Trial Outcomes & Findings for Vaccine Immunotherapy for Recurrent Medulloblastoma and Primitive Neuroectodermal Tumor (NCT NCT01326104)

Last Updated: 2026-08-13

Results Overview

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)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

26 participants

Primary outcome timeframe

up to 12 months

Results posted on

2026-08-13

Participant Flow

Thirty-six patients were assessed for eligibility in the Phase II trial. Ten subjects were excluded; eight did not meet eligibility and two declined participation prior to allocation to intervention arm. Twenty-two patients received adoptive cellular therapy, DC plus ALT.

Participant milestones

Participant milestones
Measure	Group A High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Overall Study STARTED	7	19
Overall Study COMPLETED	7	15
Overall Study NOT COMPLETED	0	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Group A High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Overall Study progressed prior to vaccine	0	2
Overall Study unable to make vaccine	0	2

Baseline Characteristics

Vaccine Immunotherapy for Recurrent Medulloblastoma and Primitive Neuroectodermal Tumor

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group A n=7 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=15 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Total n=22 Participants Total of all reporting groups
Age, Continuous	15 years n=1 Participants	10 years n=1 Participants	12.5 years n=1 Participants
Sex: Female, Male Female	2 Participants n=1 Participants	5 Participants n=1 Participants	7 Participants n=1 Participants
Sex: Female, Male Male	5 Participants n=1 Participants	10 Participants n=1 Participants	15 Participants n=1 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=1 Participants	3 Participants n=1 Participants	3 Participants n=1 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=1 Participants	12 Participants n=1 Participants	19 Participants n=1 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants
Race (NIH/OMB) Asian	0 Participants n=1 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants
Race (NIH/OMB) Black or African American	0 Participants n=1 Participants	4 Participants n=1 Participants	4 Participants n=1 Participants
Race (NIH/OMB) White	7 Participants n=1 Participants	9 Participants n=1 Participants	16 Participants n=1 Participants
Race (NIH/OMB) More than one race	0 Participants n=1 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants
Region of Enrollment United States	7 Participants n=1 Participants	15 Participants n=1 Participants	22 Participants n=1 Participants

PRIMARY outcome

Timeframe: up to 12 months

Outcome measures

Outcome measures
Measure	Group A n=7 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=15 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
12 Month Progression-free Survival (PFS-12)	29 percentage Interval 8.9 to 92.0	0 percentage Interval 0.0 to 0.0	—

SECONDARY outcome

Timeframe: Baseline MRI (prior to Adoptive Cellular Therapy (ACT)) compared to post-ACT MRI (approximately 8 weeks post-baseline MRI)

Population: Patients who have measurable residual disease prior to immunotherapy. Group A only 1 person is evaluable and showed PR. Group B 4 persons are evaluable and showed PD. Among 5 evaluable from both groups A and B, only 1 showed PR (20%, CI: 0.5% - 72%); and 4 showed PD (80%, 95% CI: 28%- 99.5%) 95% CIs were calculated based on the exact binomial distribution.

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.

Outcome measures

Outcome measures
Measure	Group A n=7 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=15 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Objective Radiographic Response Rate CR + PR	1 Participants	0 Participants	—
Objective Radiographic Response Rate SD (Stable Disease)	0 Participants	0 Participants	—
Objective Radiographic Response Rate PD (Progressive Disease)	0 Participants	4 Participants	—
Objective Radiographic Response Rate Not Assessable	6 Participants	11 Participants	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: We have samples for 12 participants for this analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=5 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Cytokine Profile for IFNg Baseline	2.51 pg/mL Interval 1.51 to 3.36	4.61 pg/mL Interval 3.22 to 12.77	—
Change in Cytokine Profile for IFNg Cytokine change from baseline to 14 days	812.5 pg/mL Interval 26.01 to 838.53	10.35 pg/mL Interval 2.42 to 48.38	—
Change in Cytokine Profile for IFNg Cytokine change from baseline to 28 days	6.22 pg/mL Interval 5.2 to 7.61	27.45 pg/mL Interval 3.92 to 66.83	—

SECONDARY outcome

Timeframe: baseline up to 12 months

Population: Group A vs benchmark and Group B vs benchmark.

12-month OS calculated based on benchmark. OS-12 is the proportion of participants with OS at 12 months.

Outcome measures

Outcome measures
Measure	Group A n=7 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=15 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Overall Survival (OS) Rate	71.43 percentage of participants Interval 45.0 to 100.0	46.67 percentage of participants Interval 27.0 to 80.0	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: Twelve participant samples available for analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=12 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=5 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Type 1 Interferon	1.45 ratio Interval 0.57 to 3.66	0.67 ratio Interval 0.11 to 3.96	2.33 ratio Interval 0.56 to 9.7

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: 12 participants available for anaysis.

Outcome measures

Outcome measures
Measure	Group A n=12 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=5 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Type 2 Interferon	0.97 ratio Interval 0.4 to 2.34	0.29 ratio Interval 0.05 to 1.8	5.99 ratio Interval 0.45 to 79.2

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: 12 samples available for analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=12 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=5 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Percentage of CD8 Naive T Cells in PBMC 28 Days	-24.28 percentage of CD8 Naive T Cells in PBMC Interval -31.89 to -18.09	-22.33 percentage of CD8 Naive T Cells in PBMC Interval -33.21 to -13.84	-24.61 percentage of CD8 Naive T Cells in PBMC Interval -30.56 to -23.79
Change in Percentage of CD8 Naive T Cells in PBMC 14 Days	-15.52 percentage of CD8 Naive T Cells in PBMC Interval -33.25 to -8.88	-10.8 percentage of CD8 Naive T Cells in PBMC Interval -16.21 to -6.97	-21.67 percentage of CD8 Naive T Cells in PBMC Interval -38.72 to -12.0

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: We have samples for 12 participants for this analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=5 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Cytokine Profile for IL10 Cytokine change from baseline to 28 days	0.32 pg/mL Interval 0.16 to 0.48	0.62 pg/mL Interval 0.38 to 1.14	—
Change in Cytokine Profile for IL10 Baseline	0.28 pg/mL Interval 0.24 to 0.31	0.54 pg/mL Interval 0.3 to 1.41	—
Change in Cytokine Profile for IL10 Cytokine change from baseline to 14 days	3.31 pg/mL Interval 0.55 to 5.05	0.41 pg/mL Interval 0.32 to 0.56	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: We have samples for 12 participants for this analysis.

Outcome measures

Outcome measures
Measure	Group A n=5 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Cytokine Profile for IL12p70 Baseline	0.05 pg/mL Interval 0.03 to 0.08	0.08 pg/mL Interval 0.07 to 0.15	—
Change in Cytokine Profile for IL12p70 Cytokine change from baseline to 14 days	0.47 pg/mL Interval 0.21 to 0.59	0.10 pg/mL Interval 0.09 to 0.12	—
Change in Cytokine Profile for IL12p70 Cytokine change from baseline to 28 days	0.30 pg/mL Interval 0.24 to 0.37	0.11 pg/mL Interval 0.04 to 0.17	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: We have samples for 12 participants for this analysis.

Outcome measures

Outcome measures
Measure	Group A n=5 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Cytokine Profile for IL2 Baseline	0.16 pg/mL Interval 0.11 to 0.3	0.18 pg/mL Interval 0.12 to 0.24	—
Change in Cytokine Profile for IL2 Cytokine change from baseline to 14 days	2.63 pg/mL Interval 0.31 to 2.86	0.31 pg/mL Interval 0.22 to 0.35	—
Change in Cytokine Profile for IL2 Cytokine change from baseline to 28 days	0.40 pg/mL Interval 0.28 to 0.82	0.28 pg/mL Interval 0.15 to 0.36	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: We have samples for 12 participants for this analysis.

Outcome measures

Outcome measures
Measure	Group A n=5 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Cytokine Profile for IL4 Baseline	0.05 pg/mL Interval 0.05 to 0.05	0.03 pg/mL Interval 0.02 to 0.05	—
Change in Cytokine Profile for IL4 Cytokine change from baseline to 14 days	0.04 pg/mL Interval 0.03 to 0.1	0.06 pg/mL Interval 0.05 to 0.08	—
Change in Cytokine Profile for IL4 Cytokine change from baseline to 28 days	0.04 pg/mL Interval 0.02 to 0.06	0.06 pg/mL Interval 0.02 to 0.07	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: We have samples for 12 participants for this analysis.

Outcome measures

Outcome measures
Measure	Group A n=5 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Cytokine Profile for IL6 Baseline	0.80 pg/mL Interval 0.23 to 1.78	1.63 pg/mL Interval 0.94 to 1.83	—
Change in Cytokine Profile for IL6 Cytokine change from baseline to 14 days	5.54 pg/mL Interval 1.94 to 17.63	0.54 pg/mL Interval 0.17 to 1.77	—
Change in Cytokine Profile for IL6 Cytokine change from baseline to 28 days	1.63 pg/mL Interval 0.31 to 3.16	0.73 pg/mL Interval 0.38 to 1.72	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: We have samples for 12 participants for this analysis.

Outcome measures

Outcome measures
Measure	Group A n=5 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Cytokine Profile for TNFa Cytokine change from baseline to 14 days	2.80 pg/mL Interval 1.2 to 3.34	1.34 pg/mL Interval 0.99 to 1.43	—
Change in Cytokine Profile for TNFa Baseline	1.06 pg/mL Interval 0.98 to 1.12	1.24 pg/mL Interval 0.89 to 1.82	—
Change in Cytokine Profile for TNFa Cytokine change from baseline to 28 days	1.21 pg/mL Interval 0.78 to 1.72	1.64 pg/mL Interval 1.16 to 1.93	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: We have samples for 12 participants for this analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=5 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in TLR Activation Status Baseline	0.94 dimensionless enrichment score Interval 0.91 to 1.0	0.96 dimensionless enrichment score Interval 0.92 to 1.05	—
Change in TLR Activation Status Change from baseline to 14 days	1.11 dimensionless enrichment score Interval 1.1 to 1.12	1.00 dimensionless enrichment score Interval 0.97 to 1.09	—
Change in TLR Activation Status Change from baseline to 28 days	1.02 dimensionless enrichment score Interval 0.97 to 1.04	1.01 dimensionless enrichment score Interval 0.85 to 1.11	—

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: 12 samples available for analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=12 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=5 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Percentage of CD8 Memory T Cells in PBMC 28 Days	-12.12 percentage of CD8 Memory T Cell Interval -16.3 to 1.25	-11.83 percentage of CD8 Memory T Cell Interval -13.05 to -2.49	-12.42 percentage of CD8 Memory T Cell Interval -19.32 to 4.99
Change in Percentage of CD8 Memory T Cells in PBMC 14 Days	-1.91 percentage of CD8 Memory T Cell Interval -14.37 to 28.03	-11.92 percentage of CD8 Memory T Cell Interval -13.05 to -3.82	13.38 percentage of CD8 Memory T Cell Interval -15.69 to 49.53

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: 12 samples available for analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=12 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=5 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Percentage of CD4 Naive T Cells in PBMC 28 Days	-9.03 percentage of CD4 Naive T Cell Interval -12.42 to -4.11	-8.44 percentage of CD4 Naive T Cell Interval -10.31 to -4.94	-9.61 percentage of CD4 Naive T Cell Interval -12.83 to -3.29
Change in Percentage of CD4 Naive T Cells in PBMC 14 Days	9.02 percentage of CD4 Naive T Cell Interval -11.04 to 22.37	9.4 percentage of CD4 Naive T Cell Interval -10.31 to 11.86	8.64 percentage of CD4 Naive T Cell Interval -11.78 to 45.0

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: 12 samples available for analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=12 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=5 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Percentage of CD4 Memory T Cells in PBMC 28 Days	-6.4 percentage of CD4 Memory T Cells Interval -15.48 to -2.21	-17.11 percentage of CD4 Memory T Cells Interval -20.52 to -3.76	-5.79 percentage of CD4 Memory T Cells Interval -13.31 to -0.66
Change in Percentage of CD4 Memory T Cells in PBMC 14 Days	-3.94 percentage of CD4 Memory T Cells Interval -9.38 to -0.02	-0.04 percentage of CD4 Memory T Cells Interval -3.25 to 2.93	-5.79 percentage of CD4 Memory T Cells Interval -13.31 to -3.9

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: 12 samples available for analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=12 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=5 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Percentage of Treg T Cells in PBMC 28 Days	-13.24 percentage of Treg T Cells in PBMC Interval -28.56 to 5.01	-7.97 percentage of Treg T Cells in PBMC Interval -21.71 to -4.17	-16.37 percentage of Treg T Cells in PBMC Interval -35.41 to 27.41
Change in Percentage of Treg T Cells in PBMC 14 Days	12.99 percentage of Treg T Cells in PBMC Interval -9.09 to 33.03	19.99 percentage of Treg T Cells in PBMC Interval 13.22 to 26.2	5.31 percentage of Treg T Cells in PBMC Interval -32.32 to 43.8

SECONDARY outcome

Timeframe: baseline prior to immunotherapy to 28 days post-vaccine #1 (both TTRNA-xALT and TTRNA-DCs vaccines administered)

Population: 12 samples available for analysis.

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.

Outcome measures

Outcome measures
Measure	Group A n=12 Participants High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=5 Participants NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=7 Participants Group B: NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Change in Percentage of NK Cells in PBMC 14 Days	3.67 percentage of NK Cells in PBMC Interval -3.15 to 9.92	5.55 percentage of NK Cells in PBMC Interval 3.82 to 14.32	0.68 percentage of NK Cells in PBMC Interval -5.54 to 3.68
Change in Percentage of NK Cells in PBMC 28 Days	2.43 percentage of NK Cells in PBMC Interval -2.57 to 4.29	3.54 percentage of NK Cells in PBMC Interval -1.06 to 5.04	2.04 percentage of NK Cells in PBMC Interval -6.73 to 3.03

Adverse Events

Group A

Serious events: 4 serious events

Other events: 7 other events

Deaths: 6 deaths

Group B

Serious events: 5 serious events

Other events: 19 other events

Deaths: 18 deaths

Serious adverse events

Serious adverse events
Measure	Group A n=7 participants at risk High dose chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.	Group B n=19 participants at risk NMA Salvage chemotherapy plus peripheral blood stem cell transplant followed by TTRNA-xALT and TTRNA-DCs. TTRNA-xALT: TTRNA-xALT 3 x 10\^7/kg by intravenous injection once. TTRNA-DCs: TTRNA-DCs 1 x 10\^7 by intradermal injection every 2 weeks for 3 total doses.
Ear and labyrinth disorders Hearing impaired	28.6% 2/7 • Number of events 2 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	0.00% 0/19 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Blood and lymphatic system disorders Febrile Neutropenia	0.00% 0/7 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	10.5% 2/19 • Number of events 2 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Vascular disorders Hypotension	28.6% 2/7 • Number of events 2 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	0.00% 0/19 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Respiratory, thoracic and mediastinal disorders Hypoxia	14.3% 1/7 • Number of events 1 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	0.00% 0/19 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Respiratory, thoracic and mediastinal disorders Respiratory Arrest	0.00% 0/7 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	5.3% 1/19 • Number of events 1 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Cardiac disorders Cardiac Arrest	0.00% 0/7 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	5.3% 1/19 • Number of events 1 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
General disorders Pain	0.00% 0/7 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	10.5% 2/19 • Number of events 2 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Infections and infestations Colitis, Infectious	0.00% 0/7 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	5.3% 1/19 • Number of events 1 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Metabolism and nutrition disorders Acidosis	0.00% 0/7 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	5.3% 1/19 • Number of events 1 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Nervous system disorders Hydrocephalus	0.00% 0/7 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	5.3% 1/19 • Number of events 2 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Cardiac disorders Tachycardia	14.3% 1/7 • Number of events 1 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	0.00% 0/19 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.
Metabolism and nutrition disorders Hyperbilirubinemia	14.3% 1/7 • Number of events 1 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.	0.00% 0/19 • 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. An "AE" will be defined as any adverse change from the subject's pre-treatment baseline condition, including any clinical or laboratory test abnormality that occurs during the course of research after treatment has started. A summary of recorded AEs will be kept which will categorize the event by organ system, relationship to treatment, its grade of severity, and resolution. All AEs, SAEs and deaths that occurred during the study were reported and are represented in the tables.

Other adverse events

Additional Information

Kristine Wynne

University of Florida

Phone: 352-273-9727

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place