A Phase 2 interventional study of TTRNA-xALT and TTRNA-DCs in Medulloblastoma and Neuroectodermal Tumor, sponsored by University of Florida. Completed at 3 sites in United States. Open to participants aged Up to 30 Years. Per ClinicalTrials.gov, last updated 2026-08-13.
Sponsored by University of Florida · Phase 2, Interventional, and Treatment
Immunotherapy is a specific approach to treating cancer that has shown promise in adult patients for the treatment of melanoma, malignant brain tumors, and other cancers. The study investigators will use the experience they have gained from these studies to try to improve the outcome for children affected by a recurrent brain tumor.
Approximately 35 patients with first recurrence of medulloblastoma (reMB)/supratentorial primitive neuroectodermal tumors (PNETs) will be treated with tumor-specific immune cells and dendritic cell vaccines to see what impact they have on the tumor.
Malignant brain tumors now represent the most frequent cause of cancer death in children. Despite aggressive and highly toxic multi-modality therapy including surgery, craniospinal radiation, and high-dose chemotherapy coupled with peripheral blood stem cell transplantation, almost half the children diagnosed with the most common malignant brain tumors, medulloblastoma (MB) and primitive neuroectodermal tumors (PNET), will still die from recurrent disease. Furthermore, survivors are often left with severe and lifelong treatment-associated cognitive and motor deficits. The development of more effective and tumor-specific therapies that will not add further toxicity to existing treatments is paramount in improving clinical outcomes for children affected by MB/PNETs. Immunotherapy targeting tumor-specific antigens expressed within brain tumors is a modality potentially capable of meeting this clear and urgent need.
Despite considerable advancements and promising clinical results observed in immunotherapy trials directed against adult malignant brain tumors, efforts in the immunologic treatment of pediatric brain tumors have been limited to relatively few notable studies. This is due, at least in part, to the often limited viable tumor tissue available for tumor cell-based vaccine preparations, and the lack of identification of consistently expressed tumor-specific antigens within these cancers.
The use of total tumor RNA (TTRNA)-loaded dendritic cells (DCs) was pioneered at Duke University, as a novel platform for inducing potent immunologic responses against the variety of uncharacterized and patient-specific antigens present within malignant tumor cells. Duke demonstrated that sufficient RNA for clinical vaccine preparations can be amplified with high fidelity using existing molecular technologies from as few as 500 isolated pediatric and adult brain tumor cells, thus allowing vaccine preparation from surgical biopsies and even microdissected archival tumor specimens.
Immunotherapy administered during recovery from chemotherapy may have tremendous advantages, as adoptive cellular therapy following lymphodepletive conditioning regimens has emerged as the most effective treatment strategy for advanced and refractory melanoma. Our hypothesis is that DC + ex vivo expanded Autologous Lymphocyte Transfer (xALT) therapy targeting recurrent MB/PNETs during recovery from myeloablative chemotherapy will be safe and will prolong survival in children and young adults with recurrent MB/PNETs.
In this study, the investigators will treat patients with first recurrence reMB/PNETs after completion of definitive radiation therapy with autologous tumor-specific T cell immunotherapy (TTRNA-xALT) plus TTRNA-loaded dendritic cell vaccine.
Following surgical resection, biopsy, or cytology examination with confirmatory pathologic diagnosis, patients will be enrolled into Group A (high-dose chemotherapy or HDC) or Group B (non-myeloablative or NMA salvage chemotherapy) based on eligibility for HDC. Patients with localized relapse and have not failed HDC+ peripheral blood stem cell transplant (PBSCT) previously will be enrolled into Group A. Patients with disseminated disease, have previously failed HDC+PBSCT, or are otherwise considered poor candidates for HDC based on overall health status, but otherwise meet eligibility criteria, will be enrolled into Group B. All patients will receive DC + xALT therapy.
317 studies on the registry are indexed under Medulloblastoma; 51 are open to participants now.
This study's enrollment of 26 is below the median of 31 across 255 interventional studies indexed under Medulloblastoma.
Browse Medulloblastoma studies →University of Florida is the lead sponsor of 1,287 studies on the registry; 199 are open to participants now.
Of its 180 completed or terminated interventional studies of FDA-regulated products, 143 (79%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Screening:
Re-MATCH Protocol:
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.
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.
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 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 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 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).
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.
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.
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.
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 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.
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 B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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 B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
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.
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.
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.
High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs.
Biological: TTRNA-xALT · Biological: TTRNA-DCs
NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs.
Biological: TTRNA-xALT · Biological: TTRNA-DCs
TTRNA-xALT 3 x 10\^7/kg by intravenous injection once.
TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
12 Month Progression-free Survival (PFS-12)
PFS-12 is the number of participants (%) with PFS at 12 months. PFS is defined as time interval from date of first DC vaccine to date of progression (death is also treated as progression) or censoring, whichever happens first. The PFS-12 of Historical Benchmark is 33%. The granular PFS of Historical Bechmark is 4,16, 5, 12, 14, 7, 5, 5, 12, 9, 24 and 13 months (Gururangan et al,. Neuro Oncol. 2008, Table 3)
Time frame: up to 12 months
Objective Radiographic Response Rate
Objective Response Rate (ORR), defined as the proportion of subjects who show partial or complete response (CR+PR) to therapy, SD (stable disease), and PD (progressive disease) or not assessable, using RECIST criteria based on their best overall response over 8 weeks when comparing pre-ACT vs. post-ACT MRI.
Time frame: Baseline MRI (prior to Adoptive Cellular Therapy (ACT)) compared to post-ACT MRI (approximately 8 weeks post-baseline MRI)
Change in Cytokine Profile for IFNg
We will measure serum cytokines in peripheral blood pre and post ACT therapy for patients in Arm A and Arm B. We will compare baseline (pre-ACT) to post treatment (both TTRNA-xALT and TTRNA-DCs vaccines administered) changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Overall Survival (OS) Rate
12-month OS calculated based on benchmark. OS-12 is the proportion of participants with OS at 12 months.
Time frame: baseline up to 12 months
Change in Type 1 Interferon
We will measure change in cellular immunity in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 28 (including Day 14) with each patient. OS Univariable Cox Regression with Change in Cellular Immunity was applied. The Hazard Ratio with 95% CI reported.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Type 2 Interferon
We will measure change in cellular immunity in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 28 (including Day 14) with each patient. OS Univariable Cox Regression with Change in Cellular Immunity was applied. The Hazard Ratio with 95% CI reported.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Percentage of CD8 Naive T Cells in PBMC
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline (pre-treatment) to 28 days with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Cytokine Profile for IL10
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Cytokine Profile for IL12p70
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Cytokine Profile for IL2
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Cytokine Profile for IL4
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Cytokine Profile for IL6
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Cytokine Profile for TNFa
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in TLR Activation Status
We will measure change in TLR activation status in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model. We quantified pathway activity using GSVA applied to normalized RNA-seq expression data. The enrichment score for each sample was defined as the maximum deviation from zero of this running sum, yielding a dimensionless GSVA score that represents the relative coordinated up- or down-regulation of TLR pathway genes within that sample compared to the background transcriptome. Higher GSVA score represents upregulation and lower GSVA score represents downregulation. There is no clinical relevance threshold.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Percentage of CD8 Memory T Cells in PBMC
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Percentage of CD4 Naive T Cells in PBMC
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Percentage of CD4 Memory T Cells in PBMC
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Percentage of Treg T Cells in PBMC
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
Change in Percentage of NK Cells in PBMC
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
Time frame: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)
| Milestone | Group A | Group B |
|---|---|---|
| Started | 7 | 19 |
| Completed | 7 | 15 |
| Not completed | 0 | 4 |
| Withdrew: Progressed prior to vaccine | 0 | 2 |
| Withdrew: Unable to make vaccine | 0 | 2 |
PFS-12 is the number of participants (%) with PFS at 12 months. PFS is defined as time interval from date of first DC vaccine to date of progression (death is also treated as progression) or censoring, whichever happens first. The PFS-12 of Historical Benchmark is 33%. The granular PFS of Historical Bechmark is 4,16, 5, 12, 14, 7, 5, 5, 12, 9, 24 and 13 months (Gururangan et al,. Neuro Oncol. 2008, Table 3)
| percentage | Group A | Group B |
|---|---|---|
| 12 Month Progression-free Survival (PFS-12) | 29 (8.9 to 92) | 0 (0 to 0) |
Objective Response Rate (ORR), defined as the proportion of subjects who show partial or complete response (CR+PR) to therapy, SD (stable disease), and PD (progressive disease) or not assessable, using RECIST criteria based on their best overall response over 8 weeks when comparing pre-ACT vs. post-ACT MRI.
| Participants | Group A | Group B |
|---|---|---|
| CR + PR | 1 | 0 |
| SD (Stable Disease) | 0 | 0 |
| PD (Progressive Disease) | 0 | 4 |
| Not Assessable | 6 | 11 |
We will measure serum cytokines in peripheral blood pre and post ACT therapy for patients in Arm A and Arm B. We will compare baseline (pre-ACT) to post treatment (both TTRNA-xALT and TTRNA-DCs vaccines administered) changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
| pg/mL | Group A | Group B |
|---|---|---|
| Baseline | 2.51 (1.51 to 3.36) | 4.61 (3.22 to 12.77) |
| Cytokine change from baseline to 14 days | 812.5 (26.01 to 838.53) | 10.35 (2.42 to 48.38) |
| Cytokine change from baseline to 28 days | 6.22 (5.2 to 7.61) | 27.45 (3.92 to 66.83) |
12-month OS calculated based on benchmark. OS-12 is the proportion of participants with OS at 12 months.
| percentage of participants | Group A | Group B |
|---|---|---|
| Overall Survival (OS) Rate | 71.43 (45 to 100) | 46.67 (27 to 80) |
We will measure change in cellular immunity in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 28 (including Day 14) with each patient. OS Univariable Cox Regression with Change in Cellular Immunity was applied. The Hazard Ratio with 95% CI reported.
| ratio | Group A Plus Group B | Group A | Group B |
|---|---|---|---|
| Change in Type 1 Interferon | 1.45 (0.57 to 3.66) | 0.67 (0.11 to 3.96) | 2.33 (0.56 to 9.7) |
We will measure change in cellular immunity in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 28 (including Day 14) with each patient. OS Univariable Cox Regression with Change in Cellular Immunity was applied. The Hazard Ratio with 95% CI reported.
| ratio | Group A Plus Group B | Group A | Group B |
|---|---|---|---|
| Change in Type 2 Interferon | 0.97 (0.40 to 2.34) | 0.29 (0.05 to 1.8) | 5.99 (0.45 to 79.2) |
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline (pre-treatment) to 28 days with each patient serving as own control using mixed linear effect model.
| percentage of CD8 Naive T Cells in PBMC | Group A Plus Group B | Group A | Group B |
|---|---|---|---|
| 28 Days | -24.28 (-31.89 to -18.09) | -22.33 (-33.21 to -13.84) | -24.61 (-30.56 to -23.79) |
| 14 Days | -15.52 (-33.25 to -8.88) | -10.8 (-16.21 to -6.97) | -21.67 (-38.72 to -12.0) |
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
| pg/mL | Group A | Group B |
|---|---|---|
| Baseline | 0.28 (0.24 to 0.31) | 0.54 (0.30 to 1.41) |
| Cytokine change from baseline to 14 days | 3.31 (0.55 to 5.05) | 0.41 (0.32 to 0.56) |
| Cytokine change from baseline to 28 days | 0.32 (0.16 to 0.48) | 0.62 (0.38 to 1.14) |
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
| pg/mL | Group A | Group B |
|---|---|---|
| Baseline | 0.05 (0.03 to 0.08) | 0.08 (0.07 to 0.15) |
| Cytokine change from baseline to 14 days | 0.47 (0.21 to 0.59) | 0.10 (0.09 to 0.12) |
| Cytokine change from baseline to 28 days | 0.30 (0.24 to 0.37) | 0.11 (0.04 to 0.17) |
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
| pg/mL | Group A | Group B |
|---|---|---|
| Baseline | 0.16 (0.11 to 0.30) | 0.18 (0.12 to 0.24) |
| Cytokine change from baseline to 14 days | 2.63 (0.31 to 2.86) | 0.31 (0.22 to 0.35) |
| Cytokine change from baseline to 28 days | 0.40 (0.28 to 0.82) | 0.28 (0.15 to 0.36) |
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
| pg/mL | Group A | Group B |
|---|---|---|
| Baseline | 0.05 (0.05 to 0.05) | 0.03 (0.02 to 0.05) |
| Cytokine change from baseline to 14 days | 0.04 (0.03 to 0.10) | 0.06 (0.05 to 0.08) |
| Cytokine change from baseline to 28 days | 0.04 (0.02 to 0.06) | 0.06 (0.02 to 0.07) |
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
| pg/mL | Group A | Group B |
|---|---|---|
| Baseline | 0.80 (0.23 to 1.78) | 1.63 (0.94 to 1.83) |
| Cytokine change from baseline to 14 days | 5.54 (1.94 to 17.63) | 0.54 (0.17 to 1.77) |
| Cytokine change from baseline to 28 days | 1.63 (0.31 to 3.16) | 0.73 (0.38 to 1.72) |
We will measure serum cytokines in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model.
| pg/mL | Group A | Group B |
|---|---|---|
| Baseline | 1.06 (0.98 to 1.12) | 1.24 (0.89 to 1.82) |
| Cytokine change from baseline to 14 days | 2.80 (1.20 to 3.34) | 1.34 (0.99 to 1.43) |
| Cytokine change from baseline to 28 days | 1.21 (0.78 to 1.72) | 1.64 (1.16 to 1.93) |
We will measure change in TLR activation status in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to Day 14 and 28 with each patient serving as own control using mixed linear effect model. We quantified pathway activity using GSVA applied to normalized RNA-seq expression data. The enrichment score for each sample was defined as the maximum deviation from zero of this running sum, yielding a dimensionless GSVA score that represents the relative coordinated up- or down-regulation of TLR pathway genes within that sample compared to the background transcriptome. Higher GSVA score represents upregulation and lower GSVA score represents downregulation. There is no clinical relevance threshold.
| dimensionless enrichment score | Group A | Group B |
|---|---|---|
| Baseline | 0.94 (0.91 to 1.00) | 0.96 (0.92 to 1.05) |
| Change from baseline to 14 days | 1.11 (1.10 to 1.12) | 1.00 (0.97 to 1.09) |
| Change from baseline to 28 days | 1.02 (0.97 to 1.04) | 1.01 (0.85 to 1.11) |
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
| percentage of CD8 Memory T Cell | Group A Plus Group B | Group A | Group B |
|---|---|---|---|
| 28 Days | -12.12 (-16.3 to 1.25) | -11.83 (-13.05 to -2.49) | -12.42 (-19.32 to 4.99) |
| 14 Days | -1.91 (-14.37 to 28.03) | -11.92 (-13.05 to -3.82) | 13.38 (-15.69 to 49.53) |
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
| percentage of CD4 Naive T Cell | Group A Plus Group B | Group A | Group B |
|---|---|---|---|
| 28 Days | -9.03 (-12.42 to -4.11) | -8.44 (-10.31 to -4.94) | -9.61 (-12.83 to -3.29) |
| 14 Days | 9.02 (-11.04 to 22.37) | 9.4 (-10.31 to 11.86) | 8.64 (-11.78 to 45.0) |
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
| percentage of CD4 Memory T Cells | Group A Plus Group B | Group A | Group B |
|---|---|---|---|
| 28 Days | -6.4 (-15.48 to -2.21) | -17.11 (-20.52 to -3.76) | -5.79 (-13.31 to -0.66) |
| 14 Days | -3.94 (-9.38 to -0.02) | -0.04 (-3.25 to 2.93) | -5.79 (-13.31 to -3.9) |
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
| percentage of Treg T Cells in PBMC | Group A Plus Group B | Group A | Group B |
|---|---|---|---|
| 28 Days | -13.24 (-28.56 to 5.01) | -7.97 (-21.71 to -4.17) | -16.37 (-35.41 to 27.41) |
| 14 Days | 12.99 (-9.09 to 33.03) | 19.99 (13.22 to 26.20) | 5.31 (-32.32 to 43.8) |
We will measure change in lymphocyte phenotype in peripheral blood pre and post therapy for patients in Arm A and Arm B. We will compare baseline to post treatment changes for each arm. Longitudinal difference baseline to 28 days with each patient serving as own control using mixed linear effect model.
| percentage of NK Cells in PBMC | Group A Plus Group B | Group A | Group B |
|---|---|---|---|
| 28 Days | 2.43 (-2.57 to 4.29) | 3.54 (-1.06 to 5.04) | 2.04 (-6.73 to 3.03) |
| 14 Days | 3.67 (-3.15 to 9.92) | 5.55 (3.82 to 14.32) | 0.68 (-5.54 to 3.68) |
Collected over AEs were assessed during two periods; initiation of non-mobilized leukapheresis and ends 24 hrs after completion of the procedure and initiation of consolidation/NMA chemotherapy and continued until 100 days after the infusion of PBSCs or completion of chemotherapy when not receiving PBSCs, assessed up to 5 months. All-cause mortality was assessed up to 60 months. Two participants survived > 60 months.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Group A | 6/7 (85.7%) | 4/7 (57.1%) | 7/7 (100%) |
| Group B | 18/19 (94.7%) | 5/19 (26.3%) | 19/19 (100%) |
| Event | Group A | Group B |
|---|---|---|
| Hearing impairedEar and labyrinth disorders | 2/7 | 0/19 |
| HypotensionVascular disorders | 2/7 | 0/19 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 1/7 | 0/19 |
| TachycardiaCardiac disorders | 1/7 | 0/19 |
| HyperbilirubinemiaMetabolism and nutrition disorders | 1/7 | 0/19 |
| Febrile NeutropeniaBlood and lymphatic system disorders | 0/7 | 2/19 |
| PainGeneral disorders | 0/7 | 2/19 |
| Respiratory ArrestRespiratory, thoracic and mediastinal disorders | 0/7 | 1/19 |
| Cardiac ArrestCardiac disorders | 0/7 | 1/19 |
| Colitis, InfectiousInfections and infestations | 0/7 | 1/19 |
| Event | Group A | Group B |
|---|---|---|
| AnemiaBlood and lymphatic system disorders | 7/7 | 10/19 |
| Lymphocyte count decreasedInvestigations | 6/7 | 14/19 |
| Mucositis oralGastrointestinal disorders | 6/7 | 1/19 |
| AnorexiaMetabolism and nutrition disorders | 5/7 | 2/19 |
| DiarrheaGastrointestinal disorders | 5/7 | 2/19 |
| White blood cell decreasedInvestigations | 5/7 | 9/19 |
| NauseaGastrointestinal disorders | 5/7 | 4/19 |
| neutrophil count decreasedInvestigations | 5/7 | 12/19 |
| Decreased platelet countBlood and lymphatic system disorders | 4/7 | 3/19 |
| VomitingGastrointestinal disorders | 4/7 | 2/19 |
| Age, Continuous(years) | Group A | Group B | Total |
|---|---|---|---|
| Median | 15 (13.5 to 19) | 10 (6.5 to 14.5) | 12.5 (7.5 to 15) |
| Sex: Female, Male(Participants) | Group A | Group B | Total |
|---|---|---|---|
| Female | 2 | 5 | 7 |
| Male | 5 | 10 | 15 |
| Ethnicity (NIH/OMB)(Participants) | Group A | Group B | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 3 | 3 |
| Not Hispanic or Latino | 7 | 12 | 19 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Group A | Group B | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 1 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 4 | 4 |
| White | 7 | 9 | 16 |
| More than one race | 0 | 1 | 1 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(Participants) | Group A | Group B | Total |
|---|---|---|---|
| United States | 7 | 15 | 22 |
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Studies of the drug tested here: Total Tumor mRNA-pulsed Tumor-specific Ex vivo-expanded Autologous Lymphocyte Transfer Cells