Trial Outcomes & Findings for A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease (NCT NCT04669535)

Last Updated: 2025-09-02

Results Overview

The measure of total numbers of total treatment emergent adverse events (TEAEs), serious adverse events (SAEs) after treatment with the vector whether related or unrelated to treatment as well as the number of adverse events (AEs) related to AXO-AAV-GM2 vector and neurosurgical procedures.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

9 participants

Primary outcome timeframe

At least through 24 weeks post-treatment (infantile-onset participants) to 48 weeks post-treatment (juvenile-onset participants). All AEs are reported through the transition of the study to long-term follow-up.

Results posted on

2025-09-02

Participant Flow

This study is a dose-escalation study in children affected with GM2 gangliosidosis who were treated in four cohorts at dosage levels ranging from 1.42E+14 to 3.56E+14 vg per patient. All subjects are treated with a 1:1 ratio of AAVrh8-HEXA and AAVrh8-HEXB, administered via bilateral thalamic (BiTh) and dual intracisterna magna (ICM)/intrathecal (IT) administration into the cerebrospinal fluid (CSF). The BiTh injection volume and vector dose was doubled between each cohort.

The study was initially designed as a 2-stage study with dose escalation in stage 1 and safety and efficacy in stage 2, with the optimal dose identified in stage 1 however due to lack of funding only stage 1 was completed in this study. No subjects were enrolled in stage 2.

Participant milestones

Participant milestones
Measure	AXO-AAV-GM2 Starting Dose Subjects treated with AXO-AAV-GM2 Starting Dose infusion	AXO-AAV-GM2 Low Dose Subjects treated with AXO-AAV-GM2 Low Dose infusion	AXO-AAV-Middle Dose Subjects treated with AXO-AAV-GM2 Middle Dose infusion	AXO-AAV-GM2 High Dose Subjects treated with AXO-AAV-GM2 High Dose infusion
Overall Study STARTED	1	3	3	2
Overall Study COMPLETED	0	2	3	2
Overall Study NOT COMPLETED	1	1	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	AXO-AAV-GM2 Starting Dose Subjects treated with AXO-AAV-GM2 Starting Dose infusion	AXO-AAV-GM2 Low Dose Subjects treated with AXO-AAV-GM2 Low Dose infusion	AXO-AAV-Middle Dose Subjects treated with AXO-AAV-GM2 Middle Dose infusion	AXO-AAV-GM2 High Dose Subjects treated with AXO-AAV-GM2 High Dose infusion
Overall Study Death	1	1	0	0

Baseline Characteristics

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	AXO-AAV-GM2 Starting Dose n=1 Participants Subjects treated with AXO-AAV-GM2 Starting Dose Infusion	AXO-AAV-GM2 Low Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Low Dose Infusion	AXO-AAV-GM2 Middle Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Mid Dose Infusion	AXO-AAV-GM2 High Dose n=2 Participants Subjects treated with AXO-AAV-GM2 High Dose Infusion	Total n=9 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	2 Participants n=4 Participants	9 Participants n=21 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	2 Participants n=4 Participants	8 Participants n=21 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	2 Participants n=4 Participants	8 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	2 Participants n=4 Participants	8 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Disease Condition Tay-Sachs Disease (Hex A mutation)	0 Participants n=5 Participants	3 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants	7 Participants n=21 Participants
Disease Condition Sandhoff Disease (Hex B mutation)	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants
Disease Type Infantile Onset	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants	6 Participants n=21 Participants
Disease Type Juvenile Onset	0 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	0 Participants n=4 Participants	3 Participants n=21 Participants

PRIMARY outcome

Timeframe: At least through 24 weeks post-treatment (infantile-onset participants) to 48 weeks post-treatment (juvenile-onset participants). All AEs are reported through the transition of the study to long-term follow-up.

Population: All subjects treated with AXO-AAV-GM2 at all doses

Outcome measures

Outcome measures
Measure	AXO-AAV-GM2 Starting Dose n=1 Participants Subjects treated with AXO-AAV-GM2 Starting Dose	AXO-AAV-GM2 Low Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Low Dose	AXO-AAV-GM2 Middle Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Middle Dose	AXO-AAV-GM2 High Dose n=2 Participants Subjects treated with AXO-AAV-GM2 High Dose
Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events Total number of TEAEs	21 number of events	43 number of events	51 number of events	18 number of events
Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events Total Number of Treatment Emergent SAEs	1 number of events	9 number of events	8 number of events	1 number of events
Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events AEs Related to Vector	0 number of events	4 number of events	6 number of events	5 number of events
Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events AEs Related to Neurosurgery	2 number of events	4 number of events	4 number of events	6 number of events

SECONDARY outcome

Timeframe: 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants)

Any abnormal vital signs that the investigator determined was clinically significant was recorded as an adverse event.

Outcome measures

Outcome measures
Measure	AXO-AAV-GM2 Starting Dose n=1 Participants Subjects treated with AXO-AAV-GM2 Starting Dose	AXO-AAV-GM2 Low Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Low Dose	AXO-AAV-GM2 Middle Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Middle Dose	AXO-AAV-GM2 High Dose n=2 Participants Subjects treated with AXO-AAV-GM2 High Dose
Number of Participants With Clinically Significant Abnormal Vital Signs Per Investigator Assessment	0 Participants	0 Participants	0 Participants	1 Participants

SECONDARY outcome

Timeframe: 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants)

Physical exam findings that the investigator determined to be clinically significant and related to the treatment with AXO-AAV-GM2, surgical procedure or immune suppression.

Outcome measures

Outcome measures
Measure	AXO-AAV-GM2 Starting Dose n=1 Participants Subjects treated with AXO-AAV-GM2 Starting Dose	AXO-AAV-GM2 Low Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Low Dose	AXO-AAV-GM2 Middle Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Middle Dose	AXO-AAV-GM2 High Dose n=2 Participants Subjects treated with AXO-AAV-GM2 High Dose
Number of Participants With Clinically Significant Abnormal Physical Exam Per Investigator Assessment Mucositis	0 Participants	2 Participants	2 Participants	0 Participants
Number of Participants With Clinically Significant Abnormal Physical Exam Per Investigator Assessment Surgical Site Swelling	0 Participants	0 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Abnormal Physical Exam Per Investigator Assessment Dystonia	0 Participants	1 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Abnormal Physical Exam Per Investigator Assessment Surgical Wound Drainage	0 Participants	0 Participants	1 Participants	1 Participants
Number of Participants With Clinically Significant Abnormal Physical Exam Per Investigator Assessment Wound Dehiscence	0 Participants	1 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Abnormal Physical Exam Per Investigator Assessment Oral Candidiasis	1 Participants	0 Participants	2 Participants	0 Participants

SECONDARY outcome

Timeframe: 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants)

Population: Subjects treated with AXO-AAV-GM2

Abnormal laboratory findings in subjects treated with AXO-AAV-GM2 that were felt to be clinically significant and reported as adverse events.

Outcome measures

Outcome measures
Measure	AXO-AAV-GM2 Starting Dose n=1 Participants Subjects treated with AXO-AAV-GM2 Starting Dose	AXO-AAV-GM2 Low Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Low Dose	AXO-AAV-GM2 Middle Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Middle Dose	AXO-AAV-GM2 High Dose n=2 Participants Subjects treated with AXO-AAV-GM2 High Dose
Number of Participants With Clinically Significant Abnormal Clinical Safety Laboratory Tests on Blood/Urine/CSF Hypokalemia	0 Participants	0 Participants	1 Participants	1 Participants
Number of Participants With Clinically Significant Abnormal Clinical Safety Laboratory Tests on Blood/Urine/CSF Abnormal results of liver function studies	0 Participants	1 Participants	2 Participants	2 Participants
Number of Participants With Clinically Significant Abnormal Clinical Safety Laboratory Tests on Blood/Urine/CSF Anemia	1 Participants	1 Participants	1 Participants	1 Participants
Number of Participants With Clinically Significant Abnormal Clinical Safety Laboratory Tests on Blood/Urine/CSF Neutropenia	1 Participants	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants)

Population: Subjects with clinically significant serum cellular and antibody immune response to vector capsid/transgene

Subjects with clinically significant serum cellular and antibody immune response to vector capsid/transgene that were reported as adverse events and treated with an increase in steroids.

Outcome measures

Outcome measures
Measure	AXO-AAV-GM2 Starting Dose n=1 Participants Subjects treated with AXO-AAV-GM2 Starting Dose	AXO-AAV-GM2 Low Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Low Dose	AXO-AAV-GM2 Middle Dose n=3 Participants Subjects treated with AXO-AAV-GM2 Middle Dose	AXO-AAV-GM2 High Dose n=2 Participants Subjects treated with AXO-AAV-GM2 High Dose
Serum Cellular and Antibody Immune Response to Vector Capsid/Transgene	0 Participants	1 Participants	3 Participants	2 Participants

Adverse Events

AXO-AAV-GM2 Starting Dose

Serious events: 1 serious events

Other events: 1 other events

Deaths: 1 deaths

AXO-AAV-GM2 Low Dose

Serious events: 3 serious events

Other events: 3 other events

Deaths: 1 deaths

AXO-AAV-GM2 Middle Dose

Serious events: 3 serious events

Other events: 3 other events

Deaths: 0 deaths

AXO-AAV-GM2 High Dose

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	AXO-AAV-GM2 Starting Dose n=1 participants at risk All subjects treated with AXO-AAV-GM2 Starting Dose	AXO-AAV-GM2 Low Dose n=3 participants at risk All subjects treated with AXO-AAV-GM2 Low Dose	AXO-AAV-GM2 Middle Dose n=3 participants at risk All subjects treated with AXO-AAV-GM2 Middle Dose	AXO-AAV-GM2 High Dose n=2 participants at risk All subjects treated with AXO-AAV-GM2 High Dose
General disorders Pyrexia	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Surgical and medical procedures Wound dehiscence	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Infections and infestations Aspiration pneumonia	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Infections and infestations Clostridium difficile infection	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Infections and infestations Covid-19	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Infections and infestations Influenza A	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Infections and infestations Pneumonia	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	66.7% 2/3 • Number of events 2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Nervous system disorders Death due to disease progression	100.0% 1/1 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Nervous system disorders Mental status changes	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Nervous system disorders Seizures	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	50.0% 1/2 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Nervous system disorders Neurological decompensation	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Respiratory, thoracic and mediastinal disorders Bronchiolitis	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.
Respiratory, thoracic and mediastinal disorders Bronchitis	0.00% 0/1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/3 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	33.3% 1/3 • Number of events 1 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.	0.00% 0/2 • Adverse events were captured from screening prior to treatment until the subjects were all moved to long-term follow-up and the initial study was closed. This time ranged from 9 months for the most recently treated patient to 3 years for the patient who was the furthest from their treatment date at the time of moving to long-term follow-up.

Other adverse events

Additional Information

Dr. Terence Flotte

UMass Chan Medical School

Phone: 508-856-8992

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place