Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Ad... | NCT02991144 | Trialant
NCT02991144
Sponsor
Ultragenyx Pharmaceutical Inc
Status
Completed
Last Update Posted
Jan 26, 2023Actual
Enrollment
16Actual
Phase
Phase 1Phase 2
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
scAAV8OTC
Reactive Corticosteroid Taper Regimen
Prophylactic Corticosteroid Taper Regimen
Countries
United States
Canada
Spain
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT02991144
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
301OTC01
Secondary IDs
ID
Type
Description
Link
2016-001057-40
EudraCT Number
Brief Title
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
Official Title
A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency
Acronym
CAPtivate
Organization
Ultragenyx Pharmaceutical IncINDUSTRY
Status Module
Record Verification Date
Jan 2023
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Jul 31, 2017Actual
Primary Completion Date
Dec 16, 2021Actual
Completion Date
Dec 16, 2021Actual
First Submitted Date
Dec 9, 2016
First Submission Date that Met QC Criteria
Dec 12, 2016
First Posted Date
Dec 13, 2016Estimated
Results Waived
Not provided
Results First Submitted Date
Dec 9, 2022
Results First Submitted that Met QC Criteria
Dec 9, 2022
Results First Posted Date
Dec 23, 2022Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Jan 23, 2023
Last Update Posted Date
Jan 26, 2023Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Ultragenyx Pharmaceutical IncINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency. The primary objective of the study is to determine the safety of single intravenous (IV) doses of DTX301.
Detailed Description
Eligible participants will receive a single IV infusion of DTX301. Dose escalation will be conducted according to a model that uses the collected data to predict the safety profile of the dose in order to determine the optimal biological dose (OBD). The decision to proceed to the next dose cohort will be made after the data monitoring committee (DMC) has evaluated the safety data for all participants in a dosing cohort. Participants will be followed for 52 weeks after dosing. After completion of this study, participants will be asked to enroll in a 4-year extension study to evaluate the long term (a total of 5 years) safety and efficacy of DTX301.
This study was previously posted by Dimension Therapeutics, which has been acquired by Ultragenyx.
Conditions Module
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Keywords
Gene Transfer
OTC Deficiency
Urea Cycle Disorder
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 1Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
16Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Cohort 1: DTX301 2.0 × 10^12 GC/kg
Experimental
DTX301 (scAAV8OTC) 2.0 × 10^12 GC/kg will be administered as a single peripheral IV infusion. A reactive corticosteroid taper regimen will be administered to control transient vector-induced hepatic effects. Sodium acetate will be used as a tracer to measure the rate of ureagenesis.
Genetic: scAAV8OTC
Drug: Reactive Corticosteroid Taper Regimen
Cohort 2: DTX301 6.0 × 10^12 GC/kg
Experimental
DTX301 (scAAV8OTC) 6.0 × 10^12 GC/kg will be administered as a single peripheral IV infusion. A reactive corticosteroid taper regimen will be administered to control transient vector-induced hepatic effects. Sodium acetate will be used as a tracer to measure the rate of ureagenesis.
Genetic: scAAV8OTC
Drug: Reactive Corticosteroid Taper Regimen
Cohort 3: DTX301 1.0 × 10^13 GC/kg
Experimental
DTX301 (scAAV8OTC) 1.0 × 10^13 GC/kg will be administered as a single peripheral IV infusion. A reactive corticosteroid taper regimen will be administered to control transient vector-induced hepatic effects. Sodium acetate will be used as a tracer to measure the rate of ureagenesis.
A prophylactic corticosteroid taper regimen (oral prednisone [or prednisolone], 60 mg tapered over 9 weeks) will be administered before dosing with DTX301 (scAAV8OTC) to prevent or minimize transient vector-induced hepatic effects. DTX301 (scAAV8OTC) 1.0x10^13 GC/kg administered as a single peripheral IV infusion. Sodium acetate will be used as a tracer to measure the rate of ureagenesis.
Interventions
Name
Type
Description
Arm Group Labels
Other Names
scAAV8OTC
Genetic
non-replicating, recombinant scAAV8 encoding human ornithine transcarbamylase (OTC)
Cohort 1: DTX301 2.0 × 10^12 GC/kg
Cohort 2: DTX301 6.0 × 10^12 GC/kg
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation
AE: any untoward medical occurrence regardless of its causal relationship to study product. TEAE: any event not present before exposure to study product or any event already present that worsens in either intensity or frequency after exposure to study product. SAE: any event that results in death; is immediately life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; is an important medical event, according to the investigator. AE intensity was rated as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life threatening), or 5 (death) according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE). The relationship or association of the study product in causing or contributing to the AE was characterized as: unrelated; possible; probably; definite.
AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug up to End of Study (Week 52).
Secondary Outcomes
Measure
Description
Time Frame
Change From Baseline Over Time in Rate of Ureagenesis
The change from baseline in the rate of ureagenesis (as measured by the generation of [13C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 52 weeks after the IV administration of DTX301. Sodium acetate was used as a tracer to measure the rate of ureagenesis.
Rate of ureagenesis was derived in the following manner:
Derive area under the curve from time zero to 240 minutes (AUC0-240min) of absolute 13C-urea (µmol/l/min) estimated by the linear trapezoidal rule
Derive percent of normal AUC0-240min of absolute 13C-urea by dividing AUC0-240min of absolute 13C-urea (µmol*min/L) by 669.56 µmol*min/L (i.e. the AUC0-240min of absolute 13C-urea for an adult control)
Derive rate of ureagenesis by multiplying % of normal AUC0-240min of absolute 13C-urea by 300 µmol*h/kg (i.e. the approximate rate of ureagenesis in healthy adults).
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Key Inclusion Criteria:
Males and females ≥18 years of age with documented diagnosis of late onset (defined as first manifestation of signs and symptoms at ≥1 month of age) OTC deficiency, confirmed via enzymatic, biochemical, or molecular testing
Documented history of ≥1 symptomatic hyperammonemia event with ammonia ≥100 µmol/L.
Subject's OTC deficiency is stable as evidenced by either a) no clinical symptoms of hyperammonemia OR b) an ammonia level <100 µmol/L within the 4 week period preceding the Screening visit.
On ongoing daily stable dose of ammonia scavenger therapy for ≥4 weeks.
Males and all females of childbearing potential must be willing to use effective contraception at the time of administration of gene transfer and for the 52 weeks following administration of DTX301
Key Exclusion Criteria:
At Screening or Baseline (Day 0), plasma ammonia level ≥ 100 μmol/L for patients who historically maintain normal ammonia levels; OR plasma ammonia level ≥ 200 μmol/L for patients who historically are not able to fully control ammonia levels with baseline management; OR signs and symptoms of hyperammonemia.
Liver transplant, including hepatocyte cell therapy/transplant.
History of liver disease
Significant hepatic inflammation or cirrhosis
Serum creatinine >2.0 mg/dL.
Participation in another investigational medicine study (including another gene transfer trial) within 3 months of Screening
Pregnant or nursing
Note additional inclusion/exclusion criteria may apply, per protocol.
Accepts Healthy Volunteers
No
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
18 Years
Maximum Age
Not provided
Standard Ages
AdultOlder Adult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Medical Director
Ultragenyx Pharmaceutical Inc
Study Director
Locations
Facility
Status
City
State
ZIP
Country
Contacts
The Children's Hospital Colorado
Aurora
Colorado
80045
United States
Boston Children's Hospital
References Module
No data available
No data is available for this block.
IPD Sharing Statement Module
No data available
No data is available for this block.
Results Section
Participant Flow Module
Pre-assignment Details
A total of 27 subjects were screened for the study, 16 of whom were enrolled. Of the 16 participants enrolled, 5 participants discontinued the study before receiving DTX301, and were not assigned to a treatment arm.
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Cohort 1: DTX301 2.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 2.0 × 10^12 GC/kg administered as a single peripheral intravenous (IV) infusion.
Oral prednisone [or oral prednisolone] 60 mg/day week 1, 40 mg/day Week 2, 30 mg/day Weeks 3 and 4, tapered by 5 mg/week Week 5 and beyond until liver enzymes return to baseline levels.
Corticosteroid treatment will be considered when a participant's alanine aminotransferase (ALT) level exceeded the upper limit of normal (ULN) and the ALT increase was considered by the Investigator to be related to DTX301.
Cohort 1: DTX301 2.0 × 10^12 GC/kg
Cohort 2: DTX301 6.0 × 10^12 GC/kg
Cohort 3: DTX301 1.0 × 10^13 GC/kg
Prophylactic Corticosteroid Taper Regimen
Drug
Oral prednisone [or oral prednisolone] 60 mg/day at least 5 days prior to DTX301 administration, tapered over 9 weeks.
A prophylactic corticosteroid taper regimen will be administered to prevent or minimize transient vector-induced hepatic effects.
Baseline (Day 0), Weeks 6, 12, 20, 24, End of Study (Week 52). AUC was derived based on the following time points: 0.5, 1, 1.5, 2, 3, and 4 hours postdose.
Change From Baseline Over Time in Area Under the Curve From Time Zero to 24 Hours (AUC0-24) of Plasma Ammonia
Baseline (Day 0), Weeks 6, 12, 24, End of Study (Week 52). AUC was derived based on predose (time 0) and approximately 2, 4, 8, 12, 16, 20, 24 hours (±5 minutes) postdose.
Boston
Massachusetts
02115
United States
Icahn School of Medicine at Mount Sinai
New York
New York
10029-6508
United States
University Hospital Cleveland Medical Center/Case Western Reserve University
Cleveland
Ohio
44106
United States
Alberta's Children's Hospital
Calgary
Alberta
T3B 6A8
Canada
Hospital Clinico Universitario de Santiago
Santiago de Compostela
A Coruna
15706
Spain
Hospital Universitario de Cruzes
Barakaldo
Vizcaya
48903
Spain
National Hospital for Neurology & Neurosurgery
London
London City
WC1N 3BG
United Kingdom
Queen Elizabeth Hospital
Birmingham
B15 2TH
United Kingdom
DTX301 (scAAV8OTC) 6.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
FG002
Cohort 3: DTX301 1.0 × 10^13 GC/kg
DTX301 (scAAV8OTC) 1.0 × 10^13 GC/kg administered as a single peripheral IV infusion.
Oral prednisone (or prednisolone), 60 mg tapered over 9 weeks, initiated before dosing with DTX301 (scAAV8OTC) and administered through Week 4. DTX301 (scAAV8OTC) 1.0x10^13 GC/kg administered as a single peripheral IV infusion.
FG0003 subjects
FG0013 subjects
FG0023 subjects
FG0032 subjects
COMPLETED
FG0003 subjects
FG0013 subjects
FG0023 subjects
FG0032 subjects
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Cohort 1: DTX301 2.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 2.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
BG001
Cohort 2: DTX301 6.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 6.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
BG002
Cohort 3: DTX301 1.0 × 10^13 GC/kg
DTX301 (scAAV8OTC) 1.0 × 10^13 GC/kg administered as a single peripheral IV infusion.
Oral prednisone (or prednisolone), 60 mg tapered over 9 weeks, initiated before dosing with DTX301 (scAAV8OTC) and administered through Week 4. DTX301 (scAAV8OTC) 1.0x10^13 GC/kg administered as a single peripheral IV infusion.
BG004
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG0003
BG0013
BG0023
BG0032
BG00411
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG0023
ParticipantsBG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG002
Rate of Ureagenesis
Rate of ureagenesis (as measured by the generation of [13C]urea over 4 hours; see outcome measure 2 for details). Baseline is defined as the average of all non-missing assessments taken before the study drug administration. If the absolute difference of results between Screening and Day 1 was ≥ 25% of normal rate of ureagenesis, then the screening value is defined as the baseline.
Mean
Standard Deviation
μmol*h/kg
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG002
Area Under the Curve From Time Zero to 24 Hours (AUC0 24) of Plasma Ammonia
Baseline is defined as the Day -1 result.
participants with a baseline assessment
Mean
Standard Deviation
μmol*h/L
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation
AE: any untoward medical occurrence regardless of its causal relationship to study product. TEAE: any event not present before exposure to study product or any event already present that worsens in either intensity or frequency after exposure to study product. SAE: any event that results in death; is immediately life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; is an important medical event, according to the investigator. AE intensity was rated as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life threatening), or 5 (death) according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE). The relationship or association of the study product in causing or contributing to the AE was characterized as: unrelated; possible; probably; definite.
Safety Set: all participants who received DTX301.
Posted
Count of Participants
Participants
AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug up to End of Study (Week 52).
ID
Title
Description
OG000
Cohort 1: DTX301 2.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 2.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
OG001
Cohort 2: DTX301 6.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 6.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
OG002
Cohort 3: DTX301 1.0 × 10^13 GC/kg
DTX301 (scAAV8OTC) 1.0 × 10^13 GC/kg administered as a single peripheral IV infusion.
Oral prednisone (or prednisolone), 60 mg tapered over 9 weeks, initiated before dosing with DTX301 (scAAV8OTC) and administered through Week 4. DTX301 (scAAV8OTC) 1.0x10^13 GC/kg administered as a single peripheral IV infusion.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Any AE prior to dosing
Title
Measurements
OG0001
OG0011
OG0020
OG003
Secondary
Change From Baseline Over Time in Rate of Ureagenesis
The change from baseline in the rate of ureagenesis (as measured by the generation of [13C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 52 weeks after the IV administration of DTX301. Sodium acetate was used as a tracer to measure the rate of ureagenesis.
Rate of ureagenesis was derived in the following manner:
Derive area under the curve from time zero to 240 minutes (AUC0-240min) of absolute 13C-urea (µmol/l/min) estimated by the linear trapezoidal rule
Derive percent of normal AUC0-240min of absolute 13C-urea by dividing AUC0-240min of absolute 13C-urea (µmol*min/L) by 669.56 µmol*min/L (i.e. the AUC0-240min of absolute 13C-urea for an adult control)
Derive rate of ureagenesis by multiplying % of normal AUC0-240min of absolute 13C-urea by 300 µmol*h/kg (i.e. the approximate rate of ureagenesis in healthy adults).
Safety Set: all participants who received DTX301.
Posted
Mean
Standard Deviation
μmol*h/kg
Baseline (Day 0), Weeks 6, 12, 20, 24, End of Study (Week 52). AUC was derived based on the following time points: 0.5, 1, 1.5, 2, 3, and 4 hours postdose.
ID
Title
Description
OG000
Cohort 1: DTX301 2.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 2.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
OG001
Cohort 2: DTX301 6.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 6.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
Secondary
Change From Baseline Over Time in Area Under the Curve From Time Zero to 24 Hours (AUC0-24) of Plasma Ammonia
Safety Set: all participants who received DTX301. Participants with an assessment at given time point.
Posted
Mean
Standard Deviation
μmol*h/L
Baseline (Day 0), Weeks 6, 12, 24, End of Study (Week 52). AUC was derived based on predose (time 0) and approximately 2, 4, 8, 12, 16, 20, 24 hours (±5 minutes) postdose.
ID
Title
Description
OG000
Cohort 1: DTX301 2.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 2.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
OG001
Cohort 2: DTX301 6.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 6.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
OG002
Cohort 3: DTX301 1.0 × 10^13 GC/kg
DTX301 (scAAV8OTC) 1.0 × 10^13 GC/kg administered as a single peripheral IV infusion.
Oral prednisone (or prednisolone), 60 mg tapered over 9 weeks, initiated before dosing with DTX301 (scAAV8OTC) and administered through Week 4. DTX301 (scAAV8OTC) 1.0x10^13 GC/kg administered as a single peripheral IV infusion.
Time Frame
All Cause Mortality: from screening up to End of Study (Week 52). Adverse Events and Serious Adverse Events: From first dose of study drug up to End of Study (Week 52).
Description
2 enrolled participants died before receiving DTX301, and were not assigned to a treatment arm. These participants are reflected in the 'total' column of the all-cause mortality table.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
DTX301 2.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 2.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
0
3
0
3
3
3
EG001
DTX301 6.0 × 10^12 GC/kg
DTX301 (scAAV8OTC) 6.0 × 10^12 GC/kg administered as a single peripheral IV infusion.
0
3
0
3
3
3
EG002
DTX301 1.0 × 10^13 GC/kg
DTX301 (scAAV8OTC) 1.0 × 10^13 GC/kg administered as a single peripheral IV infusion.
Oral prednisone (or prednisolone), 60 mg tapered over 9 weeks, initiated before dosing with DTX301 (scAAV8OTC) and administered through Week 4. DTX301 (scAAV8OTC) 1.0x10^13 GC/kg administered as a single peripheral IV infusion.
0
2
1
2
2
2
EG004
Total
All Participants
2
16
1
11
11
11
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Hyperammonaemic Crisis
Metabolism and nutrition disorders
MedDRA 23.0
Systematic Assessment
There were 2 serious adverse events of hyperammonaemic crisis that occurred in 1 subject post-dosing.
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG0031 affected2 at risk
EG004
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Extrasystoles
Cardiac disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG0031 affected2 at risk
EG0041 affected11 at risk
Cushingoid
Endocrine disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Photophobia
Eye disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Abdominal Discomfort
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Retching
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Toothache
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Catheter Site Pain
General disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Fatigue
General disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Feeling Abnormal
General disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Feeling Jittery
General disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Gait Disturbance
General disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Pyrexia
General disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Gallbladder Polyp
Hepatobiliary disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Hepatitis
Hepatobiliary disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Gastroenteritis Viral
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Respiratory Tract Infection
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Upper Respiratory Tract Infection
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Exposure To Sars-Cov-2
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Hepatic Enzyme Increased
Investigations
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Liver Function Test Abnormal
Investigations
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Liver Function Test Increased
Investigations
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Hyperammonaemia
Metabolism and nutrition disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Hypophosphataemia
Metabolism and nutrition disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Back Pain
Musculoskeletal and connective tissue disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Pain In Extremity
Musculoskeletal and connective tissue disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Dizziness
Nervous system disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Headache
Nervous system disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0022 affected3 at risk
EG003
Hypoaesthesia
Nervous system disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Neuropathy Peripheral
Nervous system disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Sciatica
Nervous system disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Somnolence
Nervous system disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Aggression
Psychiatric disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Claustrophobia
Psychiatric disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Insomnia
Psychiatric disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Irritability
Psychiatric disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Phonophobia
Psychiatric disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Dysuria
Renal and urinary disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Nasal Congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Oropharyngeal Pain
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Upper-Airway Cough Syndrome
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0020 affected3 at risk
EG003
Acne
Skin and subcutaneous tissue disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0011 affected3 at risk
EG0020 affected3 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA 23.0
Systematic Assessment
EG0000 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Hypertension
Vascular disorders
MedDRA 23.0
Systematic Assessment
EG0001 affected3 at risk
EG0010 affected3 at risk
EG0021 affected3 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
Oral prednisone (or prednisolone), 60 mg tapered over 9 weeks, initiated before dosing with DTX301 (scAAV8OTC) and administered through Week 4. DTX301 (scAAV8OTC) 1.0x10^13 GC/kg administered as a single peripheral IV infusion.