Trial Outcomes & Findings for Study of Ravulizumab in Pediatric Participants With HSCT-TMA (NCT NCT04557735)
NCT ID: NCT04557735
Last Updated: 2026-01-12
Results Overview
The criteria for TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal \[ULN\]) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, with no criteria failures or more than 1 missed scheduled visit in between. Additionally, all intervals in which the criteria were met must overlap for at least 1 day.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
41 participants
Primary outcome timeframe
Up to Week 26
Results posted on
2026-01-12
Participant Flow
Participant milestones
| Measure |
Ravulizumab
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Treatment Period (26 Weeks)
STARTED
|
41
|
|
Treatment Period (26 Weeks)
Received at Least 1 Dose of Study Drug
|
41
|
|
Treatment Period (26 Weeks)
COMPLETED
|
29
|
|
Treatment Period (26 Weeks)
NOT COMPLETED
|
12
|
|
Follow-Up Period (26 Weeks)
STARTED
|
29
|
|
Follow-Up Period (26 Weeks)
COMPLETED
|
24
|
|
Follow-Up Period (26 Weeks)
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Ravulizumab
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Treatment Period (26 Weeks)
Withdrawal by Subject
|
1
|
|
Treatment Period (26 Weeks)
Physician Decision
|
4
|
|
Treatment Period (26 Weeks)
Death
|
5
|
|
Treatment Period (26 Weeks)
Adverse Event
|
2
|
|
Follow-Up Period (26 Weeks)
Physician Decision
|
1
|
|
Follow-Up Period (26 Weeks)
Death
|
4
|
Baseline Characteristics
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
Baseline characteristics by cohort
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Age, Continuous
|
7.6 years
STANDARD_DEVIATION 5.57 • n=210 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=210 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=210 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=210 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
36 Participants
n=210 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=210 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=210 Participants
|
|
Race (NIH/OMB)
Asian
|
11 Participants
n=210 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=210 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=210 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=210 Participants
|
|
Race (NIH/OMB)
More than one race
|
6 Participants
n=210 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=210 Participants
|
PRIMARY outcome
Timeframe: Up to Week 26Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of shiga toxin-related hemolytic uremic syndrome (ST-HUS) and a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) laboratory results and are subsequently found to be ineligible after enrollment.
The criteria for TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal \[ULN\]) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, with no criteria failures or more than 1 missed scheduled visit in between. Additionally, all intervals in which the criteria were met must overlap for at least 1 day.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Participants With Thrombotic Microangiopathy (TMA) Response
|
7 Participants
|
SECONDARY outcome
Timeframe: Day 1 through Week 26Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment.
Time to TMA response was defined as the time from first infusion to the first time point at which all criteria for TMA response was met. Participants were assigned as responders at the time of their TMA response and were censored at the earlier of last assessment with all 3 TMA response components available (including measurements collected after treatment discontinuation), or death if they did not respond by then. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Time to TMA Response During the 26-Week Treatment Period
|
NA days
Interval 154.0 to
The median and the upper limit of confidence interval could not be reached because fewer than half of the participants experienced an event.
|
SECONDARY outcome
Timeframe: Up to Week 26Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment.
Hematologic response required the following: (1) Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days and (2) Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Participants With Hematologic Response
|
10 Participants
|
SECONDARY outcome
Timeframe: Day 1 through Week 26Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.
Time to Hematologic response was defined as the time from first infusion to the first time point at which all criteria for hematologic response was met. Participants were assigned as responders at the time of their response and were censored at their discontinuation time or at the end of available follow-up if they did not respond by then. Hematologic response required the following: (1) Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days and (2) Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Time to Hematologic Response During the 26-Week Treatment Period
|
NA days
Interval 154.0 to
NA signifies that the median and the upper limit of confidence interval could not be reached because fewer than half of the participants experienced an event.
|
SECONDARY outcome
Timeframe: Up to Week 26Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment.
Hemoglobin response was defined as the ability to maintain hemoglobin ≥ 10 g/dL without RBC transfusion support. The criterion must have been met at 2 separate assessments obtained at least 24 hours apart, and any measurement in between, and without RBC transfusion support during the prior 7 days.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Participants With Hemoglobin Response
|
17 Participants
|
SECONDARY outcome
Timeframe: Up to Week 26Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment.
Normalization of platelet count was defined as baseline platelet count ≤ 50000 mm\^3 or \> 50000 mm\^3, absolute platelet count \> 50000 mm\^3 or \>=50% increase in platelet count without transfusion support during the prior 7 days.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Participants With Platelet Response
|
24 Participants
|
SECONDARY outcome
Timeframe: Up to Week 26Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment.
Partial response was defined as a participant meeting at least 1, but not all, criteria for TMA response. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH, (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Participants With Partial TMA Response
|
22 Participants
|
SECONDARY outcome
Timeframe: Up to Week 26Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.
Loss of response occurred when a participant who had previously achieved a TMA response failed to meet criteria for one or more components of TMA response at a subsequent visit in treatment period. At least one parameter must fail to meet response criteria at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline.
Outcome measures
| Measure |
Ravulizumab
n=7 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Participants With Loss of TMA Response
|
2 Participants
|
SECONDARY outcome
Timeframe: Day 1 through Week 52Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.
This analysis includes data for each participant after TMA response through final follow-up. Participants with TMA response who do not experience these events are censored at their end of study date. TMA response required following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. The estimate is calculated based on Kaplan-Meier method.
Outcome measures
| Measure |
Ravulizumab
n=7 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Duration of TMA Response Through Week 52
|
NA days
Interval 22.0 to
NA signifies that the median and the upper limit of confidence interval could not be reached because fewer than half of the participants experienced an event.
|
SECONDARY outcome
Timeframe: Up to Week 52Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.
For participants that meet criteria for TMA response during 26-week Treatment Period, TMA relapse is defined as evidence of worsening hematologic and renal dysfunction due to TMA during post-treatment Follow-up Period that requires treatment intervention, as determined by Investigator. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm\^3 or \>=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline.
Outcome measures
| Measure |
Ravulizumab
n=7 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Participants With TMA Relapse
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 1 through Week 52Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment.
The Kaplan-Meier method was used to measure overall survival estimate. Overall survival was calculated from date of treatment start to date of a documented death event (death due to any cause) or date of censoring. Participants who survived were censored at the earliest of an additional hematopoietic stem cell transplant (HSCT), Week 52, or their last known date alive.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Overall Survival
|
0.734 proportion of participants
Interval 0.56 to 0.847
|
SECONDARY outcome
Timeframe: Day 1 through Week 52Population: Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment.
Cumulative incidence was estimated using a competing risk model. Non-relapse mortality was defined as a participant's death due to any cause during the study, with the exception of death due to underlying disease progression or relapse.
Outcome measures
| Measure |
Ravulizumab
n=41 Participants
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|
|
Non-relapse Mortality During the 52-Week Treatment Period
|
0.184 proportion of participants
Interval 0.079 to 0.322
|
Adverse Events
Treatment Period: Ravulizumab
Serious events: 30 serious events
Other events: 41 other events
Deaths: 6 deaths
Follow-up Period: Ravulizumab
Serious events: 15 serious events
Other events: 23 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Treatment Period: Ravulizumab
n=41 participants at risk
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight.
|
Follow-up Period: Ravulizumab
n=29 participants at risk
After the completion of the treatment period, participants were followed for 26 Weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
7.3%
3/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Blood and lymphatic system disorders
Pancytopenia
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Blood and lymphatic system disorders
Thrombotic microangiopathy
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Cardiac disorders
Pericardial effusion
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Cardiac disorders
Arrhythmia
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Cardiac disorders
Cardiac arrest
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Cardiac disorders
Cardiac failure
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Cardiac disorders
Cardiac tamponade
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Congenital, familial and genetic disorders
Phimosis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Eye disorders
Mydriasis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Eye disorders
Uveitis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Abdominal pain
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Colitis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Diarrhoea
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Gastric haemorrhage
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Pneumatosis intestinalis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Vomiting
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
General disorders
Multiple organ dysfunction syndrome
|
7.3%
3/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
General disorders
Pyrexia
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Hepatobiliary disorders
Venoocclusive liver disease
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Immune system disorders
Graft versus host disease
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Immune system disorders
Graft versus host disease in gastrointestinal tract
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
COVID-19
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Gastroenteritis clostridial
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Septic shock
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Acinetobacter sepsis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Adenovirus infection
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Appendicitis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Bacteraemia
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Device related infection
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Gastroenteritis Escherichia coli
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Gastroenteritis rotavirus
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Gastroenteritis salmonella
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Herpes zoster
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Infection
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Klebsiella infection
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Mucormycosis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Norovirus infection
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Pneumonia
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Pseudomonal bacteraemia
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Sepsis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Stenotrophomonas infection
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Vaginal abscess
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Dehydration
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Musculoskeletal and connective tissue disorders
Soft tissue necrosis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Seizure
|
7.3%
3/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Depressed level of consciousness
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Dizziness
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Headache
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Loss of consciousness
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Neurological symptom
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Partial seizures
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Syncope
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Renal and urinary disorders
Acute kidney injury
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Renal and urinary disorders
Renal failure
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
4.9%
2/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Surgical and medical procedures
Stem cell transplant
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Vascular disorders
Microangiopathy
|
2.4%
1/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Haematemesis
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Haemoperitoneum
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Device related bacteraemia
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Influenza
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Bacterial sepsis
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Device related sepsis
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Ear infection
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Gonococcal infection
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Pseudomonas infection
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Staphylococcal infection
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Streptococcal sepsis
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Investigations
Blood creatinine increased
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neuroblastoma
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Cerebral haemorrhage
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Vascular disorders
Superficial vein thrombosis
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
Other adverse events
| Measure |
Treatment Period: Ravulizumab
n=41 participants at risk
Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight.
|
Follow-up Period: Ravulizumab
n=29 participants at risk
After the completion of the treatment period, participants were followed for 26 Weeks in Follow-up period (no study drug was administered during this period).
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
24.4%
10/41 • Number of events 14 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Abdominal pain
|
22.0%
9/41 • Number of events 13 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
10.3%
3/29 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Vomiting
|
19.5%
8/41 • Number of events 10 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Nausea
|
14.6%
6/41 • Number of events 13 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Constipation
|
12.2%
5/41 • Number of events 6 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
9.8%
4/41 • Number of events 6 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Gastrointestinal disorders
Pancreatitis
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
General disorders
Pyrexia
|
24.4%
10/41 • Number of events 20 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
20.7%
6/29 • Number of events 11 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
General disorders
Pain
|
9.8%
4/41 • Number of events 5 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
General disorders
Oedema peripheral
|
9.8%
4/41 • Number of events 8 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
General disorders
Fatigue
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Immune system disorders
Graft versus host disease in skin
|
7.3%
3/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Immune system disorders
Hypogammaglobulinaemia
|
7.3%
3/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Clostridium difficile infection
|
9.8%
4/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Staphylococcal infection
|
9.8%
4/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
COVID-19
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Blood and lymphatic system disorders
Neutropenia
|
12.2%
5/41 • Number of events 13 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Cardiac disorders
Pericardial effusion
|
14.6%
6/41 • Number of events 9 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Cardiac disorders
Bradycardia
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Cardiac disorders
Tachycardia
|
2.4%
1/41 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
10.3%
3/29 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Endocrine disorders
Hypothyroidism
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Endocrine disorders
Adrenal insufficiency
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Eye disorders
Ocular hypertension
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Blood and lymphatic system disorders
Anaemia
|
14.6%
6/41 • Number of events 77 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Sepsis
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Upper respiratory tract infection
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
2.4%
1/41 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
10.3%
3/29 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Rhinovirus infection
|
2.4%
1/41 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Infections and infestations
Candida infection
|
0.00%
0/41 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Investigations
Alanine aminotransferase increased
|
19.5%
8/41 • Number of events 12 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Investigations
Aspartate aminotransferase increased
|
17.1%
7/41 • Number of events 12 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 6 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Investigations
Weight decreased
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Investigations
Blood creatinine increased
|
7.3%
3/41 • Number of events 11 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
17.1%
7/41 • Number of events 7 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
12.2%
5/41 • Number of events 5 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
9.8%
4/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
9.8%
4/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Metabolism and nutrition disorders
Dehydration
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.8%
4/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.8%
4/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
9.8%
4/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Nervous system disorders
Headache
|
26.8%
11/41 • Number of events 22 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
10.3%
3/29 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Psychiatric disorders
Insomnia
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Renal and urinary disorders
Dysuria
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Renal and urinary disorders
Proteinuria
|
7.3%
3/41 • Number of events 8 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
17.1%
7/41 • Number of events 7 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
13.8%
4/29 • Number of events 7 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
9.8%
4/41 • Number of events 5 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
3.4%
1/29 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
2.4%
1/41 • Number of events 1 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
6.9%
2/29 • Number of events 2 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
7.3%
3/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
7.3%
3/41 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Vascular disorders
Hypotension
|
12.2%
5/41 • Number of events 7 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
10.3%
3/29 • Number of events 4 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
|
Vascular disorders
Hypertension
|
7.3%
3/41 • Number of events 3 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
0.00%
0/29 • Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
|
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