Trial Outcomes & Findings for Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone (NCT NCT04167514)
NCT ID: NCT04167514
Last Updated: 2025-08-29
Results Overview
The overall response is defined as having a CR or PR at Day 28, along with: being alive, free of any next-line GVHD therapy, and free of escalation of prednisone-equivalent steroid dose to 2.5 milligrams per kilogram (mg/kg)/day or more. The percentage of participants with CR or PR is reported here. Wilson score confidence intervals (CIs) are reported here as a measure of dispersion. The CR was defined as a score of 0 for the GVHD staging in all evaluable organs. The PR was defined as an improvement in one or more organs involved with GVHD symptoms without progression in others. Acute GVHD was graded and assessed for response based on Harris (Mount Sinai Acute GVHD International Consortium \[MAGIC\]) criteria (stage 0, 1, 2, 3, 4) for skin, liver, upper gastrointestinal (GI) tract, and lower GI tract. Participants who had an escalation of prednisone-equivalent steroid dose to 2.5 mg/kg/day or higher were classified as non-responders (NR).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
136 participants
Primary outcome timeframe
At Day 28
Results posted on
2025-08-29
Participant Flow
This study was conducted at 26 investigative sites in the United States of America.
A total of 136 participants were randomized in this study.
Participant milestones
| Measure |
Alpha-1 Antitrypsin (AAT)
Participants received AAT twice weekly through Day 28. Responding participants (Complete Response/Partial Response \[CR/PR\]) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
Participants received albumin solution administered intravenously (IV) to match the investigational medicinal product (IMP) twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Overall Study
STARTED
|
65
|
71
|
|
Overall Study
Treated
|
63
|
69
|
|
Overall Study
COMPLETED
|
38
|
41
|
|
Overall Study
NOT COMPLETED
|
27
|
30
|
Reasons for withdrawal
| Measure |
Alpha-1 Antitrypsin (AAT)
Participants received AAT twice weekly through Day 28. Responding participants (Complete Response/Partial Response \[CR/PR\]) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
Participants received albumin solution administered intravenously (IV) to match the investigational medicinal product (IMP) twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Overall Study
Adverse event, not serious
|
0
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
|
Overall Study
Participant Decision
|
2
|
4
|
|
Overall Study
Physician Decision
|
3
|
2
|
|
Overall Study
Death
|
14
|
18
|
|
Overall Study
Enrolled but treatment not administered
|
2
|
0
|
|
Overall Study
Multiple reasons for study discontinuation documented; including death, primary reason not reported
|
3
|
1
|
|
Overall Study
Multiple reasons for study discontinuation documented; excluding death, primary reason not reported
|
2
|
4
|
Baseline Characteristics
Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone
Baseline characteristics by cohort
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
Total
n=136 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
56.6 years
STANDARD_DEVIATION 12.65 • n=5 Participants
|
55.3 years
STANDARD_DEVIATION 13.75 • n=7 Participants
|
55.9 years
STANDARD_DEVIATION 13.20 • n=5 Participants
|
|
Sex: Female, Male
Female
|
20 Participants
n=5 Participants
|
32 Participants
n=7 Participants
|
52 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
45 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
84 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
56 Participants
n=5 Participants
|
66 Participants
n=7 Participants
|
122 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
51 Participants
n=5 Participants
|
61 Participants
n=7 Participants
|
112 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
6 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At Day 28Population: Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
The overall response is defined as having a CR or PR at Day 28, along with: being alive, free of any next-line GVHD therapy, and free of escalation of prednisone-equivalent steroid dose to 2.5 milligrams per kilogram (mg/kg)/day or more. The percentage of participants with CR or PR is reported here. Wilson score confidence intervals (CIs) are reported here as a measure of dispersion. The CR was defined as a score of 0 for the GVHD staging in all evaluable organs. The PR was defined as an improvement in one or more organs involved with GVHD symptoms without progression in others. Acute GVHD was graded and assessed for response based on Harris (Mount Sinai Acute GVHD International Consortium \[MAGIC\]) criteria (stage 0, 1, 2, 3, 4) for skin, liver, upper gastrointestinal (GI) tract, and lower GI tract. Participants who had an escalation of prednisone-equivalent steroid dose to 2.5 mg/kg/day or higher were classified as non-responders (NR).
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment
|
60.0 percentage of participants
Interval 47.86 to 71.03
|
45.1 percentage of participants
Interval 34.05 to 56.6
|
SECONDARY outcome
Timeframe: Up to 12 monthsPopulation: Analysis was performed on the ITT population participants with a response as defined in the measure description of the primary outcome measure. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
The DOR was defined as time from the Day 28 response (CR or PR) until the first of the following events occurs: progression of acute GVHD, death, any next-line GVHD therapy, or escalation of prednisone-equivalent steroids to greater than or equal to (\>=) 2.5 mg/kg/day. Wald CIs are reported here as a measure of dispersion.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=39 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=32 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Duration of Response (DOR)
|
82 days
Interval 22.0 to 176.0
|
154 days
Interval 57.0 to
The upper limit of 95% Confidence interval was not estimable due to low number of events.
|
SECONDARY outcome
Timeframe: At 6 and 12 monthsPopulation: Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
An event of NRM was death without prior evidence of relapse/progression of the primary disease, where relapse/progression was treated as a competing risk. Cumulative incidence of NRM at 6 and 12 months post-randomization is reported here for this outcome measure.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Number of Participants With Non-relapse Mortality (NRM) Event
At 6 months
|
12 Participants
|
14 Participants
|
|
Number of Participants With Non-relapse Mortality (NRM) Event
At 12 months
|
13 Participants
|
14 Participants
|
SECONDARY outcome
Timeframe: At 6 and 12 monthsPopulation: Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
An event for overall survival (OS) was death from any cause, while an event for progression-free survival (PFS) was death from any cause or relapse/progression of the primary disease.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Number of Overall and Progression-free Survival Events
OS: At 6 months
|
16 events
|
18 events
|
|
Number of Overall and Progression-free Survival Events
OS: At 12 months
|
20 events
|
23 events
|
|
Number of Overall and Progression-free Survival Events
PFS: At 6 months
|
25 events
|
28 events
|
|
Number of Overall and Progression-free Survival Events
PFS: At 12 months
|
27 events
|
29 events
|
SECONDARY outcome
Timeframe: At Day 56Population: Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
GVHD-free survival was defined as participants being alive, free of acute or chronic GVHD, and free of any next-line GVHD therapy or escalation of steroids to \>=2.5 mg/kg/day prednisone or equivalent for treatment of GVHD.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Number of Participants With GVHD-free Survival
|
29 Participants
|
25 Participants
|
SECONDARY outcome
Timeframe: At Day 7, 14, 21, 28, 56, and 86Population: Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. Here, the 'number analyzed' for Day 86 includes responses for only those participants who remained on maintenance treatment after Day 28.
The percentage of participants with CR, PR (including subset with very good partial response \[VGPR\]), and treatment failure (TF). The designation of TF consisted of participants with NR, mixed response (MR) or progression. The initiation of additional systemic (next-line) GVHD therapies, escalation of prednisone equivalent steroid dose to \>= 2.5 mg/kg/day, or death from any cause prior to the assessment timepoint was also considered a TF. Simultaneous Goodman CIs are provided for category proportions for each arm at each assessment time.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Percentage of Participants With Response
At Day 7 - CR
|
29.2 percentage of participants
Interval 17.86 to 43.97
|
33.8 percentage of participants
Interval 22.03 to 48.0
|
|
Percentage of Participants With Response
At Day 7 - PR
|
33.8 percentage of participants
Interval 21.62 to 48.69
|
25.4 percentage of participants
Interval 15.16 to 39.22
|
|
Percentage of Participants With Response
At Day 7 - TF
|
36.9 percentage of participants
Interval 24.2 to 51.76
|
40.8 percentage of participants
Interval 28.08 to 54.98
|
|
Percentage of Participants With Response
At Day 14 - CR
|
47.7 percentage of participants
Interval 33.66 to 62.1
|
46.5 percentage of participants
Interval 33.11 to 60.38
|
|
Percentage of Participants With Response
At Day 14 - PR
|
23.1 percentage of participants
Interval 13.07 to 37.45
|
14.1 percentage of participants
Interval 6.89 to 26.65
|
|
Percentage of Participants With Response
At Day 14 - TF
|
29.2 percentage of participants
Interval 17.86 to 43.97
|
39.4 percentage of participants
Interval 26.85 to 53.61
|
|
Percentage of Participants With Response
At Day 21 - CR
|
55.4 percentage of participants
Interval 40.79 to 69.1
|
40.8 percentage of participants
Interval 28.08 to 54.98
|
|
Percentage of Participants With Response
At Day 21 - PR
|
9.2 percentage of participants
Interval 3.66 to 21.41
|
9.9 percentage of participants
Interval 4.18 to 21.54
|
|
Percentage of Participants With Response
At Day 21 - TF
|
35.4 percentage of participants
Interval 22.91 to 50.23
|
49.3 percentage of participants
Interval 35.68 to 63.01
|
|
Percentage of Participants With Response
At Day 28 - CR
|
55.4 percentage of participants
Interval 40.79 to 69.1
|
40.8 percentage of participants
Interval 28.08 to 54.98
|
|
Percentage of Participants With Response
At Day 28 - PR
|
4.6 percentage of participants
Interval 1.28 to 15.31
|
4.2 percentage of participants
Interval 1.17 to 14.12
|
|
Percentage of Participants With Response
At Day 28 - TF
|
40.0 percentage of participants
Interval 26.84 to 54.78
|
54.9 percentage of participants
Interval 40.96 to 68.16
|
|
Percentage of Participants With Response
At Day 56 - CR
|
44.6 percentage of participants
Interval 30.9 to 59.21
|
35.2 percentage of participants
Interval 23.22 to 49.42
|
|
Percentage of Participants With Response
At Day 56 - PR
|
6.2 percentage of participants
Interval 2.0 to 17.41
|
4.2 percentage of participants
Interval 1.17 to 14.12
|
|
Percentage of Participants With Response
At Day 56 - TF
|
49.2 percentage of participants
Interval 35.06 to 63.52
|
60.6 percentage of participants
Interval 46.39 to 73.15
|
|
Percentage of Participants With Response
At Day 86 - CR
|
63.4 percentage of participants
Interval 44.82 to 78.72
|
50.0 percentage of participants
Interval 32.3 to 67.7
|
|
Percentage of Participants With Response
At Day 86 - PR
|
2.4 percentage of participants
Interval 0.32 to 16.22
|
10.0 percentage of participants
Interval 3.27 to 26.76
|
|
Percentage of Participants With Response
At Day 86 - TF
|
34.1 percentage of participants
Interval 19.37 to 52.81
|
40.0 percentage of participants
Interval 23.87 to 58.64
|
SECONDARY outcome
Timeframe: At Day 7, 14, 21, 28, 56, and 86Population: Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. Here, the 'number analyzed' for Day 86 includes responses for only those participants who remained on maintenance treatment after Day 28.
The percentage of participants with CR, PR (including subset with VGPR), and TF allowing for treatment with next-line therapy. The designation of TF consisted of participants with NR, MR, or progression. The escalation of prednisone-equivalent steroid dose to \>= 2.5 mg/kg/day or death from any cause prior to the assessment timepoint was also considered a TF. Simultaneous Goodman CIs are provided for category proportions for each arm at each assessment time.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 7 - PR
|
36.9 percentage of participants
Interval 24.2 to 51.76
|
31.0 percentage of participants
Interval 19.69 to 45.12
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 7 - CR
|
29.2 percentage of participants
Interval 17.86 to 43.97
|
33.8 percentage of participants
Interval 22.03 to 48.0
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 7 - TF
|
33.8 percentage of participants
Interval 21.62 to 48.69
|
35.2 percentage of participants
Interval 23.22 to 49.42
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 14 - CR
|
50.8 percentage of participants
Interval 36.48 to 64.94
|
46.5 percentage of participants
Interval 33.11 to 60.38
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 86 - CR
|
65.9 percentage of participants
Interval 47.19 to 80.63
|
72.5 percentage of participants
Interval 53.62 to 85.74
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 14 - PR
|
29.2 percentage of participants
Interval 17.86 to 43.97
|
26.8 percentage of participants
Interval 16.27 to 40.72
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 14 - TF
|
20.0 percentage of participants
Interval 10.79 to 34.07
|
26.8 percentage of participants
Interval 16.27 to 40.72
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 21 - CR
|
56.9 percentage of participants
Interval 42.26 to 70.47
|
46.5 percentage of participants
Interval 33.11 to 60.38
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 21 - PR
|
15.4 percentage of participants
Interval 7.54 to 28.84
|
18.3 percentage of participants
Interval 9.85 to 31.51
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 21 - TF
|
27.7 percentage of participants
Interval 16.63 to 42.37
|
35.2 percentage of participants
Interval 23.22 to 49.42
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 28 - CR
|
63.1 percentage of participants
Interval 48.24 to 75.8
|
49.3 percentage of participants
Interval 35.68 to 63.01
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 28 - PR
|
9.2 percentage of participants
Interval 3.66 to 21.41
|
14.1 percentage of participants
Interval 6.89 to 26.65
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 28 - TF
|
27.7 percentage of participants
Interval 16.63 to 42.37
|
36.6 percentage of participants
Interval 24.41 to 50.82
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 56 - CR
|
52.3 percentage of participants
Interval 37.9 to 66.34
|
47.9 percentage of participants
Interval 34.39 to 61.7
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 56 - PR
|
13.8 percentage of participants
Interval 6.52 to 27.03
|
4.2 percentage of participants
Interval 1.17 to 14.12
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 56 - TF
|
33.8 percentage of participants
Interval 21.62 to 48.69
|
47.9 percentage of participants
Interval 34.39 to 61.7
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 86 - PR
|
7.3 percentage of participants
Interval 2.04 to 23.07
|
10.0 percentage of participants
Interval 3.27 to 26.76
|
|
Percentage of Participants With Response Allowing for Approved Next-line Therapy
At Day 86 - TF
|
26.8 percentage of participants
Interval 13.9 to 45.45
|
17.5 percentage of participants
Interval 7.52 to 35.63
|
SECONDARY outcome
Timeframe: Up to 90 days (including 30 days after last dose of study drug)Population: Analysis was performed on the safety population. The safety population included all randomized participants who received at least one dose of study treatment. Participants were classified according to the treatment received.
The cumulative incidence of Grade 2 to 3 systemic infections. Grade 2 to 3 systemic infections were defined according to the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Manual of Procedures.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=63 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=69 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Incidence of Grade 2 to 3 Systemic Infections
|
20 events
|
22 events
|
SECONDARY outcome
Timeframe: Up to 30 days after the last dose of study drugPopulation: Analysis was performed on the safety population. The safety population included all randomized participants who received at least one dose of study treatment. Participants were classified according to the treatment received.
The incidence of Grade 3 to 5 TEAEs (per Common Terminology Criteria for Adverse Events \[CTCAE\] Version 5.0). Wilson score CIs are reported here as a measure of dispersion.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=63 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=69 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Percentage of Participants With Grade 3 to 5 Treatment-emergent Adverse Events (TEAEs)
|
46.0 percentage of participants
Interval 34.31 to 58.21
|
65.2 percentage of participants
Interval 53.45 to 75.38
|
SECONDARY outcome
Timeframe: At 6 and 12 monthsPopulation: Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
Chronic GVHD was defined per National Institutes of Health (NIH) Consensus Criteria. Diagnosis of chronic GVHD of any severity (mild, moderate, or severe) was considered an event for this outcome measure, where death before cGVHD was treated as a competing risk.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Cumulative Incidence of Chronic GVHD
At 6 months
|
13 Participants
|
9 Participants
|
|
Cumulative Incidence of Chronic GVHD
At 12 months
|
20 Participants
|
14 Participants
|
SECONDARY outcome
Timeframe: At 6 months and 12 monthsPopulation: Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
The cumulative incidence of relapse/progression of the primary disease, with death prior to relapse/progression treated as a competing risk. Death without prior relapse/progression was treated as a competing risk for relapse/progression.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=65 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=71 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Cumulative Incidence of Disease Relapse/ Progression of Primary Disease
At 6 months
|
14 Participants
|
14 Participants
|
|
Cumulative Incidence of Disease Relapse/ Progression of Primary Disease
At 12 months
|
15 Participants
|
15 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 12 monthsPopulation: Analysis was performed on the ITT population participants with a response as defined in the measure description of the primary outcome measure. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered.
The DOR was defined as time from the Day 28 response (CR or PR) until the first of the following events occurs: death, any next-line GVHD therapy, or escalation of prednisone-equivalent steroids to \>= 2.5 mg/kg/day. Wald CIs are reported here as a measure of dispersion.
Outcome measures
| Measure |
Alpha-1 Antitrypsin (AAT)
n=39 Participants
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=32 Participants
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
DOR - Supplementary Analysis
|
NA days
Interval 85.0 to
The median and upper limit of 95% CI were not estimable due to low number of events.
|
NA days
Interval 109.0 to
The median and upper limit of 95% CI were not estimable due to low number of events.
|
Adverse Events
Alpha-1 Antitrypsin (AAT)
Serious events: 11 serious events
Other events: 37 other events
Deaths: 21 deaths
Placebo
Serious events: 23 serious events
Other events: 42 other events
Deaths: 23 deaths
Serious adverse events
| Measure |
Alpha-1 Antitrypsin (AAT)
n=63 participants at risk
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=69 participants at risk
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
5.8%
4/69 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Injury, poisoning and procedural complications
Femoral neck fracture
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Injury, poisoning and procedural complications
Vascular access complication
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Intracranial mass
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Syncope
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Toxic encephalopathy
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Depressed level of consciousness
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Metabolic encephalopathy
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Dysphagia
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
2.9%
2/69 • Number of events 2 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Small intestinal haemorrhage
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Diabetic ketoacidosis
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Blood and lymphatic system disorders
Thrombotic microangiopathy
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Blood and lymphatic system disorders
Autoimmune haemolytic anaemia
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Blood and lymphatic system disorders
Haemolytic uraemic syndrome
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Blood and lymphatic system disorders
Methaemoglobinaemia
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Cardiac disorders
Atrial tachycardia
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Cardiac disorders
Myocardial infarction
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Cardiac disorders
Myocardial ischaemia
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 2 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Cardiac disorders
Sinus bradycardia
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
General disorders
Oedema peripheral
|
3.2%
2/63 • Number of events 2 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
General disorders
Asthenia
|
1.6%
1/63 • Number of events 2 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
General disorders
Multiple organ dysfunction syndrome
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Vascular disorders
Hypotension
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Psychiatric disorders
Anxiety
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
0.00%
0/69 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Vascular disorders
Embolism
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Vascular disorders
Orthostatic hypotension
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Hepatobiliary disorders
Cholecystitis acute
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Hepatobiliary disorders
Hepatic failure
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Psychiatric disorders
Hallucination
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
2.9%
2/69 • Number of events 2 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Investigations
Transaminases increased
|
0.00%
0/63 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
Other adverse events
| Measure |
Alpha-1 Antitrypsin (AAT)
n=63 participants at risk
Participants received AAT twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once weekly dose of AAT through Day 56.
|
Placebo
n=69 participants at risk
Participants received albumin solution administered IV to match the IMP twice weekly through Day 28. Responding participants (CR/PR) continued to receive a once-weekly dose of matching placebo through Day 56.
|
|---|---|---|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
20.6%
13/63 • Number of events 15 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
15.9%
11/69 • Number of events 15 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
15.9%
10/63 • Number of events 15 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
14.5%
10/69 • Number of events 23 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
11.1%
7/63 • Number of events 16 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
17.4%
12/69 • Number of events 26 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
9.5%
6/63 • Number of events 11 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
13.0%
9/69 • Number of events 13 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
14.3%
9/63 • Number of events 27 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
8.7%
6/69 • Number of events 12 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
9.5%
6/63 • Number of events 12 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
13.0%
9/69 • Number of events 16 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
12.7%
8/63 • Number of events 15 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
7.2%
5/69 • Number of events 10 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
9.5%
6/63 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
7.2%
5/69 • Number of events 8 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
4.8%
3/63 • Number of events 3 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
8.7%
6/69 • Number of events 6 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
4.8%
3/63 • Number of events 3 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
5.8%
4/69 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Hypermagnesaemia
|
3.2%
2/63 • Number of events 2 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
5.8%
4/69 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Investigations
Alanine aminotransferase increased
|
11.1%
7/63 • Number of events 8 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
15.9%
11/69 • Number of events 17 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Investigations
Aspartate aminotransferase increased
|
7.9%
5/63 • Number of events 10 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
11.6%
8/69 • Number of events 12 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Investigations
Blood alkaline phosphatase increased
|
6.3%
4/63 • Number of events 5 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
11.6%
8/69 • Number of events 12 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Investigations
Blood bilirubin increased
|
6.3%
4/63 • Number of events 6 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
8.7%
6/69 • Number of events 8 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Investigations
Blood creatinine increased
|
6.3%
4/63 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
8.7%
6/69 • Number of events 8 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Investigations
Blood lactate dehydrogenase increased
|
7.9%
5/63 • Number of events 6 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
7.2%
5/69 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Abdominal pain
|
14.3%
9/63 • Number of events 10 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
8.7%
6/69 • Number of events 6 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Dry mouth
|
11.1%
7/63 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
4.8%
3/63 • Number of events 3 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
5.8%
4/69 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Constipation
|
6.3%
4/63 • Number of events 6 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
General disorders
Oedema peripheral
|
19.0%
12/63 • Number of events 13 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
14.5%
10/69 • Number of events 12 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
General disorders
Fatigue
|
7.9%
5/63 • Number of events 5 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
10.1%
7/69 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Vascular disorders
Hypertension
|
14.3%
9/63 • Number of events 12 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
14.5%
10/69 • Number of events 16 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Vascular disorders
Hypotension
|
9.5%
6/63 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
4.3%
3/69 • Number of events 3 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
9.5%
6/63 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
11.6%
8/69 • Number of events 8 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.3%
4/63 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
5.8%
4/69 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
1.6%
1/63 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
5.8%
4/69 • Number of events 5 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
7.9%
5/63 • Number of events 5 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
11.6%
8/69 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.9%
5/63 • Number of events 5 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Dizziness
|
1.6%
1/63 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
8.7%
6/69 • Number of events 6 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Tremor
|
6.3%
4/63 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
2.9%
2/69 • Number of events 2 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Psychiatric disorders
Insomnia
|
6.3%
4/63 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
8.7%
6/69 • Number of events 6 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Injury, poisoning and procedural complications
Fall
|
3.2%
2/63 • Number of events 2 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
8.7%
6/69 • Number of events 6 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Blood and lymphatic system disorders
Anaemia
|
7.9%
5/63 • Number of events 20 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
2.9%
2/69 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Cardiac disorders
Sinus tachycardia
|
6.3%
4/63 • Number of events 9 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
4.3%
3/69 • Number of events 3 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
|
Renal and urinary disorders
Dysuria
|
6.3%
4/63 • Number of events 4 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
1.4%
1/69 • Number of events 1 • From randomization to 12 months (end of study [EOS] follow-up): AEs and Serious AEs (SAEs) reported from randomization until 30 days post last dose. Beginning at 31 days from the last dose of the study drug through the EOS follow-up, only SAEs related to the study drug and unanticipated SAEs (regardless of relationship) required reporting. All-cause mortality includes any deaths, regardless of the timing, including those reported beyond the 12 months, up to 16 months for the last reported death.
All-cause mortality data are presented for the ITT population, which consisted of all randomized participants, classified according to their randomized treatment assignments. Analysis of serious AEs (SAEs) and non-serious AEs was performed on the safety population, which included all randomized participants who received at least one dose of study treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place