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Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)

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ClinicalTrials.gov Identifier: NCT04469465
Recruitment Status : Completed
First Posted : July 14, 2020
Results First Posted : July 24, 2023
Last Update Posted : February 6, 2024
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Paroxysmal Nocturnal Hemoglobinuria
Interventions Drug: Danicopan
Drug: Placebo
Drug: C5 Inhibitor
Enrollment 86
Recruitment Details  
Pre-assignment Details The study consists of 2 treatment periods and a Long-term Extension (LTE) period. This study is ongoing, and the results of treatment period 1 (up to Week 12) have been reported here. The final results will be reported after the completion of study.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description Participants received danicopan 3 times daily (TID) for 12 weeks, in addition to their background eculizumab or ravulizumab therapy. Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Period Title: Overall Study
Started 57 29
Received at Least 1 Dose of Study Drug 57 29
Interim Efficacy Analysis Set [1] 42 21
Completed [2] 48 23
Not Completed 9 6
Reason Not Completed
Adverse Event             2             1
Ongoing treatment             7             4
Withdrawal by Subject             0             1
[1]
Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
[2]
Completed TP1
Arm/Group Title Danicopan Placebo Total
Hide Arm/Group Description Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy. Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy. Total of all reporting groups
Overall Number of Baseline Participants 57 29 86
Hide Baseline Analysis Population Description
The safety set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 57 participants 29 participants 86 participants
52.8  (17.00) 52.9  (14.34) 52.8  (16.07)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 57 participants 29 participants 86 participants
Female
34
  59.6%
20
  69.0%
54
  62.8%
Male
23
  40.4%
9
  31.0%
32
  37.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 57 participants 29 participants 86 participants
Hispanic or Latino
6
  10.5%
1
   3.4%
7
   8.1%
Not Hispanic or Latino
46
  80.7%
24
  82.8%
70
  81.4%
Unknown or Not Reported
5
   8.8%
4
  13.8%
9
  10.5%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 57 participants 29 participants 86 participants
American Indian or Alaska Native
1
   1.8%
0
   0.0%
1
   1.2%
Asian
22
  38.6%
10
  34.5%
32
  37.2%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
   3.5%
0
   0.0%
2
   2.3%
White
28
  49.1%
14
  48.3%
42
  48.8%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
4
   7.0%
5
  17.2%
9
  10.5%
Hemoglobin (Hgb)  
Mean (Standard Deviation)
Unit of measure:  Grams (g)/liter (L)
Number Analyzed 57 participants 29 participants 86 participants
76.7  (9.47) 78.9  (10.11) 77.5  (9.69)
1.Primary Outcome
Title Change From Baseline in Hgb at Week 12
Hide Description Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 21
Least Squares Mean (Standard Error)
Unit of Measure: g/L
29.40  (2.107) 4.96  (3.128)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Danicopan, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0007
Comments [Not Specified]
Method Re-randomization test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Danicopan, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value 24.44
Confidence Interval (2-Sided) 95%
16.90 to 31.99
Parameter Dispersion
Type: Standard Error of the Mean
Value: 3.751
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Percentage of Participants With Hgb Increase of ≥2 g/dL (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12
Hide Description The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 12 and remaining transfusion free during the 12-Week TP1. Participants who withdrew from the study early during the 12-Week TP1 or had missing Hgb value at Week 12 were considered as not achieving the criterion.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 21
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
59.5
(43.28 to 74.37)
0
(0.00 to 16.11)
3.Secondary Outcome
Title Percentage of Participants With Transfusion Avoidance Through Week 12
Hide Description Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 12. Participants who discontinued study treatment early before Week 12 were considered as not achieving transfusion avoidance.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 21
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
83.3
(68.64 to 93.03)
38.1
(18.11 to 61.56)
4.Secondary Outcome
Title Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12
Hide Description The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue better health-related quality of life. LS mean and SE were produced using MMRM.
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 21
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
7.97  (1.128) 1.85  (1.581)
5.Secondary Outcome
Title Change From Baseline in Absolute Reticulocyte Count at Week 12
Hide Description LS mean and SE were produced using MMRM.
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 20
Least Squares Mean (Standard Error)
Unit of Measure: 10^12 cells/L
-0.0838  (0.00893) 0.0035  (0.01268)
6.Secondary Outcome
Title Change in the Number of Red Blood Cell (RBC) Units Transfused From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Hide Description [Not Specified]
Time Frame 24 weeks prior to initiation of treatment to post 24 weeks of treatment
Outcome Measure Data Not Reported
7.Secondary Outcome
Title Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Hide Description [Not Specified]
Time Frame 24 weeks prior to initiation of treatment to post 24 weeks of treatment
Outcome Measure Data Not Reported
8.Secondary Outcome
Title Percentage of Participants With Transfusion Avoidance Through Week 24
Hide Description [Not Specified]
Time Frame Week 24
Outcome Measure Data Not Reported
9.Secondary Outcome
Title Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
Hide Description LS mean and SE were produced using analysis of covariance (ANCOVA).
Time Frame 12 weeks prior to initiation of treatment to post 12 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 21
Least Squares Mean (Standard Error)
Unit of Measure: RBC units
-1.48  (0.271) -0.18  (0.383)
10.Secondary Outcome
Title Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
Hide Description LS mean and SE were produced using ANCOVA.
Time Frame 12 weeks prior to initiation of treatment to post 12 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 21
Least Squares Mean (Standard Error)
Unit of Measure: transfusion instances
-0.92  (0.174) -0.21  (0.246)
11.Secondary Outcome
Title Change From Baseline FACIT Fatigue Scores at Week 24
Hide Description [Not Specified]
Time Frame Baseline, Week 24
Outcome Measure Data Not Reported
12.Secondary Outcome
Title Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan
Hide Description [Not Specified]
Time Frame Week 12 to Week 24
Outcome Measure Data Not Reported
13.Secondary Outcome
Title Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24
Hide Description [Not Specified]
Time Frame Week 24
Outcome Measure Data Not Reported
14.Secondary Outcome
Title Change From Baseline in Total and Direct Bilirubin at Week 12
Hide Description Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 21
Least Squares Mean (Standard Error)
Unit of Measure: micromoles (μmol)/L
Total Bilirubin -9.77  (1.692) -2.15  (2.377)
Direct Bilirubin -2.88  (0.357) 0.30  (0.503)
15.Secondary Outcome
Title Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12
Hide Description The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 14 8
Least Squares Mean (Standard Error)
Unit of Measure: percentage of the total cell population
24.60  (4.180) -3.04  (5.864)
16.Secondary Outcome
Title Change From Baseline in C3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12
Hide Description Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 23 10
Least Squares Mean (Standard Error)
Unit of Measure: percentage of the total cell population
-15.06  (2.824) 0.89  (4.394)
17.Secondary Outcome
Title Change From Baseline in Lactate Dehydrogenase at Week 12
Hide Description Baseline was defined as the average of all available assessments prior to the first dose of study intervention. LS mean and SE were produced using MMRM.
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 20
Least Squares Mean (Standard Error)
Unit of Measure: units (U)/L
-23.49  (8.287) -2.92  (11.914)
18.Secondary Outcome
Title Percentage of Participants With Hgb Normalization at Week 12
Hide Description Hgb normalization was defined as Hgb value above lower limit of normal (LLN) reference range. For male, the LLN was 125 grams (g)/liter (L), for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 12 were considered as not meeting Hgb normalization regardless of actual value observed at Week 12.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of randomized participants formed the Interim Analysis Set for efficacy analysis.
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description:
Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
Overall Number of Participants Analyzed 42 21
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
28.6
(15.72 to 44.58)
0
(0.00 to 16.11)
19.Secondary Outcome
Title Percentage of Participants With Hgb Normalization at Week 24
Hide Description [Not Specified]
Time Frame Week 24
Outcome Measure Data Not Reported
Time Frame Baseline up to Week 12
Adverse Event Reporting Description

The safety set included all participants that received at least 1 dose of study drug (danicopan or placebo).

The AE data of treatment period 1 (up to Week 12) has been reported here. The final AE data will be reported after the completion of study.
 
Arm/Group Title Danicopan Placebo
Hide Arm/Group Description Participants received danicopan TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy. Participants received placebo TID for 12 weeks, in addition to their background eculizumab or ravulizumab therapy.
All-Cause Mortality
Danicopan Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/57 (0.00%)   0/29 (0.00%) 
Hide Serious Adverse Events
Danicopan Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   3/57 (5.26%)   2/29 (6.90%) 
Blood and lymphatic system disorders     
Anaemia † 1  0/57 (0.00%)  1/29 (3.45%) 
Gastrointestinal disorders     
Abdominal pain † 1  0/57 (0.00%)  1/29 (3.45%) 
Pancreatitis † 1  1/57 (1.75%)  0/29 (0.00%) 
Hepatobiliary disorders     
Cholecystitis † 1  1/57 (1.75%)  0/29 (0.00%) 
Infections and infestations     
COVID-19 † 1  1/57 (1.75%)  0/29 (0.00%) 
Investigations     
Blood bilirubin increased † 1  1/57 (1.75%)  0/29 (0.00%) 
Nervous system disorders     
Headache † 1  0/57 (0.00%)  1/29 (3.45%) 
1
Term from vocabulary, MedDRA 25.1
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Danicopan Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   42/57 (73.68%)   18/29 (62.07%) 
Blood and lymphatic system disorders     
Haemolysis † 1  2/57 (3.51%)  0/29 (0.00%) 
Leukopenia † 1  1/57 (1.75%)  0/29 (0.00%) 
Neutropenia † 1  1/57 (1.75%)  0/29 (0.00%) 
Splenomegaly † 1  1/57 (1.75%)  0/29 (0.00%) 
Anaemia † 1  1/57 (1.75%)  3/29 (10.34%) 
Thrombocytopenia † 1  0/57 (0.00%)  1/29 (3.45%) 
Eye disorders     
Dry eye † 1  1/57 (1.75%)  0/29 (0.00%) 
Gastrointestinal disorders     
Nausea † 1  5/57 (8.77%)  3/29 (10.34%) 
Diarrhoea † 1  4/57 (7.02%)  3/29 (10.34%) 
Vomiting † 1  3/57 (5.26%)  0/29 (0.00%) 
Abdominal discomfort † 1  1/57 (1.75%)  0/29 (0.00%) 
Abdominal pain upper † 1  1/57 (1.75%)  2/29 (6.90%) 
Constipation † 1  2/57 (3.51%)  1/29 (3.45%) 
Dyspepsia † 1  1/57 (1.75%)  1/29 (3.45%) 
Flatulence † 1  1/57 (1.75%)  0/29 (0.00%) 
Noninfective gingivitis † 1  1/57 (1.75%)  0/29 (0.00%) 
Pancreatitis † 1  1/57 (1.75%)  0/29 (0.00%) 
Salivary gland pain † 1  1/57 (1.75%)  0/29 (0.00%) 
Stomatitis † 1  1/57 (1.75%)  0/29 (0.00%) 
Abdominal pain † 1  0/57 (0.00%)  1/29 (3.45%) 
Discoloured vomit † 1  1/57 (1.75%)  0/29 (0.00%) 
General disorders     
Pyrexia † 1  3/57 (5.26%)  0/29 (0.00%) 
Chest discomfort † 1  1/57 (1.75%)  0/29 (0.00%) 
Fatigue † 1  1/57 (1.75%)  1/29 (3.45%) 
Oedema peripheral † 1  1/57 (1.75%)  1/29 (3.45%) 
Peripheral swelling † 1  1/57 (1.75%)  0/29 (0.00%) 
Asthenia † 1  0/57 (0.00%)  4/29 (13.79%) 
Vessel puncture site pain † 1  0/57 (0.00%)  1/29 (3.45%) 
Hepatobiliary disorders     
Hepatic function abnormal † 1  1/57 (1.75%)  0/29 (0.00%) 
Jaundice † 1  1/57 (1.75%)  0/29 (0.00%) 
Liver disorder † 1  1/57 (1.75%)  0/29 (0.00%) 
Infections and infestations     
Localised infection † 1  1/57 (1.75%)  0/29 (0.00%) 
COVID-19 † 1  1/57 (1.75%)  0/29 (0.00%) 
Cellulitis † 1  1/57 (1.75%)  0/29 (0.00%) 
Upper respiratory tract infection † 1  1/57 (1.75%)  0/29 (0.00%) 
Urinary tract infection † 1  2/57 (3.51%)  1/29 (3.45%) 
Viral infection † 1  2/57 (3.51%)  0/29 (0.00%) 
Gastroenteritis viral † 1  0/57 (0.00%)  1/29 (3.45%) 
Genital herpes † 1  0/57 (0.00%)  1/29 (3.45%) 
Gingivitis † 1  0/57 (0.00%)  1/29 (3.45%) 
Periodontitis † 1  0/57 (0.00%)  1/29 (3.45%) 
Tooth abscess † 1  0/57 (0.00%)  1/29 (3.45%) 
Sinusitis † 1  1/57 (1.75%)  0/29 (0.00%) 
Cystitis † 1  0/57 (0.00%)  1/29 (3.45%) 
Ear infection † 1  0/57 (0.00%)  2/29 (6.90%) 
Injury, poisoning and procedural complications     
Contusion † 1  1/57 (1.75%)  3/29 (10.34%) 
Fall † 1  1/57 (1.75%)  1/29 (3.45%) 
Febrile nonhaemolytic transfusion reaction † 1  1/57 (1.75%)  0/29 (0.00%) 
Post procedural contusion † 1  1/57 (1.75%)  0/29 (0.00%) 
Post procedural diarrhoea † 1  1/57 (1.75%)  0/29 (0.00%) 
Post procedural haemorrhage † 1  1/57 (1.75%)  0/29 (0.00%) 
Skin abrasion † 1  1/57 (1.75%)  0/29 (0.00%) 
Thoracic vertebral fracture † 1  1/57 (1.75%)  0/29 (0.00%) 
Investigations     
Alanine aminotransferase increased † 1  3/57 (5.26%)  1/29 (3.45%) 
Aspartate aminotransferase increased † 1  2/57 (3.51%)  3/29 (10.34%) 
White blood cell count decreased † 1  2/57 (3.51%)  0/29 (0.00%) 
Blood bilirubin increased † 1  1/57 (1.75%)  0/29 (0.00%) 
Blood pressure increased † 1  1/57 (1.75%)  0/29 (0.00%) 
Hepatic enzyme increased † 1  1/57 (1.75%)  0/29 (0.00%) 
Neutrophil count decreased † 1  1/57 (1.75%)  0/29 (0.00%) 
Platelet count decreased † 1  1/57 (1.75%)  0/29 (0.00%) 
Haemoglobin decreased † 1  0/57 (0.00%)  1/29 (3.45%) 
Blood lactate dehydrogenase increased † 1  1/57 (1.75%)  0/29 (0.00%) 
Lymphocyte count decreased † 1  1/57 (1.75%)  0/29 (0.00%) 
SARS-CoV-2 test positive † 1  1/57 (1.75%)  0/29 (0.00%) 
Metabolism and nutrition disorders     
Decreased appetite † 1  1/57 (1.75%)  0/29 (0.00%) 
Hypercholesterolaemia † 1  1/57 (1.75%)  0/29 (0.00%) 
Hypomagnesaemia † 1  0/57 (0.00%)  1/29 (3.45%) 
Musculoskeletal and connective tissue disorders     
Arthralgia † 1  4/57 (7.02%)  2/29 (6.90%) 
Myalgia † 1  2/57 (3.51%)  0/29 (0.00%) 
Pain in extremity † 1  3/57 (5.26%)  0/29 (0.00%) 
Bone pain † 1  1/57 (1.75%)  0/29 (0.00%) 
Osteoarthritis † 1  1/57 (1.75%)  0/29 (0.00%) 
Muscle fatigue † 1  0/57 (0.00%)  1/29 (3.45%) 
Muscle spasms † 1  0/57 (0.00%)  1/29 (3.45%) 
Osteopenia † 1  0/57 (0.00%)  1/29 (3.45%) 
Nervous system disorders     
Headache † 1  6/57 (10.53%)  2/29 (6.90%) 
Dizziness † 1  1/57 (1.75%)  2/29 (6.90%) 
Lethargy † 1  1/57 (1.75%)  0/29 (0.00%) 
Somnolence † 1  0/57 (0.00%)  1/29 (3.45%) 
Psychiatric disorders     
Hallucination † 1  1/57 (1.75%)  0/29 (0.00%) 
Insomnia † 1  1/57 (1.75%)  3/29 (10.34%) 
Renal and urinary disorders     
Chromaturia † 1  1/57 (1.75%)  1/29 (3.45%) 
Haematuria † 1  0/57 (0.00%)  1/29 (3.45%) 
Haemoglobinuria † 1  0/57 (0.00%)  1/29 (3.45%) 
Proteinuria † 1  1/57 (1.75%)  0/29 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Oropharyngeal pain † 1  2/57 (3.51%)  0/29 (0.00%) 
Rhinorrhoea † 1  2/57 (3.51%)  0/29 (0.00%) 
Epistaxis † 1  1/57 (1.75%)  0/29 (0.00%) 
Sinus pain † 1  1/57 (1.75%)  0/29 (0.00%) 
Dyspnoea † 1  0/57 (0.00%)  1/29 (3.45%) 
Dyspnoea exertional † 1  0/57 (0.00%)  1/29 (3.45%) 
Cough † 1  1/57 (1.75%)  0/29 (0.00%) 
Skin and subcutaneous tissue disorders     
Acne † 1  1/57 (1.75%)  0/29 (0.00%) 
Rash maculo-papular † 1  1/57 (1.75%)  0/29 (0.00%) 
Dyshidrotic eczema † 1  0/57 (0.00%)  1/29 (3.45%) 
Eczema † 1  0/57 (0.00%)  1/29 (3.45%) 
Vascular disorders     
Hypertension † 1  3/57 (5.26%)  1/29 (3.45%) 
Flushing † 1  1/57 (1.75%)  0/29 (0.00%) 
Hot flush † 1  1/57 (1.75%)  0/29 (0.00%) 
Pallor † 1  0/57 (0.00%)  1/29 (3.45%) 
Poor venous access † 1  0/57 (0.00%)  1/29 (3.45%) 
1
Term from vocabulary, MedDRA 25.1
†
Indicates events were collected by systematic assessment
[Not Specified]
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
Layout table for Results Point of Contact information
Name/Title: Alexion Pharmaceuticals Inc.
Organization: Alexion Pharmaceuticals Inc.
Phone: +1 855-752-2356
EMail: clinicaltrials@alexion.com
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Responsible Party: Alexion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04469465    
Other Study ID Numbers: ALXN2040-PNH-301
First Submitted: July 9, 2020
First Posted: July 14, 2020
Results First Submitted: June 28, 2023
Results First Posted: July 24, 2023
Last Update Posted: February 6, 2024