Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysm... | NCT04469465 | Trialant
NCT04469465
Sponsor
Alexion Pharmaceuticals, Inc.
Status
Completed
Last Update Posted
May 4, 2025Actual
Enrollment
86Actual
Phase
Phase 3
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Danicopan
Placebo
C5 Inhibitor
Countries
United States
Brazil
Canada
Czechia
France
Germany
Greece
Israel
Italy
Japan
Malaysia
Netherlands
Poland
South Korea
Spain
Taiwan
Thailand
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT04469465
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
ALXN2040-PNH-301
Secondary IDs
ID
Type
Description
Link
2019-003829-18
EudraCT Number
Brief Title
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
Official Title
A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
Acronym
Not provided
Organization
Alexion Pharmaceuticals, Inc.INDUSTRY
Status Module
Record Verification Date
Apr 2025
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
Not provided
Start Date
Dec 16, 2020Actual
Primary Completion Date
Jun 29, 2022Actual
Completion Date
Jan 16, 2024Actual
First Submitted Date
Jul 9, 2020
First Submission Date that Met QC Criteria
Jul 9, 2020
First Posted Date
Jul 14, 2020Actual
Results Waived
Not provided
Results First Submitted Date
Jun 28, 2023
Results First Submitted that Met QC Criteria
Jul 21, 2023
Results First Posted Date
Jul 24, 2023Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Apr 28, 2025
Last Update Posted Date
May 4, 2025Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Alexion Pharmaceuticals, Inc.INDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.
Detailed Description
This is a multiple-region, randomized, double-blind, placebo controlled, multiple-dose, study in participants with PNH who have clinically evident EVH on a C5 inhibitor (eculizumab or ravulizumab).
Participants will be randomized to receive danicopan or placebo, in a 2:1 ratio for 12 weeks (Treatment Period 1) in addition to their C5 inhibitor (eculizumab or ravulizumab) therapy. At Week 12, participants randomized to receive placebo will be switched to danicopan in addition to their C5 inhibitor for an additional 12 weeks (Treatment Period 2) and participants randomized to danicopan will continue on danicopan for an additional 12 weeks, while remaining on their ongoing C5 inhibitor therapy.
At the end of the 2 treatment periods (Week 24), participants may enter a Long-Term Extension (LTE) Period and continue to receive danicopan in addition to their C5 inhibitor therapy. The Long-Term Extension period will consist of a first year of LTE(Year1) and a second year of optional LTE(Year2).All patients will complete 72 weeks of LTE(Year 1) assessments. After Week 72 (at the end of the first year of LTE), patients have the choice to complete participation in this study or continue to the optional second year (Year2) of LTE.
Conditions Module
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Keywords
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Extravascular Hemolysis (EVH)
Factor D inhibitor
Complement
Danicopan
C5 inhibitor
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
86Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Danicopan + C5 Inhibitor
Experimental
Participants will receive danicopan, in addition to their C5 inhibitor therapy, for 24 weeks (12 weeks in Treatment Period 1, followed by 12 weeks in Treatment Period 2).
Drug: Danicopan
Drug: C5 Inhibitor
Placebo + C5 Inhibitor
Placebo Comparator
Participants will receive placebo, in addition to their C5 inhibitor therapy, for 12 weeks during Treatment Period 1. At Week 12, participants randomized to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2).
Drug: Placebo
Drug: C5 Inhibitor
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Danicopan
Drug
Oral tablet
Danicopan + C5 Inhibitor
ALXN2040
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Change From Baseline in Hgb at Week 12
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.
Baseline, Week 12
Secondary Outcomes
Measure
Description
Time Frame
Percentage of Participants With Hgb Increase of ≥2 Grams/Deciliter (g/dL) (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12
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.
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Diagnosis of PNH
Clinically Evident EVH defined by:
Anemia (Hgb ≤9.5 gram/deciliter) with absolute reticulocyte count ≥120 x 10^9/liter
Receiving an approved C5 inhibitor for at least 6 months prior to Day 1
Platelet count ≥30,000/microliters (µL)
Absolute neutrophil counts ≥500/μL
Documentation of/or willingness to receive vaccinations for N. meningiditis and prophylactic antibiotics as required
Exclusion Criteria:
History of a major organ transplant or hematopoietic stem cell transplantation (HSCT)
Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants
Known or suspected complement deficiency
Laboratory abnormalities at screening, including:
Alanine aminotransferase >2 x ULN (>3 x ULN in case of patients with documented liver iron overload defined by serum ferratin values
500 ng/ML)
Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome)
Current evidence of biliary cholestasis
Estimated glomerular filtration rate of <30 milliliters/minute/1.73 meter squared and/or are on dialysis
Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C infection at screening
Lee JW, Griffin M, Kim JS, Lee Lee LW, Piatek C, Nishimura JI, Carrillo Infante C, Jain D, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Kulasekararaj AG; ALXN2040-PNH-301 Investigators. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. Lancet Haematol. 2023 Dec;10(12):e955-e965. doi: 10.1016/S2352-3026(23)00315-0.
Danicopan (ACH-4471) in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Phase 2, Open-label, Proof of Concept Study of the Oral, Small Molecule Factor D Inhibitor
The study consists of 2 treatment periods and a long-term extension period.
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Danicopan-Danicopan
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
Periods
Title
Milestones
Reasons Not Completed
Treatment Period 1 (TP1)
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Aug 8, 2022
Jul 21, 2023
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Double
Masking Description
Not provided
Who Masked
ParticipantInvestigator
Placebo
Drug
Oral tablet
Placebo + C5 Inhibitor
C5 Inhibitor
Drug
Participants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule.
Danicopan + C5 Inhibitor
Placebo + C5 Inhibitor
Week 12
Percentage of Participants With Transfusion Avoidance Through Week 12
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.
Week 12
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12
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 and better health-related quality of life. LS mean and SE were produced using MMRM.
Baseline, Week 12
Change From Baseline in Absolute Reticulocyte Count at Week 12
LS mean and SE were produced using MMRM.
Baseline, Week 12
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
24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment
Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment
Percentage of Participants With Transfusion Avoidance Through Week 24
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 24. Participants who discontinued study treatment early before Week 24 were considered as not achieving transfusion avoidance.
24 weeks
Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
LS mean and SE were produced using analysis of covariance (ANCOVA).
12 weeks prior to initiation of treatment to 12 weeks post initiation of treatment
Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
LS mean and SE were produced using ANCOVA.
12 weeks prior to initiation of treatment to post 12 weeks of treatment (24 weeks)
Change From Baseline FACIT Fatigue Scores at Week 24
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 and better health-related quality of life. LS mean and SE were produced using MMRM.
Baseline, Week 24
Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan
The criterion was defined as Hgb stabilization avoidance of a >1 g/dL (>10 g/L) decrease in Hgb level at Week 24 from Week 12. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb stabilization regardless of the actual value observed at Week 24.
Week 12 to Week 24
Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24
The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 24 and remaining transfusion free during the 12-Week TP2. Participants who withdrew from the study early during the 12-Week TP2 or had missing Hgb value at Week 24 were considered as not achieving the criterion.
Week 24
Change From Baseline in Total and Direct Bilirubin at Week 12
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Baseline, Week 12
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12
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 non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Baseline, Week 12
Change From Baseline in Complement Component 3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Baseline, Week 12
Change From Baseline in Lactate Dehydrogenase at Week 12
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.
Baseline, Week 12
Percentage of Participants With Hgb Normalization at Week 12
Hgb normalization was defined as Hgb value above lower limit of normal (LLN) reference range. For male, the LLN was 125 g/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.
Week 12
Percentage of Participants With Hgb Normalization at Week 24
Hgb normalization was defined as Hgb value above LLN reference range. For male, the LLN was 125 g/L, for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb normalization regardless of actual value observed at Week 24.
Week 24
Weston
Florida
33331
United States
Research Site
Chicago
Illinois
60612
United States
Research Site
Kalamazoo
Michigan
49007
United States
Research Site
New York
New York
10065
United States
Research Site
Dallas
Texas
75390
United States
Research Site
Milwaukee
Wisconsin
53212
United States
Research Site
Belém
66053-000
Brazil
Research Site
Curitiba
80810-050
Brazil
Research Site
Goiânia
74605-020
Brazil
Research Site
Porto Alegre
90110-270
Brazil
Research Site
Rio de de Janeiro
20211030
Brazil
Research Site
Toronto
Ontario
M5G 2C4
Canada
Research Site
Brno
625 00
Czechia
Research Site
Lille
59037
France
Research Site
Paris
75010
France
Research Site
Pessac
33604
France
Research Site
Pierre-Bénite
69495
France
Research Site
Ulm
89081
Germany
Research Site
Athens
11527
Greece
Research Site
Thessaloniki
57010
Greece
Research Site
Haifa
31048
Israel
Research Site
Jerusalem
91120
Israel
Research Site
Avellino
83100
Italy
Research Site
Bassano del Grappa
36061
Italy
Research Site
Florence
50134
Italy
Research Site
Milan
20122
Italy
Research Site
Reggio Calabria
89131
Italy
Research Site
Roma
00161
Italy
Research Site
Bunkyō City
113 8603
Japan
Research Site
Fukuoka
812-8582
Japan
Research Site
Kashiwa-shi
277-8567
Japan
Research Site
Kyoto
605-0981
Japan
Research Site
Nagakute-shi
480-1195
Japan
Research Site
Ogaki-shi
503-8502
Japan
Research Site
Osaka
530-8480
Japan
Research Site
Sayama
589-8511
Japan
Research Site
Shibuya-ku
150-8935
Japan
Research Site
Tanabe-shi
646-8588
Japan
Research Site
Toyoake-shi
470-1192
Japan
Research Site
Tsukuba
305-8576
Japan
Research Site
Kota Kinabalu
88586
Malaysia
Research Site
Kuching
93586
Malaysia
Research Site
Miri
98000
Malaysia
Research Site
Maastricht
6229 HX
Netherlands
Research Site
Gdansk
80-214
Poland
Research Site
Daejeon
35015
South Korea
Research Site
Seoul
03722
South Korea
Research Site
Seoul
06351
South Korea
Research Site
Seoul
06591
South Korea
Research Site
Suwon
16247
South Korea
Research Site
Barcelona
08036
Spain
Research Site
Barcelona
08916
Spain
Research Site
Las Palmas de Gran Canaria
35020
Spain
Research Site
Madrid
28040
Spain
Research Site
Majadahonda
28222
Spain
Research Site
Seville
41013
Spain
Research Site
Taipei
100
Taiwan
Research Site
Bangkok
10330
Thailand
Research Site
Airdrie
ML6 0JS
United Kingdom
Research Site
Leeds
BD7 1DP
United Kingdom
Research Site
London
SE5 9NU
United Kingdom
Derived
Kulasekararaj A, Griffin M, Piatek C, Shammo J, Nishimura JI, Patriquin C, Schrezenmeier H, Barcellini W, Panse J, Gaya A, Patel Y, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Lee JW. Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH. Blood. 2025 Feb 20;145(8):811-822. doi: 10.1182/blood.2024026299.
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
FG00057 subjects
FG00129 subjects
Received at Least 1 Dose of Study Drug
Full Analysis Set/Safety Set
FG00057 subjects
FG00129 subjects
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.
FG00042 subjects
FG00121 subjects
COMPLETED
Completed TP1
FG00055 subjects
FG00127 subjects
NOT COMPLETED
FG0002 subjects
FG0012 subjects
Type
Comment
Reasons
Adverse Event
FG0002 subjects
FG0011 subjects
Withdrawal by Subject
FG0000 subjects
FG0011 subjects
Treatment Period 2 (TP2)
Type
Comment
Milestone Data
STARTED
FG00055 subjects
FG00127 subjects
Received at Least 1 Dose of Study Drug
Full Analysis Set/Safety Set
FG00055 subjects
FG00127 subjects
COMPLETED
FG00054 subjects
FG00126 subjects
NOT COMPLETED
FG0001 subjects
FG0011 subjects
Type
Comment
Reasons
Adverse Event
FG0001 subjects
FG0011 subjects
Long-Term Extension (LTE)
Type
Comment
Milestone Data
STARTED
FG00054 subjects
FG00126 subjects
Received at Least 1 Dose of Study Drug
Safety Set
FG00054 subjects
FG00126 subjects
COMPLETED
FG00046 subjects
FG00124 subjects
NOT COMPLETED
FG0008 subjects
FG0012 subjects
Type
Comment
Reasons
Withdrawal by Subject
FG0003 subjects
FG0010 subjects
Physician Decision
FG0003 subjects
FG001
Full Analysis Set: all enrolled participants who were randomized to either the danicopan or placebo treatment group.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Danicopan-Danicopan
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the long-term extension (LTE) for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
BG001
Placebo-Danicopan
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
BG002
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00057
BG00129
BG00286
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00052.8± 17.00
BG00152.9± 14.34
BG00252.8± 16.07
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00034
BG00120
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0006
BG0011
BG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0001
BG0010
BG002
Hemoglobin (Hgb)
g/L = grams/liter
Mean
Standard Deviation
g/L
Title
Denominators
Categories
Title
Measurements
BG00076.7± 9.47
BG00178.9± 10.11
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Change From Baseline in Hgb at Week 12
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.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Posted
Least Squares Mean
Standard Error
g/L
Baseline, Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Participants
OG00057
OG00128
Title
Denominators
Categories
Interim Efficacy Analysis
ParticipantsOG00042
ParticipantsOG00121
Title
Measurements
OG00029.40± 2.107
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Interim Efficacy Analysis
Re-randomization Test
0.0007
Superiority
OG000
OG001
Full Analysis
Re-randomization Test
Secondary
Percentage of Participants With Hgb Increase of ≥2 Grams/Deciliter (g/dL) (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12
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.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.
Posted
Number
95% Confidence Interval
percentage of participants
Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Secondary
Percentage of Participants With Transfusion Avoidance Through Week 12
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.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.
Posted
Number
95% Confidence Interval
percentage of participants
Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Participants
Secondary
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12
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 and better health-related quality of life. LS mean and SE were produced using MMRM.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Posted
Least Squares Mean
Standard Error
units on a scale
Baseline, Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Secondary
Change From Baseline in Absolute Reticulocyte Count at Week 12
LS mean and SE were produced using MMRM.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure.
Posted
Least Squares Mean
Standard Error
10^12 cells/L
Baseline, Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Participants
Secondary
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
Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.
Posted
Mean
Standard Deviation
RBC units
24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment
ID
Title
Description
OG000
Danicopan-Danicopan (TP2)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
Units
Counts
Participants
OG000
Secondary
Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.
Posted
Mean
Standard Deviation
transfusion instances
24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment
ID
Title
Description
OG000
Danicopan-Danicopan (TP2)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
Units
Counts
Participants
OG000
Secondary
Percentage of Participants With Transfusion Avoidance Through Week 24
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 24. Participants who discontinued study treatment early before Week 24 were considered as not achieving transfusion avoidance.
Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.
Posted
Number
95% Confidence Interval
percentage of participants
24 weeks
ID
Title
Description
OG000
Danicopan-Danicopan (TP2)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
Units
Counts
Participants
OG000
Secondary
Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
LS mean and SE were produced using analysis of covariance (ANCOVA).
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.
Posted
Least Squares Mean
Standard Error
RBC units
12 weeks prior to initiation of treatment to 12 weeks post initiation of treatment
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Participants
Secondary
Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
LS mean and SE were produced using ANCOVA.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.
Posted
Least Squares Mean
Standard Error
transfusion instances
12 weeks prior to initiation of treatment to post 12 weeks of treatment (24 weeks)
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Participants
Secondary
Change From Baseline FACIT Fatigue Scores at Week 24
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 and better health-related quality of life. LS mean and SE were produced using MMRM.
Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who were evaluable for this outcome measure.
Posted
Least Squares Mean
Standard Error
units on a scale
Baseline, Week 24
ID
Title
Description
OG000
Danicopan-Danicopan
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
OG001
Placebo-Danicopan
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
Secondary
Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan
The criterion was defined as Hgb stabilization avoidance of a >1 g/dL (>10 g/L) decrease in Hgb level at Week 24 from Week 12. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb stabilization regardless of the actual value observed at Week 24.
Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.
Posted
Number
95% Confidence Interval
percentage of participants
Week 12 to Week 24
ID
Title
Description
OG000
Danicopan-Danicopan
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
Units
Counts
Participants
OG000
Secondary
Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24
The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 24 and remaining transfusion free during the 12-Week TP2. Participants who withdrew from the study early during the 12-Week TP2 or had missing Hgb value at Week 24 were considered as not achieving the criterion.
Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.
Posted
Number
95% Confidence Interval
percentage of participants
Week 24
ID
Title
Description
OG000
Danicopan-Danicopan
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
Units
Counts
Participants
OG000
Secondary
Change From Baseline in Total and Direct Bilirubin at Week 12
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.
Posted
Least Squares Mean
Standard Error
micromoles/L
Baseline, Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Participants
Secondary
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12
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 non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure.
Posted
Least Squares Mean
Standard Error
percentage of the total cell population
Baseline, Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Secondary
Change From Baseline in Complement Component 3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12
Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure.
Posted
Least Squares Mean
Standard Error
percentage of the total cell population
Baseline, Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Secondary
Change From Baseline in Lactate Dehydrogenase at Week 12
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.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure.
Posted
Least Squares Mean
Standard Error
units/L
Baseline, Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Participants
Secondary
Percentage of Participants With Hgb Normalization at Week 12
Hgb normalization was defined as Hgb value above lower limit of normal (LLN) reference range. For male, the LLN was 125 g/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.
Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.
Posted
Number
95% Confidence Interval
percentage of participants
Week 12
ID
Title
Description
OG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
OG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Units
Counts
Participants
Secondary
Percentage of Participants With Hgb Normalization at Week 24
Hgb normalization was defined as Hgb value above LLN reference range. For male, the LLN was 125 g/L, for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb normalization regardless of actual value observed at Week 24.
Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.
Posted
Number
95% Confidence Interval
percentage of participants
Week 24
ID
Title
Description
OG000
Danicopan-Danicopan
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
Units
Counts
Participants
OG000
Time Frame
Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years)
Description
The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Danicopan (TP1)
Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP.
0
57
3
57
43
57
EG001
Placebo (TP1)
Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
0
29
2
29
18
29
EG002
Danicopan-Danicopan (TP2)
Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
0
55
3
55
40
55
EG003
Placebo-Danicopan (TP2)
At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.
0
27
6
27
18
27
EG004
Danicopan-Danicopan (LTE)
After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
0
54
7
54
48
54
EG005
Placebo-Danicopan (LTE)
After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
1
26
6
26
24
26
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0011 events1 affected29 at risk
EG0020 events0 affected55 at risk
EG0030 events0 affected27 at risk
EG0041 events1 affected54 at risk
EG0050 events0 affected26 at risk
Abdominal pain
Gastrointestinal disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0011 events1 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Cholecystitis
Hepatobiliary disorders
MedDRA 26.1
Systematic Assessment
EG0001 events1 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
COVID-19
Infections and infestations
MedDRA 26.1
Systematic Assessment
EG0001 events1 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Pancreatitis
Gastrointestinal disorders
MedDRA 26.1
Systematic Assessment
EG0001 events1 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Blood bilirubin increased
Investigations
MedDRA 26.1
Systematic Assessment
EG0001 events1 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Headache
Nervous system disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0012 events1 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Haemolysis
Blood and lymphatic system disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Haemorrhagic diathesis
Blood and lymphatic system disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Pericardial effusion
Cardiac disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Vertigo
Ear and labyrinth disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Dieulafoy's vascular malformation
Gastrointestinal disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0021 events1 affected55 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Non-cardiac chest pain
General disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Pyrexia
General disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0021 events1 affected55 at risk
EG003
Stent-graft endoleak
General disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Cholelithiasis
Hepatobiliary disorders
MedDRA 26.1
Systematic Assessment
EG0001 events1 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
COVID-19 pneumonia
Infections and infestations
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0021 events1 affected55 at risk
EG003
Cystitis
Infections and infestations
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Neutropenic sepsis
Infections and infestations
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Pneumonia
Infections and infestations
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Staphylococcal sepsis
Infections and infestations
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0021 events1 affected55 at risk
EG003
Femur fracture
Injury, poisoning and procedural complications
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Body temperature increased
Investigations
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Haemoglobin decreased
Investigations
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Platelet count decreased
Investigations
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 26.1
Systematic Assessment
EG0000 events0 affected57 at risk
EG0010 events0 affected29 at risk
EG0020 events0 affected55 at risk
EG003
Invasive ductal breast carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)