Trial Outcomes & Findings for Carboxylesterase-Expressing Allogeneic Neural Stem Cells and Irinotecan Hydrochloride in Treating Patients With Recurrent High-Grade Gliomas (NCT NCT02192359)
NCT ID: NCT02192359
Last Updated: 2025-11-14
Results Overview
Toxicities will be graded using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. A DLT is defined as an adverse event that is related to the administration of NSCs and/or irinotecan, occurs during the first treatment cycle and meets any of the following: 1. Received less than 80% of study treatments except due to CNS toxicity 2. Grade 4 thrombocytopenia or anemia or neutropenia lasting \> 7 days 3. Febrile neutropenia with ANC \< 0.5 x10\^9/L 4. Grade 3 central nervous system (CNS) disorder lasting \> 7 days not attributed to tumor or surgery and not present at baseline 5. Second occurrence of grade 3 CNS disorder not attributed to tumor or surgery and not present at baseline 6. Any grade 4 CNS disorder not attributed to tumor or surgery and not present at baseline 7. Grade 3 toxicity despite therapy lasting \> 7 days 8. Grade 3 toxicity resulting in study agent discontinuation 9. Grade 4 toxicity, except grade 4 diarrhea responding to therapy within 3 days
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE1
Target enrollment
18 participants
Primary outcome timeframe
28 days post first dose of NSC treatment on day 1, cycle 1
Results posted on
2025-11-14
Participant Flow
Participant milestones
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
4
|
3
|
4
|
7
|
|
Overall Study
COMPLETED
|
3
|
3
|
3
|
6
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
1
|
1
|
Reasons for withdrawal
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Overall Study
Taken off study treatment due to persistent gr 2 decreased platelets requiring transfusions
|
0
|
0
|
1
|
0
|
|
Overall Study
Could not receive irinotecan due to low KPS score post surgery
|
1
|
0
|
0
|
0
|
|
Overall Study
Taken off study treatment to catheter infection not related to treatment
|
0
|
0
|
0
|
1
|
Baseline Characteristics
Carboxylesterase-Expressing Allogeneic Neural Stem Cells and Irinotecan Hydrochloride in Treating Patients With Recurrent High-Grade Gliomas
Baseline characteristics by cohort
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=4 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=4 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
n=7 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
0 Participants
n=44 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=10 Participants
|
3 Participants
n=10 Participants
|
4 Participants
n=20 Participants
|
6 Participants
n=45 Participants
|
17 Participants
n=44 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
1 Participants
n=45 Participants
|
1 Participants
n=44 Participants
|
|
Age, Continuous
|
49.5 years
n=10 Participants
|
56 years
n=10 Participants
|
56.5 years
n=20 Participants
|
44 years
n=45 Participants
|
51 years
n=44 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=10 Participants
|
2 Participants
n=10 Participants
|
1 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
3 Participants
n=44 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=10 Participants
|
1 Participants
n=10 Participants
|
3 Participants
n=20 Participants
|
7 Participants
n=45 Participants
|
15 Participants
n=44 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=10 Participants
|
1 Participants
n=10 Participants
|
3 Participants
n=20 Participants
|
4 Participants
n=45 Participants
|
9 Participants
n=44 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=10 Participants
|
2 Participants
n=10 Participants
|
1 Participants
n=20 Participants
|
3 Participants
n=45 Participants
|
9 Participants
n=44 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=10 Participants
|
1 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
1 Participants
n=44 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=10 Participants
|
2 Participants
n=10 Participants
|
4 Participants
n=20 Participants
|
7 Participants
n=45 Participants
|
17 Participants
n=44 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=45 Participants
|
0 Participants
n=44 Participants
|
|
Region of Enrollment
United States
|
4 Participants
n=10 Participants
|
3 Participants
n=10 Participants
|
4 Participants
n=20 Participants
|
7 Participants
n=45 Participants
|
18 Participants
n=44 Participants
|
|
Tumor Type
anaplastic astrocytoma, anaplastic oligodendroglioma, anaplastic oligoastrocytoma
|
1 Participants
n=10 Participants
|
0 Participants
n=10 Participants
|
1 Participants
n=20 Participants
|
2 Participants
n=45 Participants
|
4 Participants
n=44 Participants
|
|
Tumor Type
glioblastoma, gliosarcoma
|
3 Participants
n=10 Participants
|
3 Participants
n=10 Participants
|
3 Participants
n=20 Participants
|
5 Participants
n=45 Participants
|
14 Participants
n=44 Participants
|
PRIMARY outcome
Timeframe: 28 days post first dose of NSC treatment on day 1, cycle 1Population: 1 participant in each of dose levels 1, 3, and 4 was not evaluable for DLT because they were removed from the study due to adverse events not related to study treatment.
Toxicities will be graded using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. A DLT is defined as an adverse event that is related to the administration of NSCs and/or irinotecan, occurs during the first treatment cycle and meets any of the following: 1. Received less than 80% of study treatments except due to CNS toxicity 2. Grade 4 thrombocytopenia or anemia or neutropenia lasting \> 7 days 3. Febrile neutropenia with ANC \< 0.5 x10\^9/L 4. Grade 3 central nervous system (CNS) disorder lasting \> 7 days not attributed to tumor or surgery and not present at baseline 5. Second occurrence of grade 3 CNS disorder not attributed to tumor or surgery and not present at baseline 6. Any grade 4 CNS disorder not attributed to tumor or surgery and not present at baseline 7. Grade 3 toxicity despite therapy lasting \> 7 days 8. Grade 3 toxicity resulting in study agent discontinuation 9. Grade 4 toxicity, except grade 4 diarrhea responding to therapy within 3 days
Outcome measures
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
n=6 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Number of Participants Experiencing a Dose-limiting Toxicity (DLT)
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: Followed 30 days post treatment for all toxicities (min=33,max 142 days), up to 5 years for gene therapy toxicitiesGrade 3 or higher toxicity profile as assessed by the NCI CTCAE version version 4.0. Toxicities reported are possibly, probably or definitely related to NSCs.
Outcome measures
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=4 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=4 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
n=7 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to NSCs
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Followed 30 days post treatment for all toxicities (min=33,max 142 days), up to 5 years for gene therapy toxicitiesGrade 3 or higher toxicity profile as assessed by the NCI CTCAE version version 4.0. Toxicities reported are possibly, probably or definitely related to Irinotecan.
Outcome measures
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=4 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=4 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
n=7 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Platelet count decreased · Yes
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Platelet count decreased · No
|
3 Participants
|
3 Participants
|
4 Participants
|
7 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
White blood cell decreased · Yes
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
White blood cell decreased · No
|
3 Participants
|
3 Participants
|
4 Participants
|
7 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Meningitis · Yes
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Meningitis · No
|
4 Participants
|
2 Participants
|
4 Participants
|
7 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Hypophosphatemia · Yes
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Hypophosphatemia · No
|
4 Participants
|
2 Participants
|
4 Participants
|
7 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Brain abscess · Yes
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Brain abscess · No
|
4 Participants
|
3 Participants
|
3 Participants
|
7 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Catheter related infection · Yes
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Catheter related infection · No
|
4 Participants
|
3 Participants
|
4 Participants
|
6 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Fatigue · No
|
4 Participants
|
3 Participants
|
3 Participants
|
7 Participants
|
|
Number of Participants With Grade 3 or Higher Toxicity Profile Attributed to Irinotecan
Fatigue · Yes
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Pre-dose, at 90 minutes (just prior to the end of the infusion), and then at 30 minutes, 1, 2, 4, 8, 24, and 48 hours after the end of the infusion after day 1, cycle 1Population: All 4 participants in dose level 3 and one participant in dose level 4 did not have plasma samples collected.
Pharmacokinetic data from patients who undergo intracerebral microdialysis will be summarized using descriptive statistics. hCE1m6-NSC dose and liposomal SN-38 concentrations in brain interstitium using microdialysis data from the patients treated with the initial neural stem cells (NSC) doses and from the patients in the cohort treated with the highest NSC dose. Ratios are reported as ratio x 100.
Outcome measures
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=6 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Median Ratio of SN-38 Area Under the Curve (AUC) to CPT-11 AUC in Plasma
|
4.8 ratio x 100
Interval 3.8 to 5.5
|
0.8 ratio x 100
Interval 0.3 to 1.7
|
2.35 ratio x 100
Interval 1.2 to 3.8
|
—
|
SECONDARY outcome
Timeframe: Pre-dose, at 90 minutes (just prior to the end of the infusion), and then at 30 minutes, 1, 2, 4, 8, 24, and 48 hours after the end of the infusion after day 1, cycle 1Population: All 4 participants in dose level 3 did not have brain samples collected.
Pharmacokinetic data from patients who undergo intracerebral microdialysis will be summarized using descriptive statistics. hCE1m6-NSC dose and liposomal SN-38 concentrations in brain interstitium using microdialysis data from the patients treated with the initial neural stem cells (NSC) doses and from the patients in the cohort treated with the highest NSC dose. Ratio is reported as ratio x 100
Outcome measures
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=7 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Median Ratio of SN-38 AUC to CPT-11 AUC in the Brain
|
6.9 ratio x 100
Interval 6.0 to 9.3
|
8.8 ratio x 100
Interval 4.9 to 28.2
|
12.2 ratio x 100
Interval 3.7 to 26.3
|
—
|
SECONDARY outcome
Timeframe: Until death or disease progression, a median of 2 months, up to 6 monthsPopulation: 1 participant in dose levels 1 and 3 and 2 is dose level 4 were unevaluable for response due to not receiving the full treatment.
Clinical benefit is defined by participants achieving stable disease (SD), partial response (PR), or complete response (CR). CR: Complete disappearance of all enhancing disease (measurable and nonmeasureable) that is sustained for at least 4 weeks, stable or improved non-enhancing FLAIR/T2 lesions, no new lesions, off corticosteroids (physiologic replacement doses allowed), and neurologically stable or improved. PR: ≥ 50% decrease of all measurable enhancing lesions, sustained for at least 4 weeks, no progression of non-measurable disease, stable or improved non-enhancing FLAIR/T2 lesions, no new lesions, corticosteroid dose stable or reduced (compared to baseline), and neurologically stable or improved. SD: Does not qualify for CR, PR, or PD, stable non-enhancing FLAIR/T2 lesions, stable or reduced corticosteroids (compared to baseline), clinically stable.
Outcome measures
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=3 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
n=5 Participants
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Number of Participants With Clinical Benefit Defined by Response Assessment in Neuro-Oncology (RANO)
|
0 Participants
|
1 Participants
|
1 Participants
|
3 Participants
|
Adverse Events
Dose Level 1: 5x10^7 (1 Dose)
Serious events: 0 serious events
Other events: 4 other events
Deaths: 4 deaths
Dose Level 2: 5x10^7 (2 Doses)
Serious events: 1 serious events
Other events: 3 other events
Deaths: 3 deaths
Dose Level 3: 1x10^8 (2 Doses)
Serious events: 2 serious events
Other events: 4 other events
Deaths: 4 deaths
Dose Level 4: 1.5x10^8 (2 Doses)
Serious events: 5 serious events
Other events: 7 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=4 participants at risk
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 participants at risk
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=4 participants at risk
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
n=7 participants at risk
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Infections and infestations
Catheter related infection
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Infections and infestations
Brain abscess
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Infections and infestations
Meningitis
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Myelodysplastic syndrome
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Cerebrospinal fluid leakage
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Headache
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Intracranial hemorrhage
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Altered mental status
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
Confusion
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Vascular disorders
Hematoma
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
Other adverse events
| Measure |
Dose Level 1: 5x10^7 (1 Dose)
n=4 participants at risk
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (1 dose)
Irinotecan: 180 mg/m2
|
Dose Level 2: 5x10^7 (2 Doses)
n=3 participants at risk
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 5x10\^7 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 3: 1x10^8 (2 Doses)
n=4 participants at risk
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
Dose Level 4: 1.5x10^8 (2 Doses)
n=7 participants at risk
Carboxylesterase-expressing Allogeneic Neural Stem Cells: Given intracranially, 1.5x10\^8 (2 doses)
Irinotecan: 180 mg/m2
|
|---|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Scalp pain
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Periorbital edema
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
abrasion on forehead
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
Emotional lability
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
flat affect
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Renal and urinary disorders
Proteinuria
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Renal and urinary disorders
Urinary frequency
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Respiratory, thoracic and mediastinal disorders
Postnasal drip
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Respiratory, thoracic and mediastinal disorders
sinus congestion
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Bullous dermatitis
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Endocrine disorders
Cushingoid
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Eye disorders
Blurred vision
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Eye disorders
Photophobia
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Eye disorders
limited peripheral vision
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Bloating
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Constipation
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
71.4%
5/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
71.4%
5/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Nausea
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
57.1%
4/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
dry throat
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Gastrointestinal disorders
thrush
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Chills
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Edema face
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Facial pain
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Fatigue
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
85.7%
6/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Fever
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Gait disturbance
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
4/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Irritability
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Pain
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
3/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
4/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Edema limbs
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Localized edema
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
balance impairment
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
General disorders
generalized weakness
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Infections and infestations
Upper respiratory infection
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Injury, poisoning and procedural complications
Bruising
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Injury, poisoning and procedural complications
Fall
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Injury, poisoning and procedural complications
Intraoperative head and neck injury
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Injury, poisoning and procedural complications
Leakage from microdialysis catheters
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Investigations
Alanine aminotransferase increased
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Investigations
Alkaline phosphatase increased
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Investigations
Aspartate aminotransferase increase
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Investigations
Lymphocyte count decreased
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Investigations
Platelet count decreased
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Investigations
Weight loss
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Investigations
White blood cell decreased
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Alkalosis
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Anorexia
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
100.0%
4/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
57.1%
4/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hypokalemia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
57.1%
4/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hyponatremia
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
3/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
71.4%
5/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Metabolism and nutrition disorders
Glucose intolerance
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Ataxia
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
Restlessness
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Cerebrospinal fluid leakage
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Cognitive disturbance
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Concentration impairment
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Depressed level of consciousness
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Dizziness
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Dysarthria
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Dysphasia
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Headache
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
4/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
7/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Hypersomnia
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Lethargy
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Memory impairment
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Paresthesia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Seizure
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Somnolence
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Tremor
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Facial muscle weakness
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
balance impairment
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
facial twitching
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
pseudomeningocele
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
subgaleal fluid collection
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
Agitation
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
Anxiety
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
3/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
Confusion
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
Depression
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
28.6%
2/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Cardiac disorders
tachypnea
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Ear and labyrinth disorders
Tinnitus
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Blood and lymphatic system disorders
Anemia
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
57.1%
4/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Cardiac disorders
Sinus bradycardia
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
66.7%
2/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
71.4%
5/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Cardiac disorders
Sinus tachycardia
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness left-sided
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness right-sided
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Joint range of motion decreased ce
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness upper limb
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Musculoskeletal and connective tissue disorders
Right upper extremity twitching
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Nervous system disorders
Aphonia
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
cutibacterium (propionibacterium) acnes
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Skin and subcutaneous tissue disorders
erythema at site of incision
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
14.3%
1/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Vascular disorders
Hematoma
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
25.0%
1/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
0.00%
0/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Vascular disorders
Hypertension
|
75.0%
3/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
3/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
4/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
100.0%
7/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
|
Vascular disorders
Hypotension
|
0.00%
0/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
33.3%
1/3 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
50.0%
2/4 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
42.9%
3/7 • Followed 30 days post treatment for all toxicities (min=33,max 142 days) up to 5 years for gene therapy toxicities
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place