Trial Outcomes & Findings for Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B (NCT NCT02618915)
NCT ID: NCT02618915
Last Updated: 2018-11-14
Results Overview
An AE was defined as any untoward medical occurrence in a participant enrolled into this study (from the time the participant signed the informed consent form until his or her exit from the study), regardless of its causal relationship to study treatment. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
6 participants
Primary outcome timeframe
up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2)
Results posted on
2018-11-14
Participant Flow
Participant milestones
| Measure |
DTX101, Cohort 1
a single peripheral intravenous (IV) infusion of 1.6 x 10\^12 genome copies (GC)/kg DTX101
|
DTX101, Cohort 2
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Baseline characteristics by cohort
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
18-49 years
|
0 participants
n=5 Participants
|
3 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Age, Customized
50-84 years
|
3 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Factor IX (FIX) Activity
|
1.67 IU/dL
STANDARD_DEVIATION 0.577 • n=5 Participants
|
0.87 IU/dL
STANDARD_DEVIATION 0.709 • n=7 Participants
|
1.27 IU/dL
STANDARD_DEVIATION 0.566 • n=5 Participants
|
PRIMARY outcome
Timeframe: up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2)Population: Safety Set: all participants who received DTX101 including participants who received a partial dose or failed infusion.
An AE was defined as any untoward medical occurrence in a participant enrolled into this study (from the time the participant signed the informed consent form until his or her exit from the study), regardless of its causal relationship to study treatment. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product.
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Number of Participants With Adverse Events (AEs), Treatment-Related Adverse Events (TEAEs), and Serious AEs (SAEs)
All TEAEs
|
3 Participants
|
3 Participants
|
|
Number of Participants With Adverse Events (AEs), Treatment-Related Adverse Events (TEAEs), and Serious AEs (SAEs)
All Serious TEAEs
|
1 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 6Population: As Treated Set: all participants who received any amount of DTX101.
Peak plasma level of FIX after IV administration as determined by the activated partial thromboplastin time (aPTT) clot-based assay. Change from baseline: postbaseline value - baseline value. For the change from baseline, only participants with a value at both baseline visit and the specific postbaseline visit were included.
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Change From Baseline in FIX Activity at Week 6
|
5.00 IU/dL
Standard Deviation 1.732
|
9.80 IU/dL
Standard Deviation 4.687
|
SECONDARY outcome
Timeframe: Week 0 to Week 52Population: As Treated Set: all participants who received any amount of DTX101.
The number of bleeding episodes per participant was recorded, and the annualized number of bleeding episodes was calculated.
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Annualized Bleeding Rate
|
8.7 bleeding episodes/year
Standard Deviation 5.53
|
5.0 bleeding episodes/year
Standard Deviation 1.00
|
SECONDARY outcome
Timeframe: Baseline, Weeks 2, 4, 6, 8, 12, 16, 24, 32, 40, End of Study (Week 52 for Cohort 1, Week 44 for Cohort 2)/Early WithdrawalPopulation: As Treated Set: all participants who received any amount of DTX101.
Peak plasma level of FIX after IV administration as determined by the aPTT clot-based assay. Change from baseline: postbaseline value - baseline value. For the change from baseline, only participants with a value at both baseline visit and the specific postbaseline visit were included. Participants were not required to stop prophylactic treatment with recombinant FIX until after Week 4 and may have been restarted on their prophylactic recombinant FIX treatment after Week 14.
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Change From Baseline in FIX Activity Over Time
Week 2
|
16.33 IU/dL
Standard Deviation 14.503
|
10.13 IU/dL
Standard Deviation 7.988
|
|
Change From Baseline in FIX Activity Over Time
Week 4
|
8.67 IU/dL
Standard Deviation 7.234
|
14.47 IU/dL
Standard Deviation 10.835
|
|
Change From Baseline in FIX Activity Over Time
Week 6
|
5.00 IU/dL
Standard Deviation 1.732
|
9.80 IU/dL
Standard Deviation 4.687
|
|
Change From Baseline in FIX Activity Over Time
Week 8
|
14.67 IU/dL
Standard Deviation 17.786
|
11.80 IU/dL
Standard Deviation 6.646
|
|
Change From Baseline in FIX Activity Over Time
Week 12
|
11.33 IU/dL
Standard Deviation 4.933
|
5.80 IU/dL
Standard Deviation 2.207
|
|
Change From Baseline in FIX Activity Over Time
Week 16
|
7.67 IU/dL
Standard Deviation 3.215
|
5.47 IU/dL
Standard Deviation 3.066
|
|
Change From Baseline in FIX Activity Over Time
Week 24
|
2.33 IU/dL
Standard Deviation 1.528
|
2.47 IU/dL
Standard Deviation 2.214
|
|
Change From Baseline in FIX Activity Over Time
Week 32
|
9.33 IU/dL
Standard Deviation 11.846
|
10.13 IU/dL
Standard Deviation 2.702
|
|
Change From Baseline in FIX Activity Over Time
Week 40
|
15.33 IU/dL
Standard Deviation 23.965
|
12.80 IU/dL
Standard Deviation 16.008
|
|
Change From Baseline in FIX Activity Over Time
End of Study/Early Withdrawal
|
1.67 IU/dL
Standard Deviation 0.577
|
22.47 IU/dL
Standard Deviation 1.380
|
SECONDARY outcome
Timeframe: Week 0 to Week 52Population: As Treated Set: all participants who received any amount of DTX101.
The use of on-demand FIX replacement therapy was recorded by dose (IU/kg) administered, and the annualized use of FIX replacement therapy was calculated. Participants were not required to stop prophylactic treatment with recombinant FIX until after Week 4 and may have been restarted on their prophylactic recombinant FIX treatment after Week 14.
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Annualized FIX Replacement Therapy
|
350115.2 IU/kg
Standard Deviation 522106.19
|
64246.5 IU/kg
Standard Deviation 805.34
|
SECONDARY outcome
Timeframe: Day 0 (predose), Weeks 6, 8, 16, 32, 40, End of Study (Week 52 for Cohort 1, Week 44 for Cohort 2)/Early WithdrawalPopulation: As Treated Set: all participants who received any amount of DTX101.
The development of neutralizing antibodies to FIX (FIX inhibitors), as determined by a Bethesda assay. A value of \< 0.3 inhibitor units was considered to be no neutralizing antibodies.
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Number of Participants With Neutralizing Antibodies to FIX (FIX Inhibitors)
Day 0 (predose)
|
0 Participants
|
0 Participants
|
|
Number of Participants With Neutralizing Antibodies to FIX (FIX Inhibitors)
Week 6
|
0 Participants
|
0 Participants
|
|
Number of Participants With Neutralizing Antibodies to FIX (FIX Inhibitors)
Week 8
|
0 Participants
|
0 Participants
|
|
Number of Participants With Neutralizing Antibodies to FIX (FIX Inhibitors)
Week 16
|
0 Participants
|
0 Participants
|
|
Number of Participants With Neutralizing Antibodies to FIX (FIX Inhibitors)
Week 32
|
0 Participants
|
0 Participants
|
|
Number of Participants With Neutralizing Antibodies to FIX (FIX Inhibitors)
Week 40
|
0 Participants
|
0 Participants
|
|
Number of Participants With Neutralizing Antibodies to FIX (FIX Inhibitors)
End of Study/Early Withdrawal
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 0 (predose), Weeks 6, 8, 12, 16, 32, 40, 48, End of Study (Week 52 for Cohort 1, Week 44 for Cohort 2)/Early WithdrawalPopulation: Safety Set: all participants who received DTX101 including subjects who received a partial dose or failed infusion.
The development of a cell-mediated immune response to FIX, as determined by enzyme-linked immunospot assay (ELISPOT).
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Number of Participants With Cell-Mediated Immune Response to FIX
Day 0 (predose)
|
0 Participants
|
0 Participants
|
|
Number of Participants With Cell-Mediated Immune Response to FIX
Week 6
|
0 Participants
|
0 Participants
|
|
Number of Participants With Cell-Mediated Immune Response to FIX
Week 8
|
0 Participants
|
0 Participants
|
|
Number of Participants With Cell-Mediated Immune Response to FIX
Week 12
|
0 Participants
|
0 Participants
|
|
Number of Participants With Cell-Mediated Immune Response to FIX
Week 16
|
0 Participants
|
0 Participants
|
|
Number of Participants With Cell-Mediated Immune Response to FIX
Week 32
|
0 Participants
|
0 Participants
|
|
Number of Participants With Cell-Mediated Immune Response to FIX
Week 40
|
0 Participants
|
0 Participants
|
|
Number of Participants With Cell-Mediated Immune Response to FIX
Week 48
|
0 Participants
|
0 Participants
|
|
Number of Participants With Cell-Mediated Immune Response to FIX
End of Study/Early Withdrawal
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 0 predose), Weeks 24, 36, 48, End of Study/Early Withdrawal (up to Week 52)Population: Safety Set: all participants who received DTX101 including participants who received a partial dose or failed infusion.
EQ-5D-5L is a standardized, subject-rated instrument for use as a measure of health outcomes. The EQ 5D-5L includes 2 components: the EQ-5D-5L descriptive system and the EQ visual analogue scale (EQ-VAS). The EQ-5D-5L descriptive system provides a profile of the participant's health state in 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each dimension, the participant is instructed to indicate whether he or she has "no problems" (1), "some problems" (2), or "severe problems" (3).
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Number of Participants Responding to the EuroQoL-5D-5 Level (EQ-5D-5L) Questionnaire
Day 0
|
3 Participants
|
3 Participants
|
|
Number of Participants Responding to the EuroQoL-5D-5 Level (EQ-5D-5L) Questionnaire
Week 24
|
3 Participants
|
3 Participants
|
|
Number of Participants Responding to the EuroQoL-5D-5 Level (EQ-5D-5L) Questionnaire
Week 36
|
3 Participants
|
2 Participants
|
|
Number of Participants Responding to the EuroQoL-5D-5 Level (EQ-5D-5L) Questionnaire
Week 48
|
2 Participants
|
1 Participants
|
|
Number of Participants Responding to the EuroQoL-5D-5 Level (EQ-5D-5L) Questionnaire
End of Study/Early Withdrawal
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 0 predose), Weeks 24, 36, 48, End of Study/Early Withdrawal (up to Week 52)Population: Safety Set: all participants who received DTX101 including participants who received a partial dose or failed infusion.
The Haemophilia-Specific Quality of Life questionnaire asks subjects about their perceptions of their health and treatment. The questionnaire is divided into the following 10 dimensions: physical health, feelings, view of themselves, sports \& leisure, work \& school, dealing with hemophilia, treatment, future, family planning, and partnership \& sexuality. Questions are based on a 5-point Likert-scale (1=never, 2=rarely, 3=sometimes, 4=often, 5=all the time). If the question does not apply to the subject, the "not applicable" response is allowed in 3 of the domains (sport \& leisure, work \& school, family planning). Positively worded items need to be re-coded and domains will be transformed ranging from 0 to 100; higher domain and total scores indicating a higher impairment of health-related quality of life.
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Number of Participants Responding to the Haemophilia-Specific Quality of Life Questionnaire
Day 0
|
3 Participants
|
3 Participants
|
|
Number of Participants Responding to the Haemophilia-Specific Quality of Life Questionnaire
Week 24
|
3 Participants
|
3 Participants
|
|
Number of Participants Responding to the Haemophilia-Specific Quality of Life Questionnaire
Week 36
|
3 Participants
|
2 Participants
|
|
Number of Participants Responding to the Haemophilia-Specific Quality of Life Questionnaire
Week 48
|
2 Participants
|
1 Participants
|
|
Number of Participants Responding to the Haemophilia-Specific Quality of Life Questionnaire
End of Study/Early Withdrawal
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline (Screening), Week 0 through Week 52Population: As Treated Set: all participants who received any amount of DTX101, with an assessment at given time point.
The use of on-demand FIX replacement therapy was recorded by dose (IU/kg) administered and the average weekly use of FIX replacement therapy was calculated. Participants were not required to stop prophylactic treatment with recombinant FIX until after Week 4 and may have been restarted on their prophylactic recombinant FIX treatment after Week 14.
Outcome measures
| Measure |
DTX101, Cohort 1
n=3 Participants
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 Participants
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Average Weekly Use of FIX Replacement Therapy
Week 39 to 40
|
26795.0 IU/kg
Standard Deviation 35645.25
|
2375.0 IU/kg
Standard Deviation 883.88
|
|
Average Weekly Use of FIX Replacement Therapy
Baseline
|
9659.1 IU/kg
Standard Deviation 12502.19
|
5037.2 IU/kg
Standard Deviation 3502.95
|
|
Average Weekly Use of FIX Replacement Therapy
Week 0 to 2
|
12120.0 IU/kg
Standard Deviation 11144.00
|
5000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
|
Average Weekly Use of FIX Replacement Therapy
Week 3 to 4
|
24000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
7000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
|
Average Weekly Use of FIX Replacement Therapy
Week 5 to 6
|
9068.5 IU/kg
Standard Deviation 10611.55
|
3000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
|
Average Weekly Use of FIX Replacement Therapy
Week 7 to 8
|
5120.0 IU/kg
Standard Deviation 1244.51
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 9 to 10
|
32000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 11 to 12
|
8076.7 IU/kg
Standard Deviation 10325.91
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 13 to 14
|
12000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 15 to 16
|
16000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 17 to 18
|
8000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
3375.0 IU/kg
Standard Deviation 883.88
|
|
Average Weekly Use of FIX Replacement Therapy
Week 19 to 20
|
12000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
8000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
|
Average Weekly Use of FIX Replacement Therapy
Week 21 to 22
|
7832.8 IU/kg
Standard Deviation 3057.68
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 23 to 24
|
8782.5 IU/kg
Standard Deviation 10207.09
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 25 to 26
|
8902.5 IU/kg
Standard Deviation 4380.53
|
5445.0 IU/kg
Standard Deviation 3952.73
|
|
Average Weekly Use of FIX Replacement Therapy
Week 27 to 28
|
8120.0 IU/kg
Standard Deviation 5487.15
|
4725.0 IU/kg
Standard Deviation 1025.30
|
|
Average Weekly Use of FIX Replacement Therapy
Week 29 to 30
|
12120.0 IU/kg
Standard Deviation 11144.00
|
2250.0 IU/kg
Standard Deviation 353.55
|
|
Average Weekly Use of FIX Replacement Therapy
Week 31 to 32
|
28000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
2750.0 IU/kg
Standard Deviation 1767.77
|
|
Average Weekly Use of FIX Replacement Therapy
Week 33 to 34
|
12000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
2816.7 IU/kg
Standard Deviation 1877.72
|
|
Average Weekly Use of FIX Replacement Therapy
Week 35 to 36
|
12000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 37 to 38
|
4795.0 IU/kg
Standard Deviation 4532.55
|
3500.0 IU/kg
Standard Deviation 2828.43
|
|
Average Weekly Use of FIX Replacement Therapy
Week 41 to 42
|
24000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
1500.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
|
Average Weekly Use of FIX Replacement Therapy
Week 43 to 44
|
20000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
1750.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
|
Average Weekly Use of FIX Replacement Therapy
Week 45 to 46
|
20000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
5000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
|
Average Weekly Use of FIX Replacement Therapy
Week 47 to 48
|
20000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
7500.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
|
Average Weekly Use of FIX Replacement Therapy
Week 49 to 50
|
24000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
—
|
|
Average Weekly Use of FIX Replacement Therapy
Week 51 to 52
|
20000.0 IU/kg
Standard Deviation NA
Only 1 participant analyzed at this time point.
|
—
|
Adverse Events
DTX101, Cohort 1
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
DTX101, Cohort 2
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
DTX101, Cohort 1
n=3 participants at risk
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 participants at risk
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorders
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
0.00%
0/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
Other adverse events
| Measure |
DTX101, Cohort 1
n=3 participants at risk
a single peripheral IV infusion of 1.6 x 10\^12 GC/kg DTX101
|
DTX101, Cohort 2
n=3 participants at risk
a single peripheral IV infusion of 5.0 x 10\^12 GC/kg DTX101
|
|---|---|---|
|
Investigations
Investigations
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
100.0%
3/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Infections and infestations
Infections and infestations
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
66.7%
2/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications
|
66.7%
2/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorders
|
66.7%
2/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
General disorders
General disorders and administration site conditions
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Nervous system disorders
Nervous system disorders
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
|
0.00%
0/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
66.7%
2/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Eye disorders
Eye disorders
|
0.00%
0/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Psychiatric disorders
Psychiatric disorders
|
0.00%
0/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
|
Vascular disorders
Vascular disorders
|
33.3%
1/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
0.00%
0/3 • From first dose of study drug up to 52 weeks after dosing (Cohort 1) or 44 weeks after dosing (Cohort 2).
TEAEs are presented. A TEAE was defined as any event not present before exposure to study product or any event already present that worsened in severity or increased in frequency after exposure to study product. Due to concerns related to participant re-identification in this study, events are presented by system organ class only.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER