Trial Outcomes & Findings for A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM (NCT NCT04622046)
NCT ID: NCT04622046
Last Updated: 2026-06-10
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
2026-06-10
Participant Flow
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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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|---|---|
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Intervention Period (Part A + Part B)
STARTED
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25
|
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Intervention Period (Part A + Part B)
Received at Least 1 Dose of Study Drug
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25
|
|
Intervention Period (Part A + Part B)
COMPLETED
|
22
|
|
Intervention Period (Part A + Part B)
NOT COMPLETED
|
3
|
|
Extension Period
STARTED
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21
|
|
Extension Period
COMPLETED
|
13
|
|
Extension Period
NOT COMPLETED
|
8
|
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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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|---|---|
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Intervention Period (Part A + Part B)
Adverse Event
|
1
|
|
Intervention Period (Part A + Part B)
Withdrawal by Subject
|
2
|
|
Extension Period
Adverse Event
|
4
|
|
Extension Period
Death
|
1
|
|
Extension Period
Withdrawal by Subject
|
3
|
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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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|---|---|
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Age, Continuous
|
76.5 years
STANDARD_DEVIATION 6.31 • n=9 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=9 Participants
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Sex: Female, Male
Male
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22 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
25 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Asian
|
25 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
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|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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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
|
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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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|---|---|
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Parts A and B: Number of Cardiovascular (CV)-Related Hospitalizations Over A 30-month Period
|
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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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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
|
|
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
|
|
Parts A and B: Change From Baseline In Distance Walked During The 6MWT
Month 18
|
-26.27 meters
Standard Error 12.532
|
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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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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
|
|
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
|
|
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
|
|
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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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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
|
|
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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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|---|---|
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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
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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
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|
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
|
|
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
|
|
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
|
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 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
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|---|---|
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Parts A and B: Change From Baseline In TTR Stabilization
Month 30, Predose
|
95.60 percentage stabilization
Interval 91.6 to 103.6
|
|
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 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
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
|
Adverse Events
ALXN2060
Serious events: 17 serious events
Other events: 25 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
ALXN2060
n=25 participants at risk
Participants received ALXN2060 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
|
|---|---|
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Cardiac disorders
Atrial fibrillation
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Atrioventricular block complete
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac discomfort
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac failure
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac failure chronic
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Sinus arrest
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Sinus node dysfunction
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Diverticulum intestinal haemorrhagic
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Inguinal hernia
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Large intestine polyp
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Appendicitis perforated
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Bacterial prostatitis
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Pneumonia
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Investigations
Blood creatine phosphokinase MB increased
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Dehydration
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Soft tissue mass
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bladder cancer
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate cancer
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Product Issues
Device dislocation
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Bradycardia
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
General disorders
Non-cardiac chest pain
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Pneumonia aspiration
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Pneumonia pneumococcal
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Injury, poisoning and procedural complications
Cervical vertebral fracture
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Injury, poisoning and procedural complications
Extradural haematoma
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Injury, poisoning and procedural complications
Laryngeal injury
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adult T-cell lymphoma/leukaemia
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Nervous system disorders
Cerebral infarction
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Nervous system disorders
Embolic stroke
|
4.0%
1/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
Other adverse events
| Measure |
ALXN2060
n=25 participants at risk
Participants received ALXN2060 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 in Part B, 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) or until the start of commercial drug use if the marketing approval of investigational product is obtained in Japan, whichever occurred first.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Atrial flutter
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac failure
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Palpitations
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Ear and labyrinth disorders
Vertigo
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Eye disorders
Dry eye
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Constipation
|
32.0%
8/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Nausea
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Gastrointestinal disorders
Vomiting
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
General disorders
Malaise
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
General disorders
Pyrexia
|
16.0%
4/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Cystitis
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Nasopharyngitis
|
24.0%
6/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Injury, poisoning and procedural complications
Limb injury
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Investigations
Blood creatinine increased
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Gout
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
5/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Nervous system disorders
Dizziness
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Renal and urinary disorders
Haematuria
|
16.0%
4/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Renal and urinary disorders
Renal impairment
|
16.0%
4/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Reproductive system and breast disorders
Benign prostatic hyperplasia
|
13.6%
3/22 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Skin and subcutaneous tissue disorders
Rash
|
16.0%
4/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Cardiac failure chronic
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Atrial fibrillation
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Cardiac disorders
Atrial tachycardia
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Endocrine disorders
Hypothyroidism
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Eye disorders
Conjunctivitis allergic
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
COVID-19
|
20.0%
5/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Infections and infestations
Pneumonia
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Injury, poisoning and procedural complications
Contusion
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Metabolism and nutrition disorders
Dehydration
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Nervous system disorders
Carpal tunnel syndrome
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Psychiatric disorders
Insomnia
|
12.0%
3/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who received at least 1 dose of ALXN2060.
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
8.0%
2/25 • Up to approximately Month 57
All-cause mortality is reported for all enrolled participants. Serious and other adverse events are reported for the overall study based upon the Long-term Safety and Efficacy analysis set which included all participants who 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