.0. Toxicities reported are possibly, probably or definitely related to Irinotecan.
Time frame: Followed 30 days post treatment for all toxicities (min=33,max 142 days), up to 5 years for gene therapy toxicities
Median Ratio of SN-38 Area Under the Curve (AUC) to CPT-11 AUC in Plasma
Pharmacokinetic data from patients who undergo intracerebral microdialysis will be summarized using descriptive statistics. hCE1m6-NSC dose and liposomal SN-38 concentrations in brain interstitium using microdialysis data from the patients treated with the initial neural stem cells (NSC) doses and from the patients in the cohort treated with the highest NSC dose. Ratios are reported as ratio x 100.
Time frame: Pre-dose, at 90 minutes (just prior to the end of the infusion), and then at 30 minutes, 1, 2, 4, 8, 24, and 48 hours after the end of the infusion after day 1, cycle 1
Median Ratio of SN-38 AUC to CPT-11 AUC in the Brain
Pharmacokinetic data from patients who undergo intracerebral microdialysis will be summarized using descriptive statistics. hCE1m6-NSC dose and liposomal SN-38 concentrations in brain interstitium using microdialysis data from the patients treated with the initial neural stem cells (NSC) doses and from the patients in the cohort treated with the highest NSC dose. Ratio is reported as ratio x 100
Time frame: Pre-dose, at 90 minutes (just prior to the end of the infusion), and then at 30 minutes, 1, 2, 4, 8, 24, and 48 hours after the end of the infusion after day 1, cycle 1
Number of Participants With Clinical Benefit Defined by Response Assessment in Neuro-Oncology (RANO)
Clinical benefit is defined by participants achieving stable disease (SD), partial response (PR), or complete response (CR). CR: Complete disappearance of all enhancing disease (measurable and nonmeasureable) that is sustained for at least 4 weeks, stable or improved non-enhancing FLAIR/T2 lesions, no new lesions, off corticosteroids (physiologic replacement doses allowed), and neurologically stable or improved. PR: ≥ 50% decrease of all measurable enhancing lesions, sustained for at least 4 weeks, no progression of non-measurable disease, stable or improved non-enhancing FLAIR/T2 lesions, no new lesions, corticosteroid dose stable or reduced (compared to baseline), and neurologically stable or improved. SD: Does not qualify for CR, PR, or PD, stable non-enhancing FLAIR/T2 lesions, stable or reduced corticosteroids (compared to baseline), clinically stable.
Time frame: Until death or disease progression, a median of 2 months, up to 6 months
| Milestone | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|---|
| Started | 4 | 3 | 4 | 7 |
| Completed | 3 | 3 | 3 | 6 |
| Not completed | 1 | 0 | 1 | 1 |
| Withdrew: Taken off study treatment due to persistent gr 2 decreased platelets requiring transfusions | 0 | 0 | 1 | 0 |
| Withdrew: Could not receive irinotecan due to low kps score post surgery | 1 | 0 | 0 | 0 |
| Withdrew: Taken off study treatment to catheter infection not related to treatment | 0 | 0 | 0 | 1 |
Toxicities will be graded using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. A DLT is defined as an adverse event that is related to the administration of NSCs and/or irinotecan, occurs during the first treatment cycle and meets any of the following: 1. Received less than 80% of study treatments except due to CNS toxicity 2. Grade 4 thrombocytopenia or anemia or neutropenia lasting \> 7 days 3. Febrile neutropenia with ANC \< 0.5 x10\^9/L 4. Grade 3 central nervous system (CNS) disorder lasting \> 7 days not attributed to tumor or surgery and not present at baseline 5. Second occurrence of grade 3 CNS disorder not attributed to tumor or surgery and not present at baseline 6. Any grade 4 CNS disorder not attributed to tumor or surgery and not present at baseline 7. Grade 3 toxicity despite therapy lasting \> 7 days 8. Grade 3 toxicity resulting in study agent discontinuation 9. Grade 4 toxicity, except grade 4 diarrhea responding to therapy within 3 days
| Participants | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|---|
| Number of Participants Experiencing a Dose-limiting Toxicity (DLT) | 0 | 0 | 0 | 1 |
Grade 3 or higher toxicity profile as assessed by the NCI CTCAE version version 4.0. Toxicities reported are possibly, probably or definitely related to NSCs.
| Participants | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|---|
| Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to NSCs | 0 | 0 | 0 | 0 |
Grade 3 or higher toxicity profile as assessed by the NCI CTCAE version version 4.0. Toxicities reported are possibly, probably or definitely related to Irinotecan.
| Participants | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|---|
| Fatigue — Yes | 0 | 0 | 1 | 0 |
| Fatigue — No | 4 | 3 | 3 | 7 |
| Platelet count decreased — Yes | 1 | 0 | 0 | 0 |
| Platelet count decreased — No | 3 | 3 | 4 | 7 |
| White blood cell decreased — Yes | 1 | 0 | 0 | 0 |
| White blood cell decreased — No | 3 | 3 | 4 | 7 |
| Meningitis — Yes | 0 | 1 | 0 | 0 |
| Meningitis — No | 4 | 2 | 4 | 7 |
| Hypophosphatemia — Yes | 0 | 1 | 0 | 0 |
| Hypophosphatemia — No | 4 | 2 | 4 | 7 |
| Brain abscess — Yes | 0 | 0 | 1 | 0 |
| Brain abscess — No | 4 | 3 | 3 | 7 |
| Catheter related infection — Yes | 0 | 0 | 0 | 1 |
| Catheter related infection — No | 4 | 3 | 4 | 6 |
Pharmacokinetic data from patients who undergo intracerebral microdialysis will be summarized using descriptive statistics. hCE1m6-NSC dose and liposomal SN-38 concentrations in brain interstitium using microdialysis data from the patients treated with the initial neural stem cells (NSC) doses and from the patients in the cohort treated with the highest NSC dose. Ratios are reported as ratio x 100.
| ratio x 100 | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|
| Median Ratio of SN-38 Area Under the Curve (AUC) to CPT-11 AUC in Plasma | 4.8 (3.8 to 5.5) | 0.8 (0.3 to 1.7) | 2.35 (1.2 to 3.8) |
Pharmacokinetic data from patients who undergo intracerebral microdialysis will be summarized using descriptive statistics. hCE1m6-NSC dose and liposomal SN-38 concentrations in brain interstitium using microdialysis data from the patients treated with the initial neural stem cells (NSC) doses and from the patients in the cohort treated with the highest NSC dose. Ratio is reported as ratio x 100
| ratio x 100 | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|
| Median Ratio of SN-38 AUC to CPT-11 AUC in the Brain | 6.9 (6.0 to 9.3) | 8.8 (4.9 to 28.2) | 12.2 (3.7 to 26.3) |
Clinical benefit is defined by participants achieving stable disease (SD), partial response (PR), or complete response (CR). CR: Complete disappearance of all enhancing disease (measurable and nonmeasureable) that is sustained for at least 4 weeks, stable or improved non-enhancing FLAIR/T2 lesions, no new lesions, off corticosteroids (physiologic replacement doses allowed), and neurologically stable or improved. PR: ≥ 50% decrease of all measurable enhancing lesions, sustained for at least 4 weeks, no progression of non-measurable disease, stable or improved non-enhancing FLAIR/T2 lesions, no new lesions, corticosteroid dose stable or reduced (compared to baseline), and neurologically stable or improved. SD: Does not qualify for CR, PR, or PD, stable non-enhancing FLAIR/T2 lesions, stable or reduced corticosteroids (compared to baseline), clinically stable.
| Participants | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|---|
| Number of Participants With Clinical Benefit Defined by Response Assessment in Neuro-Oncology (RANO) | 0 | 1 | 1 | 3 |
Collected over Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Dose Level 1: 5x10^7 (1 Dose) | 4/4 (100%) | 0/4 (0%) | 4/4 (100%) |
| Dose Level 2: 5x10^7 (2 Doses) | 3/3 (100%) | 1/3 (33.3%) | 3/3 (100%) |
| Dose Level 3: 1x10^8 (2 Doses) | 4/4 (100%) | 2/4 (50%) | 4/4 (100%) |
| Dose Level 4: 1.5x10^8 (2 Doses) | 6/7 (85.7%) | 5/7 (71.4%) | 7/7 (100%) |
| Event | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|---|
| MeningitisInfections and infestations | 0/4 | 1/3 | 0/4 | 0/7 |
| Catheter related infectionInfections and infestations | 0/4 | 0/3 | 0/4 | 2/7 |
| Brain abscessInfections and infestations | 0/4 | 0/3 | 1/4 | 0/7 |
| Myelodysplastic syndromeNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/4 | 0/3 | 1/4 | 0/7 |
| Intracranial hemorrhageNervous system disorders | 0/4 | 0/3 | 1/4 | 0/7 |
| Altered mental statusNervous system disorders | 0/4 | 0/3 | 1/4 | 0/7 |
| HematomaVascular disorders | 0/4 | 0/3 | 1/4 | 0/7 |
| Cerebrospinal fluid leakageNervous system disorders | 0/4 | 0/3 | 0/4 | 1/7 |
| HeadacheNervous system disorders | 0/4 | 0/3 | 0/4 | 1/7 |
| ConfusionPsychiatric disorders | 0/4 | 0/3 | 0/4 | 1/7 |
| Event | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) |
|---|---|---|---|---|
| Gait disturbanceGeneral disorders | 0/4 | 1/3 | 4/4 | 3/7 |
| PainGeneral disorders | 1/4 | 3/3 | 4/4 | 3/7 |
| HypoalbuminemiaMetabolism and nutrition disorders | 4/4 | 2/3 | 3/4 | 4/7 |
| HyponatremiaMetabolism and nutrition disorders | 2/4 | 3/3 | 3/4 | 5/7 |
| HeadacheNervous system disorders | 3/4 | 2/3 | 4/4 | 7/7 |
| AnxietyPsychiatric disorders | 3/4 | 3/3 | 2/4 | 3/7 |
| HypertensionVascular disorders | 3/4 | 3/3 | 4/4 | 7/7 |
| FatigueGeneral disorders | 3/4 | 2/3 | 3/4 | 6/7 |
| AnemiaBlood and lymphatic system disorders | 3/4 | 1/3 | 3/4 | 4/7 |
| Sinus tachycardiaCardiac disorders | 2/4 | 1/3 | 3/4 | 3/7 |
| Age, Categorical(Participants) | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) | Total |
|---|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 4 | 3 | 4 | 6 | 17 |
| >=65 years | 0 | 0 | 0 | 1 | 1 |
| Age, Continuous(years) | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) | Total |
|---|---|---|---|---|---|
| Median | 49.5 (25 to 58) | 56 (55 to 57) | 56.5 (32 to 62) | 44 (32 to 65) | 51 (25 to 65) |
| Sex: Female, Male(Participants) | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) | Total |
|---|---|---|---|---|---|
| Female | 0 | 2 | 1 | 0 | 3 |
| Male | 4 | 1 | 3 | 7 | 15 |
| Ethnicity (NIH/OMB)(Participants) | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 1 | 3 | 4 | 9 |
| Not Hispanic or Latino | 3 | 2 | 1 | 3 | 9 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 1 | 0 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 | 0 |
| White | 4 | 2 | 4 | 7 | 17 |
| More than one race | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 |
| Region of Enrollment(Participants) | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) | Total |
|---|---|---|---|---|---|
| United States | 4 | 3 | 4 | 7 | 18 |
| Tumor Type(Participants) | Dose Level 1: 5x10^7 (1 Dose) | Dose Level 2: 5x10^7 (2 Doses) | Dose Level 3: 1x10^8 (2 Doses) | Dose Level 4: 1.5x10^8 (2 Doses) | Total |
|---|---|---|---|---|---|
| anaplastic astrocytoma, anaplastic oligodendroglioma, anaplastic oligoastrocytoma | 1 | 0 | 1 | 2 | 4 |
| glioblastoma, gliosarcoma | 3 | 3 | 3 | 5 | 14 |
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Studies of the drug tested here: Irinotecan