Trial Outcomes & Findings for A Study of BAX 888 in Male Adults With Severe Hemophilia A (NCT NCT03370172)
NCT ID: NCT03370172
Last Updated: 2025-09-11
Results Overview
An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A Serious adverse event (SAE) is an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. AEs include both serious and non-serious adverse events including development of FVIII inhibitory antibodies, clinically significant changes in standard laboratory parameters, physical exam, and vital signs.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
4 participants
Primary outcome timeframe
From first dose up to end of the study (approximately 6 years)
Results posted on
2025-09-11
Participant Flow
A total of 4 participants took part in the study globally from 27 February 2018 to 09 July 2024.
Participants with severe Hemophilia A participated in the study to receive BAX 888.
Participant milestones
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
|
Overall Study
COMPLETED
|
2
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
A Study of BAX 888 in Male Adults With Severe Hemophilia A
Baseline characteristics by cohort
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
29.5 years
STANDARD_DEVIATION 13.44 • n=99 Participants
|
27.5 years
STANDARD_DEVIATION 3.54 • n=107 Participants
|
28.5 years
STANDARD_DEVIATION 8.1 • n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: From first dose up to end of the study (approximately 6 years)Population: The Safety Set consisted of all participants who received any amount of investigational product.
An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A Serious adverse event (SAE) is an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. AEs include both serious and non-serious adverse events including development of FVIII inhibitory antibodies, clinically significant changes in standard laboratory parameters, physical exam, and vital signs.
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Number of Participants With BAX 888-Related Adverse Events (AEs)
|
2 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline, up to Month 60Population: The Safety Set consisted of all participants who received any amount of investigational product. Overall number analyzed is the number of participants with data available for analysis for this outcome measure.
Change from baseline in circulating plasma FVIII activity level, based on one-stage clotting assay was assessed.
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=1 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Change From Baseline in Circulating Plasma FVIII Activity Level
|
11.40 International Units per deciliter(IU/dL)
Standard Deviation 1.131
|
248.30 International Units per deciliter(IU/dL)
Standard Deviation NA
Standard Deviation (SD) is not estimable when there is only a single participant.
|
SECONDARY outcome
Timeframe: Baseline, up to Month 60Population: The Safety Set consisted of all participants who received any amount of investigational product.
Change from baseline in circulating plasma FVIII antigen (protein) levels were to be assessed and number of participants with clinically significant change as determined by the principal investigator (PI) were reported.
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Number of Participants With Clinically Significant Change From Baseline in Circulating Plasma FVIII Antigen Level
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to approximately 6 years 4 monthsPopulation: The Safety Set consisted of all participants who received any amount of investigational product.
ABR in comparison to before gene transfer will be assessed. A bleed is defined as subjective or objective evidence of bleeding which may or may not require treatment with FVIII. ABR was calculated as (number of bleeding episodes/observed treatment period in days)\*365.25.
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Annualized Bleed Rate (ABR)
|
1.0 bleeds per year
Standard Deviation 1.41
|
0.5 bleeds per year
Standard Deviation 0.71
|
SECONDARY outcome
Timeframe: Up to approximately 6 years 4 monthsPopulation: The Safety Set consisted of all participants who received any amount of investigational product.
The reduction in consumption of exogenous FVIII was assessed by comparing the amount of exogenous FVIII taken at earliest time point available (prior to BAX 888 infusion) with the amount taken at the last post-infusion timepoint available, during the study. Percentage of participants with reduction in consumption of exogenous FVIII are reported.
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Percentage of Participants With a Reduction in Consumption of Exogenous FVIII
|
0.0 percentage of participants
|
0.0 percentage of participants
|
SECONDARY outcome
Timeframe: Up to approximately 6 years 4 monthsPopulation: The Safety Set consisted of all participants who received any amount of investigational product.
Participants were assessed to check if they developed inhibitory antibodies to FVIII.
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Number of Participants Who Developed Inhibitory Antibodies to FVIII
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to approximately 6 years 4 monthsPopulation: The Safety Set consisted of all participants who received any amount of investigational product.
Participants were assessed to check if they developed total binding antibodies to FVIII (Immunoglobulin G \[IgG\], Immunoglobulin M \[IgM\]).
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Number of Participants Who Developed Total Binding Antibodies to FVIII
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to approximately 6 years 4 monthsPopulation: The Safety Set consisted of all participants who received any amount of investigational product. Number analyzed is the number of participants with data available for analysis for the specified categories.
The humoral (antibody-mediated) and cell-mediated immune response to adeno-associated virus (AAV8) (the vector) and FVIII proteins, was assessed. Humoral Immune Response: It is indicated by presence of specific antibodies. The anti-AAV8 binding antibodies, IgG or IgM were measured by the enzyme-linked immunosorbent assay (ELISA) method. Neutralizing antibodies were measured by a cell-based luminescent assay. Cell-mediated Immune response: The AAV8 and FVIII specific cell mediated immunity was assessed using validated interferon-γ (IFN-γ) enzyme-linked immunosorbent spot (ELISpot) assays. This assay tests the human T-cell recall response to the AAV8 and FVIII proteins. These proteins were called antigens for these tests (AAV8 peptide pools 1, 2, 3 and two pooled test antigens (1 and 2) for FVIII). Number of participants who had humoral and/or cell mediated immune response to AAV8 and FVIII proteins, are reported by humoral and cell mediated immune response categories.
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII Proteins
Humoral: Binding Ab to AAV8 IgG Titer
|
2 Participants
|
1 Participants
|
|
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII Proteins
Humoral: Neutralising Ab to AAV8 Titer
|
2 Participants
|
1 Participants
|
|
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII Proteins
Cell-Mediated: AAV8 Peptide Pool 1 Mean
|
0 Participants
|
1 Participants
|
|
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII Proteins
Cell-Mediated: AAV8 Peptide Pool 2 Mean
|
1 Participants
|
1 Participants
|
|
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII Proteins
Cell-Mediated: AAV8 Peptide Pool 3 Mean
|
1 Participants
|
1 Participants
|
|
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII Proteins
Cell-Mediated: FVIII Peptide Pool 1 Mean
|
0 Participants
|
0 Participants
|
|
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII Proteins
Cell-Mediated: FVIII Peptide Pool 2 Mean
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Blood: Day 1, weekly at Clinic Visits between Weeks 1-15, and at Months 4 and 5; Saliva, Semen, and Stool: Day 1 and Week 1; Urine: Day 1 and Weeks 1,2,3Population: The Safety Set consisted of all participants who received any amount of investigational product. The data was collected for each category until 2 consecutive measurements were negative. Number analyzed is the number of participants with data available for analysis at specified time points.
Surveillance of AAV8 genome shedding in blood, saliva, semen, stool and urine until two consecutive negative results were assessed.
Outcome measures
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 Participants
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 Participants
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 13
|
654.0 Genome copies per 100 ng of sample
Standard Deviation 22.63
|
3044.5 Genome copies per 100 ng of sample
Standard Deviation 1191.47
|
|
Surveillance of AAV8 Genome Shedding
Blood: Day 1
|
—
|
3684434.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 1
|
3634.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
18608.0 Genome copies per 100 ng of sample
Standard Deviation 664.68
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 2
|
2076.0 Genome copies per 100 ng of sample
Standard Deviation 295.57
|
10670.0 Genome copies per 100 ng of sample
Standard Deviation 3900.40
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 3
|
2069.0 Genome copies per 100 ng of sample
Standard Deviation 741.05
|
9845.0 Genome copies per 100 ng of sample
Standard Deviation 6488.41
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 4
|
1098.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
9033.5 Genome copies per 100 ng of sample
Standard Deviation 4978.74
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 5
|
1483.0 Genome copies per 100 ng of sample
Standard Deviation 1097.43
|
7884.0 Genome copies per 100 ng of sample
Standard Deviation 288.50
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 6
|
904.0 Genome copies per 100 ng of sample
Standard Deviation 335.17
|
5372.5 Genome copies per 100 ng of sample
Standard Deviation 441.94
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 7
|
1004.0 Genome copies per 100 ng of sample
Standard Deviation 656.20
|
6808.0 Genome copies per 100 ng of sample
Standard Deviation 1121.47
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 8
|
828.5 Genome copies per 100 ng of sample
Standard Deviation 267.99
|
7384.0 Genome copies per 100 ng of sample
Standard Deviation 1011.16
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 9
|
714.5 Genome copies per 100 ng of sample
Standard Deviation 222.74
|
6384.5 Genome copies per 100 ng of sample
Standard Deviation 195.87
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 10
|
773.0 Genome copies per 100 ng of sample
Standard Deviation 9.90
|
6144.5 Genome copies per 100 ng of sample
Standard Deviation 939.74
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 11
|
755.0 Genome copies per 100 ng of sample
Standard Deviation 241.83
|
3935.0 Genome copies per 100 ng of sample
Standard Deviation 1547.15
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 12
|
728.0 Genome copies per 100 ng of sample
Standard Deviation 130.11
|
3893.5 Genome copies per 100 ng of sample
Standard Deviation 276.48
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 14
|
735.5 Genome copies per 100 ng of sample
Standard Deviation 82.73
|
2856.0 Genome copies per 100 ng of sample
Standard Deviation 1473.61
|
|
Surveillance of AAV8 Genome Shedding
Blood: Week 15
|
742.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
2812.0 Genome copies per 100 ng of sample
Standard Deviation 2083.14
|
|
Surveillance of AAV8 Genome Shedding
Blood: Month 4
|
422.0 Genome copies per 100 ng of sample
Standard Deviation 192.33
|
3066.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
|
Surveillance of AAV8 Genome Shedding
Blood: Month 5
|
—
|
210.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
|
Surveillance of AAV8 Genome Shedding
Saliva: Day 1
|
935.0 Genome copies per 100 ng of sample
Standard Deviation 268.70
|
2076.5 Genome copies per 100 ng of sample
Standard Deviation 473.05
|
|
Surveillance of AAV8 Genome Shedding
Saliva: Week 1
|
—
|
352.5 Genome copies per 100 ng of sample
Standard Deviation 12.02
|
|
Surveillance of AAV8 Genome Shedding
Semen: Day 1
|
55.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
230.5 Genome copies per 100 ng of sample
Standard Deviation 188.80
|
|
Surveillance of AAV8 Genome Shedding
Semen: Week 1
|
—
|
194.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
|
Surveillance of AAV8 Genome Shedding
Stool: Day 1
|
—
|
5985.0 Genome copies per 100 ng of sample
Standard Deviation NA
SD was not estimable for a single participant.
|
|
Surveillance of AAV8 Genome Shedding
Stool: Week 1
|
—
|
7164.0 Genome copies per 100 ng of sample
Standard Deviation 7993.14
|
|
Surveillance of AAV8 Genome Shedding
Urine: Day 1
|
NA Genome copies per 100 ng of sample
Standard Deviation NA
Mean and SD were not estimable due to genome concentrations being Below Limit of Detection.
|
NA Genome copies per 100 ng of sample
Standard Deviation NA
Mean and SD were not estimable due to genome concentrations being Below Limit of Detection.
|
|
Surveillance of AAV8 Genome Shedding
Urine: Week 1
|
NA Genome copies per 100 ng of sample
Standard Deviation NA
Mean and SD were not estimable due to genome concentrations being Below Limit of Detection.
|
NA Genome copies per 100 ng of sample
Standard Deviation NA
Mean and SD were not estimable due to genome concentrations being Below Limit of Detection.
|
|
Surveillance of AAV8 Genome Shedding
Urine: Week 2
|
NA Genome copies per 100 ng of sample
Standard Deviation NA
Mean and SD were not estimable due to genome concentrations being Below Limit of Detection.
|
NA Genome copies per 100 ng of sample
Standard Deviation NA
Mean and SD were not estimable due to genome concentrations being Below Limit of Detection.
|
|
Surveillance of AAV8 Genome Shedding
Urine: Week 3
|
NA Genome copies per 100 ng of sample
Standard Deviation NA
Mean and SD were not estimable due to genome concentrations being Below Limit of Detection.
|
NA Genome copies per 100 ng of sample
Standard Deviation NA
Mean and SD were not estimable due to genome concentrations being Below Limit of Detection.
|
Adverse Events
Cohort 1: BAX 888 2.0*10^12 cp/kg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 2: BAX 888 6.0*10^12 cp/kg
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 participants at risk
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 participants at risk
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
Other adverse events
| Measure |
Cohort 1: BAX 888 2.0*10^12 cp/kg
n=2 participants at risk
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
|
Cohort 2: BAX 888 6.0*10^12 cp/kg
n=2 participants at risk
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
General disorders
Asthenia
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Infections and infestations
COVID-19
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Skin and subcutaneous tissue disorders
Dermatosis
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Eye disorders
Dry eye
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Nervous system disorders
Headache
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Metabolism and nutrition disorders
Hyperphagia
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Vascular disorders
Hypertension
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Hepatobiliary disorders
Hypertransaminasaemia
|
100.0%
2/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
100.0%
2/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Metabolism and nutrition disorders
Increased appetite
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
General disorders
Influenza like illness
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Psychiatric disorders
Initial insomnia
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Psychiatric disorders
Irritability
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Nervous system disorders
Migraine
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal discomfort
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Infections and infestations
Nasopharyngitis
|
100.0%
2/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Gastrointestinal disorders
Nausea
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Nervous system disorders
Paraesthesia
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Investigations
SARS-CoV-2 test positive
|
100.0%
2/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Nervous system disorders
Somnolence
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Musculoskeletal and connective tissue disorders
Synovitis
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Musculoskeletal and connective tissue disorders
Tendon disorder
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Gastrointestinal disorders
Tongue geographic
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Infections and infestations
Urinary tract infection
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
|
Investigations
Weight increased
|
0.00%
0/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
50.0%
1/2 • All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years)
The Safety Set consisted of all participants who received any amount of investigational product.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place