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Trial record 18 of 73 for:    "Paroxysmal Nocturnal Hemoglobinuria"

Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02605993
Recruitment Status : Active, not recruiting
First Posted : November 17, 2015
Results First Posted : February 18, 2019
Last Update Posted : February 18, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Paroxysmal Nocturnal Hemoglobinuria
PNH
Intervention Biological: Ravulizumab
Enrollment 26
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description

During the Treatment Period, participants were administered ravulizumab 1400 milligrams (mg) on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kilograms (kg), 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Period Title: Treatment Period
Started 6 6 7 7
Received at Least 1 Dose of Study Drug 6 6 7 7
Completed 6 6 7 7
Not Completed 0 0 0 0
Period Title: Extension Period
Started 6 6 7 7
Completed [1] 0 0 0 0
Not Completed 6 6 7 7
[1]
Extension Period ongoing
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4 Total
Hide Arm/Group Description

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Total of all reporting groups
Overall Number of Baseline Participants 6 6 7 7 26
Hide Baseline Analysis Population Description
Safety set: All participants who received at least 1 dose of ravulizumab.
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 6 participants 7 participants 7 participants 26 participants
43.1  (14.57) 48.6  (23.48) 37.3  (14.03) 48.5  (13.43) 44.3  (16.33)
[1]
Measure Description: Age at first infusion of study drug.
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 7 participants 7 participants 26 participants
Female
2
  33.3%
1
  16.7%
1
  14.3%
2
  28.6%
6
  23.1%
Male
4
  66.7%
5
  83.3%
6
  85.7%
5
  71.4%
20
  76.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 7 participants 7 participants 26 participants
Hispanic or Latino
1
  16.7%
0
   0.0%
0
   0.0%
0
   0.0%
1
   3.8%
Not Hispanic or Latino
4
  66.7%
4
  66.7%
7
 100.0%
7
 100.0%
22
  84.6%
Unknown or Not Reported
1
  16.7%
2
  33.3%
0
   0.0%
0
   0.0%
3
  11.5%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 7 participants 7 participants 26 participants
White
5
  83.3%
4
  66.7%
3
  42.9%
3
  42.9%
15
  57.7%
Asian
0
   0.0%
0
   0.0%
4
  57.1%
3
  42.9%
7
  26.9%
Not Reported
1
  16.7%
2
  33.3%
0
   0.0%
0
   0.0%
3
  11.5%
Other
0
   0.0%
0
   0.0%
0
   0.0%
1
  14.3%
1
   3.8%
Lactate Dehydrogenase (LDH) Levels  
Mean (Standard Deviation)
Unit of measure:  Units/liter (U/L)
Number Analyzed 6 participants 6 participants 7 participants 7 participants 26 participants
1026.88  (547.843) 1223.55  (149.693) 2127.57  (815.875) 2142.24  (366.511) 1668.90  (724.341)
1.Primary Outcome
Title Percent Change In LDH Levels From Baseline To Day 253 And Day 281
Hide Description The percent change in LDH levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time Frame Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: Participants in the safety set with a Baseline and at least 1 LDH measurement after first dose of ravulizumab. Data summarized only for participants with data at Baseline and the specified time point. Last observation carried forward (LOCF) was used for participants with a missing Day 253 or Day 281 assessment.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Overall Number of Participants Analyzed 6 6 7 7
Mean (Standard Deviation)
Unit of Measure: Percent Change
Day 253 Number Analyzed 6 participants 6 participants 7 participants 7 participants
-72.85  (12.082) -77.82  (6.474) -84.96  (4.423) -87.63  (6.923)
Day 281 Number Analyzed 0 participants 0 participants 0 participants 6 participants
-89.58  (3.037)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1, Cohort 2, Cohort 3, Cohort 4
Comments Statistical Analysis presented is of Cohorts 1 to 4 combined at Day 253. A sample size of 20 participants from the combined cohorts was required to provide approximately 95% power to detect a mean paired difference in LDH from Baseline of –40% at Day 253 for Cohorts 1 to 4, with an estimated standard deviation (SD) of 45%. This was based on a 2-sided paired t-test, with 5% type I error rate. To account for a possible 15% dropout rate, up to 26 participants were enrolled.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0 for the combined cohorts.
Method Mixed Model for Repeated Measures (MMRM)
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 4
Comments Statistical Analysis presented is of Cohort 4 at Day 281. A sample size of 20 participants from the combined cohorts was required to provide approximately 95% power to detect a mean paired difference in LDH from Baseline of –40% at Day 281 for Cohort 4 only, with an estimated SD of 45%. This was based on a 2-sided paired t-test, with 5% type I error rate. To account for a possible 15% dropout rate, up to 26 participants were enrolled.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0.
Method MMRM
Comments [Not Specified]
2.Secondary Outcome
Title Percent Change In Free Hemoglobin Levels From Baseline To Day 253 And Day 281
Hide Description The percent change in free hemoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time Frame Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: Participants in the safety set with a Baseline and at least 1 LDH measurement after first dose of ravulizumab. Data summarized only for participants with data at Baseline and the specified time point. LOCF was used for participants with a missing Day 253 or Day 281 assessment.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Overall Number of Participants Analyzed 6 6 7 5
Mean (Standard Deviation)
Unit of Measure: Percent Change
Day 253 Number Analyzed 6 participants 6 participants 7 participants 5 participants
14.07  (124.755) -10.00  (55.812) -22.14  (94.388) -40.80  (27.474)
Day 281 Number Analyzed 0 participants 0 participants 0 participants 5 participants
-34.74  (26.534)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1, Cohort 2, Cohort 3, Cohort 4
Comments Statistical Analysis presented is of Cohorts 1 to 4 combined at Day 253.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6980
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0 for the combined cohorts.
Method MMRM
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 4
Comments Statistical Analysis presented is of Cohort 4 at Day 281.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3641
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0.
Method MMRM
Comments [Not Specified]
3.Secondary Outcome
Title Percent Change In Haptoglobin Levels From Baseline To Day 253 And Day 281
Hide Description The percent change in haptoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time Frame Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: Participants in the safety set with a Baseline and at least 1 LDH measurement after first dose of ravulizumab. Data summarized only for participants with data at Baseline and the specified time point. LOCF was used for participants with a missing Day 253 or Day 281 assessment.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Overall Number of Participants Analyzed 6 6 7 6
Mean (Standard Deviation)
Unit of Measure: Percent Change
Day 253 Number Analyzed 6 participants 6 participants 7 participants 6 participants
21.67  (53.072) 81.67  (200.042) 4.29  (11.339) 34.29  (74.578)
Day 281 Number Analyzed 0 participants 0 participants 0 participants 6 participants
6.67  (16.330)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1, Cohort 2, Cohort 3, Cohort 4
Comments Statistical Analysis presented is of Cohorts 1 to 4 combined at Day 253.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4037
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0 for the combined cohorts.
Method MMRM
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 4
Comments Statistical Analysis presented is of Cohort 4 at Day 281.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2520
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0.
Method MMRM
Comments [Not Specified]
4.Secondary Outcome
Title Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 253 And Day 281
Hide Description The percent change in reticulocyte/erythrocyte count levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time Frame Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: Participants in the safety set with a Baseline and at least 1 LDH measurement after first dose of ravulizumab. Data summarized only for participants with data at Baseline and the specified time point. LOCF was used for participants with a missing Day 253 or Day 281 assessment.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Overall Number of Participants Analyzed 6 6 7 7
Mean (Standard Deviation)
Unit of Measure: Percent Change
Day 253 Number Analyzed 6 participants 6 participants 7 participants 7 participants
-1.27  (43.019) -2.35  (34.538) 10.46  (59.510) 14.66  (81.390)
Day 281 Number Analyzed 0 participants 0 participants 0 participants 7 participants
2.12  (72.427)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1, Cohort 2, Cohort 3, Cohort 4
Comments Statistical Analysis presented is of Cohorts 1 to 4 combined at Day 253.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4686
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0 for the combined cohorts.
Method MMRM
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 4
Comments Statistical Analysis presented is of Cohort 4 at Day 281.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6296
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0.
Method MMRM
Comments [Not Specified]
5.Secondary Outcome
Title Percent Change In PNH RBC Types II And III Clone Size From Baseline To Day 253
Hide Description The percent change in paroxysmal nocturnal hemoglobinuria (PNH) red blood cell (RBC), summed types II and III, clone size levels were assessed from Baseline to Day 253 for Cohorts 1 to 4.
Time Frame Baseline, Day 253 (Cohorts 1 to 4)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: Participants in the safety set with a Baseline and at least 1 LDH measurement after first dose of ravulizumab. Data summarized only for participants with data at Baseline and the specified time point. LOCF was used for participants with a missing Day 253 assessment.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Overall Number of Participants Analyzed 6 6 7 6
Mean (Standard Deviation)
Unit of Measure: Percent Change
-5.28  (35.096) -1.15  (19.175) 36.44  (103.514) 46.53  (54.093)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1, Cohort 2, Cohort 3, Cohort 4
Comments Statistical Analysis presented is of Cohorts 1 to 4 combined at Day 253.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0705
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0 for the combined cohorts.
Method MMRM
Comments [Not Specified]
6.Secondary Outcome
Title Percent Change In D-dimer From Baseline To Day 253 And Day 281
Hide Description The percent change in D-dimer levels were assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time Frame Baseline to Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: Participants in the safety set with a Baseline and at least 1 LDH measurement after first dose of ravulizumab. Data summarized only for participants with data at Baseline and the specified time point. LOCF is used for participants with a missing Day 253 or Day 281 assessment.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Overall Number of Participants Analyzed 6 6 7 7
Mean (Standard Deviation)
Unit of Measure: Percent Change
Day 253 Number Analyzed 6 participants 6 participants 7 participants 7 participants
-24.80  (32.436) -29.47  (26.547) -10.90  (41.032) -16.08  (34.783)
Day 281 Number Analyzed 0 participants 0 participants 0 participants 5 participants
-32.46  (27.149)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 1, Cohort 2, Cohort 3, Cohort 4
Comments Statistical Analysis presented is of Cohorts 1 to 4 combined at Day 253.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0900
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0 for the combined cohorts.
Method MMRM
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort 4
Comments Statistical Analysis presented is of Cohort 4 at Day 281.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2302
Comments Hypothesis testing was performed at the 0.05 level of significance. P-value tested whether the percent changes differed from 0.
Method MMRM
Comments [Not Specified]
7.Secondary Outcome
Title Change In Clinical Manifestations Of PNH From Baseline To Day 253 And Day 281
Hide Description Clinical manifestations were assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only. Clinical manifestations were defined as fatigue, abdominal pain, dyspnea, dysphagia, chest pain, and erectile dysfunction (male participants only). Improvement was defined as present at Baseline and absent at Day endpoint. Worsening was defined as absent at Baseline and present at Day endpoint. No Change was defined as no change from Baseline and time point of endpoint.
Time Frame Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: Participants in the safety set with a Baseline and at least 1 LDH measurement after first dose of ravulizumab. Data summarized only for participants with data at Baseline and the specified time point. LOCF is used for participants with a missing Day 253 or Day 281 assessment. Erectile dysfunction affects only male participants.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

Overall Number of Participants Analyzed 6 6 7 7
Measure Type: Count of Participants
Unit of Measure: Participants
Fatigue at Day 253 Number Analyzed 6 participants 6 participants 7 participants 7 participants
Improved from Baseline
4
  66.7%
2
  33.3%
3
  42.9%
3
  42.9%
Worsened from Baseline
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
No Change
2
  33.3%
4
  66.7%
4
  57.1%
4
  57.1%
Fatigue at Day 281 Number Analyzed 0 participants 0 participants 0 participants 6 participants
Improved from Baseline
4
  66.7%
Worsened from Baseline
0
   0.0%
No Change
2
  33.3%
Abdominal Pain at Day 253 Number Analyzed 6 participants 6 participants 7 participants 7 participants
Improved from Baseline
1
  16.7%
1
  16.7%
1
  14.3%
0
   0.0%
Worsened from Baseline
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
No Change
5
  83.3%
5
  83.3%
6
  85.7%
7
 100.0%
Abdominal Pain at Day 281 Number Analyzed 0 participants 0 participants 0 participants 6 participants
Improved from Baseline
0
   0.0%
Worsened from Baseline
0
   0.0%
No Change
6
 100.0%
Dyspnea at Day 253 Number Analyzed 6 participants 6 participants 7 participants 7 participants
Improved from Baseline
1
  16.7%
1
  16.7%
4
  57.1%
2
  28.6%
Worsened from Baseline
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
No Change
5
  83.3%
5
  83.3%
3
  42.9%
5
  71.4%
Dyspnea at Day 281 Number Analyzed 0 participants 0 participants 0 participants 6 participants
Improved from Baseline
2
  33.3%
Worsened from Baseline
0
   0.0%
No Change
4
  66.7%
Dysphagia at Day 253 Number Analyzed 6 participants 6 participants 7 participants 7 participants
Improved from Baseline
0
   0.0%
1
  16.7%
1
  14.3%
1
  14.3%
Worsened from Baseline
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
No Change
6
 100.0%
5
  83.3%
6
  85.7%
6
  85.7%
Dysphagia at Day 281 Number Analyzed 0 participants 0 participants 0 participants 6 participants
Improved from Baseline
1
  16.7%
Worsened from Baseline
0
   0.0%
No Change
5
  83.3%
Chest Pain at Day 253 Number Analyzed 6 participants 6 participants 7 participants 7 participants
Improved from Baseline
1
  16.7%
0
   0.0%
2
  28.6%
0
   0.0%
Worsened from Baseline
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
No Change
5
  83.3%
6
 100.0%
5
  71.4%
7
 100.0%
Chest Pain at Day 281 Number Analyzed 0 participants 0 participants 0 participants 6 participants
Improved from Baseline
0
   0.0%
Worsened from Baseline
0
   0.0%
No Change
6
 100.0%
Erectile Dysfunction at Day 253 Number Analyzed 4 participants 5 participants 6 participants 5 participants
Improved from Baseline
2
  50.0%
0
   0.0%
1
  16.7%
1
  20.0%
Worsened from Baseline
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
No Change
2
  50.0%
5
 100.0%
5
  83.3%
4
  80.0%
Erectile Dysfunction at Day 281 Number Analyzed 0 participants 0 participants 0 participants 4 participants
Improved from Baseline
1
  25.0%
Worsened from Baseline
0
   0.0%
No Change
3
  75.0%
Time Frame Adverse events were monitored continuously from Screening to Day 253 (for Cohorts 1, 2, and 3) and Day 281 (for Cohort 4) (Treatment Period).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description

During the Treatment Period, participants were administered ravulizumab 1400 mg on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses.

In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.

During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses.

During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.

All-Cause Mortality
Cohort 1 Cohort 2 Cohort 3 Cohort 4
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/6 (0.00%)   0/6 (0.00%)   0/7 (0.00%)   0/7 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Cohort 1 Cohort 2 Cohort 3 Cohort 4
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/6 (33.33%)   2/6 (33.33%)   3/7 (42.86%)   1/7 (14.29%) 
Blood and lymphatic system disorders         
Febrile neutropenia  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Haemolysis  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Gastrointestinal disorders         
Nausea  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
General disorders         
Pyrexia  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Infections and infestations         
Meningococcal infection  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Meningococcal sepsis  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Urinary tract infection  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Injury, poisoning and procedural complications         
Post procedural complication  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Papillary thyroid cancer  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Nervous system disorders         
Headache  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Skin and subcutaneous tissue disorders         
Rash maculo-papular  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
1
Term from vocabulary, MedDRA (20.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cohort 1 Cohort 2 Cohort 3 Cohort 4
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   6/6 (100.00%)   6/6 (100.00%)   7/7 (100.00%)   7/7 (100.00%) 
Blood and lymphatic system disorders         
Anaemia  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Extravascular haemolysis  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Neutropenia  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Cardiac disorders         
Angina pectoris  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Cardiogenic shock  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Myocarditis  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Tachycardia  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Ear and labyrinth disorders         
Vertigo  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Eye disorders         
Dry eye  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Photophobia  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Gastrointestinal disorders         
Abdominal distension  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Abdominal pain  1  2/6 (33.33%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Aphthous ulcer  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Constipation  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  1/7 (14.29%) 
Diarrhoea  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Dyspepsia  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Dysphagia  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Gastritis  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Gastrointestinal sounds abnormal  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Nausea  1  2/6 (33.33%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Vomiting  1  0/6 (0.00%)  0/6 (0.00%)  2/7 (28.57%)  0/7 (0.00%) 
General disorders         
Asthenia  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Chest pain  1  1/6 (16.67%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Fatigue  1  3/6 (50.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Infusion site swelling  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Localised oedema  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Mass  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Pain  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Vaccination site bruising  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Immune system disorders         
Seasonal allergy  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Infections and infestations         
Bronchitis  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Cystitis  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  1/7 (14.29%) 
Gastroenteritis  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Hordeolum  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Otitis media  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Sinusitis  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Tooth abscess  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Upper respiratory tract infection  1  2/6 (33.33%)  0/6 (0.00%)  2/7 (28.57%)  3/7 (42.86%) 
Urinary tract infection  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Viral infection  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Viral skin infection  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Viral upper respiratory tract infection  1  2/6 (33.33%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Injury, poisoning and procedural complications         
Ligament sprain  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Post-traumatic pain  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Investigations         
Alanine aminotransferase increased  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Antinuclear antibody positive  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Aspartate aminotransferase increased  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Blood bilirubin increased  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Blood creatinine increased  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Blood lactate dehydrogenase increased  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Neutrophil count decreased  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Metabolism and nutrition disorders         
Dyslipidaemia  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Fluid overload  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Hypokalaemia  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Hypophosphataemia  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Iron deficiency  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Musculoskeletal and connective tissue disorders         
Arthralgia  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Back pain  1  1/6 (16.67%)  0/6 (0.00%)  1/7 (14.29%)  1/7 (14.29%) 
Bone pain  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Flank pain  1  0/6 (0.00%)  1/6 (16.67%)  1/7 (14.29%)  0/7 (0.00%) 
Muscle spasms  1  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%)  2/7 (28.57%) 
Musculoskeletal stiffness  1  1/6 (16.67%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Myalgia  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Neck pain  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Tendonitis  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Nervous system disorders         
Dizziness  1  0/6 (0.00%)  0/6 (0.00%)  2/7 (28.57%)  0/7 (0.00%) 
Headache  1  3/6 (50.00%)  3/6 (50.00%)  4/7 (57.14%)  3/7 (42.86%) 
Presyncope  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Psychiatric disorders         
Insomnia  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Restlessness  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Renal and urinary disorders         
Chromaturia  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Haemoglobinuria  1  1/6 (16.67%)  0/6 (0.00%)  1/7 (14.29%)  1/7 (14.29%) 
Renal colic  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  0/7 (0.00%) 
Reproductive system and breast disorders         
Dysmenorrhoea  1 [1]  1/2 (50.00%)  0/1 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Ovarian cyst  1 [1]  1/2 (50.00%)  0/1 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Testicular pain  1 [2]  0/4 (0.00%)  1/5 (20.00%)  0/6 (0.00%)  0/5 (0.00%) 
Testicular swelling  1 [2]  0/4 (0.00%)  1/5 (20.00%)  0/6 (0.00%)  0/5 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Cough  1  0/6 (0.00%)  0/6 (0.00%)  2/7 (28.57%)  0/7 (0.00%) 
Dyspnoea  1  3/6 (50.00%)  0/6 (0.00%)  0/7 (0.00%)  1/7 (14.29%) 
Oropharyngeal pain  1  1/6 (16.67%)  1/6 (16.67%)  1/7 (14.29%)  0/7 (0.00%) 
Pleural effusion  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Rhinitis allergic  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Rhinorrhoea  1  1/6 (16.67%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Skin and subcutaneous tissue disorders         
Night sweats  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Petechiae  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Skin lesion  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Swelling face  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
Vascular disorders         
Extremity necrosis  1  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%)  0/7 (0.00%) 
Hypertension  1  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%)  1/7 (14.29%) 
Thrombophlebitis superficial  1  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%)  0/7 (0.00%) 
1
Term from vocabulary, MedDRA (20.0)
Indicates events were collected by systematic assessment
[1]
This was a sex-specific adverse event that only affected female participants.
[2]
This was a sex-specific adverse event that only affected male participants.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Alexion Pharmaceuticals Inc.
Organization: Alexion Pharmaceuticals Inc.
Phone: 855-752-2356
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02605993     History of Changes
Other Study ID Numbers: ALXN1210-PNH-201
2015-002674-20 ( EudraCT Number )
First Submitted: November 11, 2015
First Posted: November 17, 2015
Results First Submitted: January 18, 2019
Results First Posted: February 18, 2019
Last Update Posted: February 18, 2019