Trial Outcomes & Findings for A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH) (NCT NCT02651675)
NCT ID: NCT02651675
Last Updated: 2023-07-13
Results Overview
Physical examinations; Clinical laboratory parameters; and adverse event reporting
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
9 participants
Primary outcome timeframe
Up to 24 weeks
Results posted on
2023-07-13
Participant Flow
Participant milestones
| Measure |
Cohort 1
2.5E12 GC (genome copies)/kg (kilogram body weight (E means exponential constant)
|
Cohort 2
7.5E12 GC/kg body weight
|
Cohort 2 Expansion
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
|
|---|---|---|---|
|
Overall Study
STARTED
|
3
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
3
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
1
|
Reasons for withdrawal
| Measure |
Cohort 1
2.5E12 GC (genome copies)/kg (kilogram body weight (E means exponential constant)
|
Cohort 2
7.5E12 GC/kg body weight
|
Cohort 2 Expansion
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
|
|---|---|---|---|
|
Overall Study
Death
|
0
|
0
|
1
|
Baseline Characteristics
A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
Baseline characteristics by cohort
| Measure |
Cohort 1
n=3 Participants
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: a novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=3 Participants
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: a novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=3 Participants
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: a novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
9 Participants
n=4 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
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
8 Participants
n=4 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
|
|
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
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 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
|
|
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
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
7 Participants
n=4 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
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Region of Enrollment
Canada
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
Netherlands
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Region of Enrollment
Italy
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Up to 24 weeksPhysical examinations; Clinical laboratory parameters; and adverse event reporting
Outcome measures
| Measure |
Cohort 1
n=3 Participants
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=3 Participants
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=3 Participants
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
|---|---|---|---|
|
Number of Participants With IP (Investigational Product) Related Adverse Events
|
1 Participants
|
3 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 18 weeks, 12 weeks for cohort 1 only, compared to baselinePercent change in LDL-C compared to baseline
Outcome measures
| Measure |
Cohort 1
n=3 Participants
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=3 Participants
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=3 Participants
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
|---|---|---|---|
|
Percent Change in LDL-C
|
7.21 percentage of change from baseline
Standard Deviation 12.24
|
27.35 percentage of change from baseline
Standard Deviation 32.51
|
8.98 percentage of change from baseline
Standard Deviation 31.34
|
SECONDARY outcome
Timeframe: 18 weeks, 12 weeks for cohort 1 only, compared to baselinetotal cholesterol (TC); non-high density lipoprotein cholesterol (non-HDL-C); HDL-C; fasting triglycerides (TG); overflow density lipoprotein cholesterol (VLDL-C); lipoprotein(a) (Lp(a)); apolipoprotein B (apoB) and apolipoprotein A-I (apo A-I)
Outcome measures
| Measure |
Cohort 1
n=3 Participants
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=3 Participants
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=3 Participants
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
|---|---|---|---|
|
Percent Change in Lipid Parameters Compared to Baseline Values
lipoprotein(a) (Lp(a))
|
-0.3 percentage of change from baseline
Standard Deviation 26.31
|
-31.4 percentage of change from baseline
Standard Deviation 16.08
|
30.3 percentage of change from baseline
Standard Deviation 17.83
|
|
Percent Change in Lipid Parameters Compared to Baseline Values
apolipoprotein B (apoB)
|
4.53 percentage of change from baseline
Standard Deviation 9.61
|
21.15 percentage of change from baseline
Standard Deviation 25.21
|
11.36 percentage of change from baseline
Standard Deviation 26.43
|
|
Percent Change in Lipid Parameters Compared to Baseline Values
total cholesterol (TC)
|
7.89 percentage of change from baseline
Standard Deviation 15.72
|
24.91 percentage of change from baseline
Standard Deviation 34.45
|
7.17 percentage of change from baseline
Standard Deviation 37.35
|
|
Percent Change in Lipid Parameters Compared to Baseline Values
overflow density lipoprotein cholesterol (VLDL-C)
|
32.63 percentage of change from baseline
Standard Deviation 85.34
|
54.17 percentage of change from baseline
Standard Deviation 135.53
|
-5.46 percentage of change from baseline
Standard Deviation 111.43
|
|
Percent Change in Lipid Parameters Compared to Baseline Values
fasting triglycerides (TG)
|
7.92 percentage of change from baseline
Standard Deviation 26.43
|
-9.16 percentage of change from baseline
Standard Deviation 8.23
|
15.60 percentage of change from baseline
Standard Deviation 10.46
|
|
Percent Change in Lipid Parameters Compared to Baseline Values
HDL-C
|
9.10 percentage of change from baseline
Standard Deviation 22.58
|
7.92 percentage of change from baseline
Standard Deviation 21.68
|
-10.07 percentage of change from baseline
Standard Deviation 25.12
|
|
Percent Change in Lipid Parameters Compared to Baseline Values
non-high density lipoprotein cholesterol (non-HDL-C)
|
8.43 percentage of change from baseline
Standard Deviation 15.93
|
27.06 percentage of change from baseline
Standard Deviation 36.63
|
8.26 percentage of change from baseline
Standard Deviation 38.16
|
|
Percent Change in Lipid Parameters Compared to Baseline Values
apolipoprotein A-I (apo A-I)
|
-2.71 percentage of change from baseline
Standard Deviation 15.18
|
12.25 percentage of change from baseline
Standard Deviation 18.90
|
-16.18 percentage of change from baseline
Standard Deviation 11.04
|
SECONDARY outcome
Timeframe: up to 104 weeksPhysical examinations; Clinical laboratory parameters; and adverse event reporting
Outcome measures
| Measure |
Cohort 1
n=3 Participants
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=3 Participants
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=3 Participants
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
|---|---|---|---|
|
Number of Participants With IP Related Adverse Events
|
1 Participants
|
3 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: up to 104 weeksAmount of virus secreted in urine
Outcome measures
| Measure |
Cohort 1
n=3 Participants
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=1 Participants
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=1 Participants
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
|---|---|---|---|
|
Amount of Vector Shedding, Urine
|
25.0 GC/12 μL (microliter) specimen) (urine)
Standard Deviation 0.0
|
25.0 GC/12 μL (microliter) specimen) (urine)
Standard Deviation NA
No Standard Deviation could be calculated as there is only one patient assessable at this timepoint
|
0 GC/12 μL (microliter) specimen) (urine)
Standard Deviation NA
No Standard Deviation could be calculated as there is only one patient assessable at this timepoint
|
SECONDARY outcome
Timeframe: up to 104 weeksAmount of virus secreted in plasma
Outcome measures
| Measure |
Cohort 1
n=3 Participants
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=1 Participants
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=1 Participants
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene
|
|---|---|---|---|
|
Amount of Vector Shedding, Plasma
|
50.0 GC/ μg (microgram) DNA
Standard Deviation 0.0
|
50.0 GC/ μg (microgram) DNA
Standard Deviation NA
No Standard Deviation could be calculated as there is only one patient assessable at this timepoint
|
0.0 GC/ μg (microgram) DNA
Standard Deviation NA
No Standard Deviation could be calculated as there is only one patient assessable at this timepoint
|
Adverse Events
Cohort 1
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 2
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2 Expansion
Serious events: 1 serious events
Other events: 2 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Cohort 1
n=3 participants at risk
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=3 participants at risk
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=3 participants at risk
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV) vector with human low-density lipoprotein receptor (hLDLR) gene
|
|---|---|---|---|
|
Product Issues
device failure
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Cardiac disorders
cardiac arrest
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Infections and infestations
pnuemonia
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Respiratory, thoracic and mediastinal disorders
acute respiratory failure
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Cardiac disorders
acute coronary syndrome
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Respiratory, thoracic and mediastinal disorders
dyspnoea
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
Vascular disorders
hypotension
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
Cardiac disorders
Angina pectoris
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
General disorders
chest pain
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
Infections and infestations
appendicitis
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
Investigations
hepatic enzyme increased
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
Cardiac disorders
Acute left ventricular failure
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
Other adverse events
| Measure |
Cohort 1
n=3 participants at risk
2.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2
n=3 participants at risk
7.5E12 GC/kg body weight
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV) vector with human low-density lipoprotein receptor (hLDLR) gene
|
Cohort 2 Expansion
n=3 participants at risk
7.5E12 GC/kg body weight
DSMB (Data Safety Monitoring Board) approved expansion of Dose 2 cohort, 3 additional subjects enrolled and received prophylactic corticosteroids
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
AAV directed hLDLR gene therapy: A novel adeno-associated viral (AAV) vector with human low-density lipoprotein receptor (hLDLR) gene
|
|---|---|---|---|
|
Cardiac disorders
Angina Pectoris
|
66.7%
2/3 • Number of events 4 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
Cardiac disorders
Acute Coronary Syndrome
|
33.3%
1/3 • Number of events 2 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Cardiac disorders
Palpitations
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
100.0%
3/3 • Number of events 3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
General disorders
Chest Pain
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
66.7%
2/3 • Number of events 2 • up to 104 weeks
regular investigator assessment
|
|
General disorders
Fatigue
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
66.7%
2/3 • Number of events 2 • up to 104 weeks
regular investigator assessment
|
|
General disorders
Pyrexia
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
General disorders
Swelling Face
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Investigations
Hepatic Enzyme Increased
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
100.0%
3/3 • Number of events 12 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
66.7%
2/3 • Number of events 2 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
|
Nervous system disorders
Dizziness
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
66.7%
2/3 • Number of events 2 • up to 104 weeks
regular investigator assessment
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
|
Psychiatric disorders
Restlessness
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
66.7%
2/3 • Number of events 2 • up to 104 weeks
regular investigator assessment
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
0.00%
0/3 • up to 104 weeks
regular investigator assessment
|
33.3%
1/3 • Number of events 1 • up to 104 weeks
regular investigator assessment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place