Trial Outcomes & Findings for A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM (NCT NCT04622046)
NCT ID: NCT04622046
Last Updated: 2026-01-09
Results Overview
The 6MWT measures how far a participant can walk in 6 minutes. Change from baseline was calculated as the difference between the distance walked during the 6MWT at 12 months and the distance walked during the 6MWT at baseline. To determine the baseline, at least 2 6MWTs were conducted \> 24 hours to ≤ 3 weeks apart prior to the first dose of ALXN2060. The baseline 6MWT is the average of the total distance walked by participants for the 2 qualifying 6MWTs that met all the protocol-defined criteria. Least squares mean change from baseline data were adjusted for baseline measures and visits.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
25 participants
Primary outcome timeframe
Baseline, Month 12
Results posted on
2026-01-09
Participant Flow
Data presented in this results posting are for analyses up to the Month 30 visit (Intervention Period). The Extension Period of the study is ongoing. Data for the Extension Period will be reported once the study has completed.
Participant milestones
| Measure |
ALXN2060
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Intervention Period
STARTED
|
25
|
|
Intervention Period
Received at Least 1 Dose of Study Drug
|
25
|
|
Intervention Period
COMPLETED
|
22
|
|
Intervention Period
NOT COMPLETED
|
3
|
|
Extension Period
STARTED
|
21
|
|
Extension Period
COMPLETED
|
0
|
|
Extension Period
NOT COMPLETED
|
21
|
Reasons for withdrawal
| Measure |
ALXN2060
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Intervention Period
Adverse Event
|
1
|
|
Intervention Period
Withdrawal by Subject
|
2
|
|
Extension Period
Ongoing in the Extension Period
|
21
|
Baseline Characteristics
A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
Baseline characteristics by cohort
| Measure |
ALXN2060
n=25 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Age, Continuous
|
76.5 years
STANDARD_DEVIATION 6.31 • n=8 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
25 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Asian
|
25 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=8 Participants
|
PRIMARY outcome
Timeframe: Baseline, Month 12Population: Measured in the Full Analysis Set, which included all participants who have received at least 1 dose of ALXN2060. Number of participants analyzed = number of participants evaluable for the outcome measure at the specified timepoint.
The 6MWT measures how far a participant can walk in 6 minutes. Change from baseline was calculated as the difference between the distance walked during the 6MWT at 12 months and the distance walked during the 6MWT at baseline. To determine the baseline, at least 2 6MWTs were conducted \> 24 hours to ≤ 3 weeks apart prior to the first dose of ALXN2060. The baseline 6MWT is the average of the total distance walked by participants for the 2 qualifying 6MWTs that met all the protocol-defined criteria. Least squares mean change from baseline data were adjusted for baseline measures and visits.
Outcome measures
| Measure |
ALXN2060
n=23 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Part A: Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT)
|
-3.86 meters
Standard Error 9.182
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PRIMARY outcome
Timeframe: 30 monthsPopulation: Measured in the Full Analysis Set, which included all participants who have received at least 1 dose of ALXN2060.
CV-related hospitalization was defined as the mean number of CV-related hospitalizations per participant per year over a 30-month period. CV-related hospitalizations were also reported as adverse events and were reviewed and adjudicated by an independent Clinical Events Committee (CEC).
Outcome measures
| Measure |
ALXN2060
n=25 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Parts A and B: Number of Cardiovascular (CV)-Related Hospitalizations Over A 30-month Period
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0.1329 CV-related hospitalizations
Interval 0.0511 to 0.3457
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PRIMARY outcome
Timeframe: 30 monthsPopulation: Measured in the Full Analysis Set, which included all participants who have received at least 1 dose of ALXN2060.
ACM was assessed as time from the date of first initiation of study treatment to the date of death during a 30-month period, and was analyzed using Kaplan-Meier analysis. Data are reported for the number of participants with ACM over the 30-month period.
Outcome measures
| Measure |
ALXN2060
n=25 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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All-cause Mortality (ACM) Over A 30-month Period
|
0 Participants
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SECONDARY outcome
Timeframe: Baseline, Months 6, 9, 18, 24 and 30Population: Measured in the Full Analysis Set, which included all participants who have received at least 1 dose of ALXN2060. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. "Number analyzed" = participants evaluable for this outcome measure at the specified timepoint.
The 6MWT measures how far a participant can walk in 6 minutes. Change from baseline was calculated as the difference between the distance walked during the 6MWT at specified timepoints and the distance walked during the 6MWT at baseline. To determine the baseline, at least 2 6MWTs were conducted \> 24 hours to ≤ 3 weeks apart prior to the first dose of ALXN2060. The baseline 6MWT is the average of the total distance walked by participants for the 2 qualifying 6MWTs that met all the protocol-defined criteria. Least squares mean change from baseline data were adjusted for baseline measures and visits.
Outcome measures
| Measure |
ALXN2060
n=25 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Parts A and B: Change From Baseline In Distance Walked During The 6MWT
Month 6
|
-29.54 meters
Standard Error 9.316
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|
Parts A and B: Change From Baseline In Distance Walked During The 6MWT
Month 9
|
-23.12 meters
Standard Error 11.610
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|
Parts A and B: Change From Baseline In Distance Walked During The 6MWT
Month 18
|
-26.27 meters
Standard Error 12.532
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|
Parts A and B: Change From Baseline In Distance Walked During The 6MWT
Month 24
|
-24.89 meters
Standard Error 8.534
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Parts A and B: Change From Baseline In Distance Walked During The 6MWT
Month 30
|
-36.20 meters
Standard Error 10.798
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SECONDARY outcome
Timeframe: Baseline, Months 6, 9, 12, 18, 24 and 30Population: Measured in the Full Analysis Set, which included all participants who have received at least 1 dose of ALXN2060. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. "Number analyzed" = participants evaluable for this outcome measure at the specified timepoint.
The KCCQ is a 23-item questionnaire developed to measure health status and health-related quality of life in participants with heart failure. Items include heart failure symptoms, impact on physical and social functions, and how their heart failure impacts their quality of life. The overall summary score ranged from 0-100, with higher scores indicating better health status. Data presented are for change from baseline to specified timepoints. Least squares mean change from baseline data were adjusted for baseline measures and visits.
Outcome measures
| Measure |
ALXN2060
n=25 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
Month 6
|
0.94 scores on a scale
Standard Error 2.779
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|
Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
Month 9
|
-1.73 scores on a scale
Standard Error 3.291
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|
Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
Month 12
|
2.71 scores on a scale
Standard Error 2.766
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|
Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
Month 18
|
-5.34 scores on a scale
Standard Error 3.747
|
|
Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
Month 24
|
-0.68 scores on a scale
Standard Error 3.029
|
|
Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
Month 30
|
-6.97 scores on a scale
Standard Error 3.755
|
SECONDARY outcome
Timeframe: Up to Month 30Population: Measured in the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
A treatment-emergent AE was defined as any untoward medical occurrence in a participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention that occurred after first dose. A treatment-emergent SAE was defined as any untoward medical occurrence that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent disability/incapacity, or was a congenital anomaly/birth defect that occurred after first dose. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
ALXN2060
n=25 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Parts A and B: Number of Participants With Treatment-emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Treatment Discontinuation
Treatment-emergent AE
|
25 Participants
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|
Parts A and B: Number of Participants With Treatment-emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Treatment Discontinuation
Treatment-emergent SAE
|
12 Participants
|
|
Parts A and B: Number of Participants With Treatment-emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Treatment Discontinuation
AEs Leading to Treatment Discontinuation
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline, pre-dose on Days 14, 28 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27 and 30Population: Measured in the Full Analysis Set, which included all participants who have received at least 1 dose of ALXN2060. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. "Number analyzed" = participants evaluable for this outcome measure at the specified timepoint.
Least squares mean change from baseline derived from with visits and baseline serum TTR as a covariate. Other covariates were included as needed.
Outcome measures
| Measure |
ALXN2060
n=24 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Day 28, Predose
|
9.13 milligrams (mg)/deciliter (dL)
Standard Error 1.256
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Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 3, Predose
|
10.64 milligrams (mg)/deciliter (dL)
Standard Error 1.056
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|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 6, Predose
|
9.61 milligrams (mg)/deciliter (dL)
Standard Error 0.847
|
|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 9, Predose
|
11.16 milligrams (mg)/deciliter (dL)
Standard Error 0.744
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|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 12, Predose
|
11.80 milligrams (mg)/deciliter (dL)
Standard Error 1.157
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|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 15, Predose
|
10.79 milligrams (mg)/deciliter (dL)
Standard Error 1.347
|
|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 18, Predose
|
10.12 milligrams (mg)/deciliter (dL)
Standard Error 1.253
|
|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 21, Predose
|
9.23 milligrams (mg)/deciliter (dL)
Standard Error 0.704
|
|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 24, Predose
|
11.06 milligrams (mg)/deciliter (dL)
Standard Error 0.848
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Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 27, Predose
|
9.57 milligrams (mg)/deciliter (dL)
Standard Error 1.020
|
|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Month 30, Predose
|
9.36 milligrams (mg)/deciliter (dL)
Standard Error 1.280
|
|
Parts A and B: Change From Baseline In Serum Transthyretin (TTR) Concentration
Day 14, Predose
|
9.73 milligrams (mg)/deciliter (dL)
Standard Error 0.886
|
SECONDARY outcome
Timeframe: Baseline, Pre-dose Days 14, 28 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27 and 30Population: Measured in the Full Analysis Set, which included all participants who have received at least 1 dose of ALXN2060. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. "Number analyzed" = participants evaluable for this outcome measure at the specified timepoint.
TTR stabilization was measured using fluorescent probe exclusion (FPE). Data presented are for change from baseline in FPE percentage stabilization.
Outcome measures
| Measure |
ALXN2060
n=24 Participants
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
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|---|---|
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Parts A and B: Change From Baseline In TTR Stabilization
Day 14, Predose
|
97.15 percentage stabilization
Interval 94.45 to 100.05
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Day 28, Predose
|
97.29 percentage stabilization
Interval 95.16 to 99.66
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 3, Predose
|
99.09 percentage stabilization
Interval 95.24 to 102.64
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 6, Predose
|
98.56 percentage stabilization
Interval 94.2 to 104.44
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 9, Predose
|
96.47 percentage stabilization
Interval 94.07 to 101.73
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 12, Predose
|
100.89 percentage stabilization
Interval 99.95 to 102.51
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 15, Predose
|
97.65 percentage stabilization
Interval 96.3 to 100.03
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 18, Predose
|
96.75 percentage stabilization
Interval 94.4 to 101.0
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 21, Predose
|
101.30 percentage stabilization
Interval 99.8 to 103.0
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 24, Predose
|
102.60 percentage stabilization
Interval 98.2 to 104.8
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 27, Predose
|
102.65 percentage stabilization
Interval 101.0 to 105.6
|
|
Parts A and B: Change From Baseline In TTR Stabilization
Month 30, Predose
|
95.60 percentage stabilization
Interval 91.6 to 103.6
|
Adverse Events
ALXN2060
Serious events: 12 serious events
Other events: 25 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ALXN2060
n=25 participants at risk
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Atrioventricular block complete
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac discomfort
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac failure
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac failure chronic
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Sinus arrest
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Sinus node dysfunction
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Diverticulum intestinal haemorrhagic
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Inguinal hernia
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Large intestine polyp
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Appendicitis perforated
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Bacterial prostatitis
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Pneumonia pneumococcal
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Investigations
Blood creatine phosphokinase MB increased
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Dehydration
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Soft tissue mass
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bladder cancer
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate cancer
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Product Issues
Device dislocation
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
4.0%
1/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
Other adverse events
| Measure |
ALXN2060
n=25 participants at risk
Participants received ALXN2060 twice daily (bid) for 12 months (Part A). Following the Month 12 visit, participants continued to receive ALXN2060 bid for an additional 18 months (up to 30 months from Day 1, \[Part B\]). Following the completion of Month 30 assessments, participants were offered the opportunity to continue into the Extension Period for up to 30 additional months or until ALXN2060 could be provided via an Alexion post study access program (as allowed by local laws and regulations), whichever occurred first. The Extension Period of the study is ongoing.
|
|---|---|
|
Ear and labyrinth disorders
Vertigo
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Eye disorders
Dry eye
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Constipation
|
28.0%
7/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Nausea
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Vomiting
|
12.0%
3/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
General disorders
Malaise
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
General disorders
Pyrexia
|
20.0%
5/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Infections and infestations
COVID-19
|
16.0%
4/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Cystitis
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Nasopharyngitis
|
24.0%
6/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Injury, poisoning and procedural complications
Limb injury
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Investigations
Blood creatinine increased
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Gout
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.0%
3/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
5/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Nervous system disorders
Carpal tunnel syndrome
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Renal and urinary disorders
Haematuria
|
16.0%
4/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Renal and urinary disorders
Renal impairment
|
16.0%
4/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Reproductive system and breast disorders
Benign prostatic hyperplasia
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Skin and subcutaneous tissue disorders
Rash
|
12.0%
3/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Blood and lymphatic system disorders
Anaemia
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Atrial flutter
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac failure
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Palpitations
|
8.0%
2/25 • Up to Month 30
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the Safety Set, which included all participants who have received at least 1 dose of ALXN2060.
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Phone: 855-752-2356
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place