Trial Outcomes & Findings for A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471 (NCT NCT03459443)
NCT ID: NCT03459443
Last Updated: 2023-08-21
Results Overview
The composite biopsy score was based on a score incorporating changes in the activity index, glomerular C3c staining, and glomerular macrophage infiltration at the end of the initial 12 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
22 participants
Primary outcome timeframe
Baseline, end of initial 12-Month Treatment Period
Results posted on
2023-08-21
Participant Flow
To enroll in the study, participants were required to have a biopsy-confirmed diagnosis of C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN).
Participant milestones
| Measure |
Danicopan
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Overall Study
STARTED
|
22
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
22
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
22
|
Reasons for withdrawal
| Measure |
Danicopan
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Overall Study
Lack of Efficacy
|
3
|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Sponsor's decision to close the study
|
18
|
Baseline Characteristics
A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471
Baseline characteristics by cohort
| Measure |
Danicopan
n=22 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Age, Continuous
|
24.3 years
STANDARD_DEVIATION 9.90 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
22 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
19 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, end of initial 12-Month Treatment PeriodPopulation: All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
The composite biopsy score was based on a score incorporating changes in the activity index, glomerular C3c staining, and glomerular macrophage infiltration at the end of the initial 12 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.
Outcome measures
| Measure |
Danicopan
n=16 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Change From Baseline In Composite Biopsy Score At End Of Initial 12-Month Treatment Period
Baseline
|
10.6 score on a scale
Standard Deviation 3.59
|
|
Change From Baseline In Composite Biopsy Score At End Of Initial 12-Month Treatment Period
End of 12-Month Initial Treatment Period
|
8.0 score on a scale
Standard Deviation 4.53
|
|
Change From Baseline In Composite Biopsy Score At End Of Initial 12-Month Treatment Period
Change from Baseline
|
-0.9 score on a scale
Standard Deviation 1.89
|
PRIMARY outcome
Timeframe: Baseline, end of initial 12-Month Treatment PeriodPopulation: All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
Proteinuria reduction was defined as ≥30% decrease from baseline based on 24-hour urine protein (mg/day).
Outcome measures
| Measure |
Danicopan
n=19 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Participants With Reduction In Proteinuria At End Of Initial 12-Month Treatment Period
|
8 participants
|
SECONDARY outcome
Timeframe: Baseline, end of initial 12-Month Treatment PeriodPopulation: All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
Proteinuria was assessed based on 24-hour urine collections at baseline and end of the initial 12-month Treatment Period.
Outcome measures
| Measure |
Danicopan
n=22 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
Baseline
|
4252.28 mg/day
Standard Deviation 2684.959
|
|
Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
End of Initial 12-Month Treatment Period
|
3512.63 mg/day
Standard Deviation 3335.765
|
|
Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
Mean Change from Baseline
|
-671.11 mg/day
Standard Deviation 2695.592
|
SECONDARY outcome
Timeframe: Baseline, end of initial 12-Month Treatment PeriodPopulation: All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
Proteinuria was assessed based on 24-hour urine collections at baseline and end of initial 12-month Treatment Period.
Outcome measures
| Measure |
Danicopan
n=19 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Percent Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
|
-17.1 percent change
Standard Deviation 53.17
|
SECONDARY outcome
Timeframe: End of initial 12-Month Treatment PeriodPopulation: All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
Slope of eGFR was estimated using a simple linear regression for each participant, including all data values from baseline until the end of the Initial 12-Month Treatment Period, with eGFR as the dependent variable and time as the independent variable.
Outcome measures
| Measure |
Danicopan
n=22 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To End Of Initial 12-Month Treatment Period
|
-1.24917 mL/min/1.73 m^2 per month
Standard Deviation 1.811457
|
SECONDARY outcome
Timeframe: Baseline, end of initial 12-Month Treatment PeriodPopulation: All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
Change from baseline in eGFR at end of initial 12-Month Treatment Period is presented.
Outcome measures
| Measure |
Danicopan
n=22 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period
Baseline
|
90.692 mL/min/1.73 m^2
Standard Deviation 35.4939
|
|
Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period
End of Initial 12-Month Treatment Period
|
81.412 mL/min/1.73 m^2
Standard Deviation 38.3320
|
|
Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period
Change from Baseline
|
-9.795 mL/min/1.73 m^2
Standard Deviation 14.3806
|
SECONDARY outcome
Timeframe: Baseline, end of initial 12-Month Treatment PeriodPopulation: All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
Significant improvement relative to baseline was defined as a ≥ 25% increase from baseline in eGFR.
Outcome measures
| Measure |
Danicopan
n=20 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Participants With Significant Improvement In eGFR Relative To Baseline At End Of Initial 12-Month Treatment Period
|
0 participants
|
SECONDARY outcome
Timeframe: End of initial 12-Month Treatment PeriodPopulation: All participants who received at least 1 dose of study drug and had analyzable data at the specified timepoints.
Participants were eligible for enrollment if inclusion criteria were met including having an eGFR \>=30 milliliters (mL)/minute (min)/1.73 square meter (m\^2) at the time of screening or at any time over the preceding 4 weeks. This Outcome Measure was registered in case there were participants who were enrolled and ended up not meeting the Eligibility Criteria and was intended to report data for change from baseline in eGFR for only the participants who met the eligibility criteria (that is, participants who did not meet the eligibility criteria would have been excluded from analysis for this Outcome Measure). Since all enrolled participants met the Eligibility Criteria, none of the participants were excluded from this analysis. Therefore, this data is the same data that is presented in Outcome Measure #6 "Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period". Change from baseline in eGFR at end of initial 12-Month Treatment Period is presented.
Outcome measures
| Measure |
Danicopan
n=22 Participants
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Change From Baseline in eGFR Over 12 Months of Treatment For Participants Meeting eGFR Inclusion Criteria
Baseline
|
90.692 mL/min/1.73 m^2
Standard Deviation 35.4939
|
|
Change From Baseline in eGFR Over 12 Months of Treatment For Participants Meeting eGFR Inclusion Criteria
End of Initial 12-Month Treatment Period
|
81.412 mL/min/1.73 m^2
Standard Deviation 38.3320
|
|
Change From Baseline in eGFR Over 12 Months of Treatment For Participants Meeting eGFR Inclusion Criteria
Change from Baseline
|
-9.795 mL/min/1.73 m^2
Standard Deviation 14.3806
|
SECONDARY outcome
Timeframe: End of initial 12-Month Treatment PeriodPopulation: Analysis was not performed, as data were not collected for this Outcome Measure.
Data for this Outcome Measure was to be collected where available. None of the sites collected data for this Outcome Measure.
Outcome measures
Outcome data not reported
Adverse Events
Danicopan
Serious events: 3 serious events
Other events: 22 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Danicopan
n=22 participants at risk
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Pyrexia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Rhinovirus infection
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Renal and urinary disorders
Renal impairment
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
Other adverse events
| Measure |
Danicopan
n=22 participants at risk
Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
22.7%
5/22 • Number of events 5 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Blood and lymphatic system disorders
Leukopenia
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Blood and lymphatic system disorders
Thrombocytosis
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Blood and lymphatic system disorders
Anaemia macrocytic
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Blood and lymphatic system disorders
Leukocytosis
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Blood and lymphatic system disorders
Neutrophilia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Cardiac disorders
Palpitations
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Cardiac disorders
Left ventricular failure
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Ear and labyrinth disorders
Ear pain
|
9.1%
2/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Ear and labyrinth disorders
Vertigo
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Endocrine disorders
Hyperparathyroidism secondary
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Endocrine disorders
Hypothyroidism
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Eye disorders
Periorbital oedema
|
9.1%
2/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Eye disorders
Conjunctivitis allergic
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Eye disorders
Vision blurred
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Vomiting
|
27.3%
6/22 • Number of events 7 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Diarrhoea
|
22.7%
5/22 • Number of events 7 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Nausea
|
18.2%
4/22 • Number of events 4 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Abdominal distension
|
9.1%
2/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Abdominal pain upper
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Abdominal pain
|
4.5%
1/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Constipation
|
4.5%
1/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Abdominal pain lower
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Bowel movement irregularity
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Dry mouth
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Dyspepsia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Haemorrhoids
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Hypertrophy of tongue papillae
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Paraesthesia oral
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Gastrointestinal disorders
Retroperitoneal haematoma
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Pyrexia
|
50.0%
11/22 • Number of events 16 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Oedema peripheral
|
36.4%
8/22 • Number of events 15 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Fatigue
|
22.7%
5/22 • Number of events 7 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Asthenia
|
18.2%
4/22 • Number of events 5 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Influenza like illness
|
4.5%
1/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Chills
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Facial pain
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Localised oedema
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Non-cardiac chest pain
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Oedema
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
General disorders
Pain
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Immune system disorders
Hypersensitivity
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Immune system disorders
Multiple allergies
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Gastroenteritis
|
27.3%
6/22 • Number of events 11 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Pharyngitis
|
27.3%
6/22 • Number of events 6 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Influenza
|
18.2%
4/22 • Number of events 4 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Nasopharyngitis
|
13.6%
3/22 • Number of events 9 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Upper respiratory tract infection
|
9.1%
2/22 • Number of events 4 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Urinary tract infection
|
9.1%
2/22 • Number of events 4 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Rhinitis
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Tonsillitis
|
4.5%
1/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Viral infection
|
4.5%
1/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Bacteriuria
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Conjunctivitis
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Corona virus infection
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Ear lobe infection
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Enterobiasis
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Herpes zoster
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Impetigo
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Respiratory tract infection viral
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Sinusitis
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Viral upper respiratory tract infection
|
13.6%
3/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Infections and infestations
Bronchitis
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Injury, poisoning and procedural complications
Contusion
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Injury, poisoning and procedural complications
Lip injury
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Injury, poisoning and procedural complications
Post procedural haematuria
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Blood creatine phosphokinase increased
|
22.7%
5/22 • Number of events 11 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Blood creatinine increased
|
9.1%
2/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Aspartate aminotransferase increased
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Alanine aminotransferase increased
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Blood potassium increased
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Blood uric acid increased
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Crystal urine present
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Electrocardiogram QT prolonged
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Haemoglobin decreased
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Lipids increased
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Transaminases increased
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Weight increased
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
22.7%
5/22 • Number of events 10 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Decreased appetite
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Dehydration
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Dyslipidaemia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Hypovitaminosis
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Metabolism and nutrition disorders
Iron deficiency
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
13.6%
3/22 • Number of events 5 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
13.6%
3/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
13.6%
3/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
9.1%
2/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
9.1%
2/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Joint effusion
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Muscle tightness
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Headache
|
27.3%
6/22 • Number of events 13 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Dizziness
|
13.6%
3/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Migraine
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Syncope
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Restless legs syndrome
|
4.5%
1/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Dizziness postural
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Migraine with aura
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Somnolence
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Nervous system disorders
Taste disorder
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Pregnancy, puerperium and perinatal conditions
Pregnancy
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Psychiatric disorders
Anxiety
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Psychiatric disorders
Abnormal dreams
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Renal and urinary disorders
Renal impairment
|
22.7%
5/22 • Number of events 6 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Renal and urinary disorders
Acute kidney injury
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Renal and urinary disorders
Nephrotic syndrome
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Reproductive system and breast disorders
Menstruation irregular
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Reproductive system and breast disorders
Oligomenorrhoea
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Reproductive system and breast disorders
Premature menopause
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
22.7%
5/22 • Number of events 8 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
18.2%
4/22 • Number of events 5 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.2%
4/22 • Number of events 4 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
13.6%
3/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
4.5%
1/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Upper-airway cough syndrome
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Rash
|
18.2%
4/22 • Number of events 4 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
9.1%
2/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Erythema
|
9.1%
2/22 • Number of events 2 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Acne
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Dermatitis atopic
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Surgical and medical procedures
Sinus operation
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Vascular disorders
Hypertension
|
9.1%
2/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Vascular disorders
Hypertensive crisis
|
4.5%
1/22 • Number of events 3 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Vascular disorders
Arteriovenous fistula
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Vascular disorders
Haematoma
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Vascular disorders
Hot flush
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Vascular disorders
Hypotension
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Vascular disorders
Pallor
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
|
Investigations
Blood potassium decreased
|
4.5%
1/22 • Number of events 1 • Day 1 (after dosing) up to 4 weeks after last dose of study drug (up to Week 108)
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Phone: 1.855.752.2356
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place