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Trial Outcomes & Findings for A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G) (NCT NCT03369236)

NCT ID: NCT03369236

Last Updated: 2022-10-14

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 6 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

Baseline, Week 28

Results posted on

2022-10-14

Participant Flow

To enroll in the study, participants were required to have a biopsy confirmed diagnosis of C3 Glomerulopathy (C3G), with initial diagnosis made at least 3 months prior to dosing and significant proteinuria.

Participant milestones

Participant milestones
Measure
Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period)
Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and were to receive danicopan 200 mg TID.
Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period)
Placebo was administered TID during the 6-month treatment period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and were to receive danicopan 200 mg TID.
Double-blind Treatment Period
STARTED
6
7
Double-blind Treatment Period
Received at Least 1 Dose of Study Drug
6
7
Double-blind Treatment Period
COMPLETED
6
6
Double-blind Treatment Period
NOT COMPLETED
0
1
Open-label Extension Period
STARTED
6
6
Open-label Extension Period
Received at Least 1 Dose of Study Drug
6
6
Open-label Extension Period
COMPLETED
1
0
Open-label Extension Period
NOT COMPLETED
5
6

Reasons for withdrawal

Reasons for withdrawal
Measure
Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period)
Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and were to receive danicopan 200 mg TID.
Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period)
Placebo was administered TID during the 6-month treatment period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and were to receive danicopan 200 mg TID.
Double-blind Treatment Period
Accidental unblinding
0
1
Open-label Extension Period
Lack of Efficacy
2
1
Open-label Extension Period
Adverse Event
0
1
Open-label Extension Period
Sponsor decision to close study
3
4

Baseline Characteristics

A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Danicopan (Double-blind Treatment Period)
n=6 Participants
Danicopan was administered at a starting dose of 100 mg 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=7 Participants
Placebo was administered TID during the 6-month treatment period.
Total
n=13 Participants
Total of all reporting groups
Age, Continuous
25.5 years
STANDARD_DEVIATION 10.54 • n=99 Participants
24.9 years
STANDARD_DEVIATION 4.85 • n=107 Participants
25.2 years
STANDARD_DEVIATION 7.63 • n=206 Participants
Sex: Female, Male
Female
2 Participants
n=99 Participants
2 Participants
n=107 Participants
4 Participants
n=206 Participants
Sex: Female, Male
Male
4 Participants
n=99 Participants
5 Participants
n=107 Participants
9 Participants
n=206 Participants
Race/Ethnicity, Customized
Asian
0 Participants
n=99 Participants
2 Participants
n=107 Participants
2 Participants
n=206 Participants
Race/Ethnicity, Customized
White
5 Participants
n=99 Participants
4 Participants
n=107 Participants
9 Participants
n=206 Participants
Race/Ethnicity, Customized
Other
1 Participants
n=99 Participants
1 Participants
n=107 Participants
2 Participants
n=206 Participants
Race/Ethnicity, Customized
Hispanic or Latino
3 Participants
n=99 Participants
1 Participants
n=107 Participants
4 Participants
n=206 Participants
Race/Ethnicity, Customized
Not Hispanic or Latino
3 Participants
n=99 Participants
6 Participants
n=107 Participants
9 Participants
n=206 Participants
Region of Enrollment
United States
6 participants
n=99 Participants
6 participants
n=107 Participants
12 participants
n=206 Participants
Region of Enrollment
United Kingdom
0 participants
n=99 Participants
1 participants
n=107 Participants
1 participants
n=206 Participants

PRIMARY outcome

Timeframe: Baseline, Week 28

Population: 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 6 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.

Outcome measures

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=6 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=6 Participants
Placebo was administered TID during the 6-month treatment period.
Total
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Change From Baseline In Composite Biopsy Score At Week 28
Baseline
11.7 score on a scale
Standard Deviation 4.23
9.3 score on a scale
Standard Deviation 3.50
—
Change From Baseline In Composite Biopsy Score At Week 28
Week 28
9.2 score on a scale
Standard Deviation 4.87
10.7 score on a scale
Standard Deviation 3.39
—
Change From Baseline In Composite Biopsy Score At Week 28
Change from Baseline
-2.0 score on a scale
Standard Deviation 1.87
1.3 score on a scale
Standard Deviation 2.88
—

PRIMARY outcome

Timeframe: Week 28

Population: 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

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=4 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=5 Participants
Placebo was administered TID during the 6-month treatment period.
Total
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Participants With Reduction In Proteinuria At Week 28
0 participants
1 participants
—

SECONDARY outcome

Timeframe: Baseline, Week 28

Population: 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 Week 28.

Outcome measures

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=6 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=7 Participants
Placebo was administered TID during the 6-month treatment period.
Total
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Change From Baseline In Proteinuria At Week 28
Baseline
6137.67 mg/day
Standard Deviation 2904.359
4274.47 mg/day
Standard Deviation 2992.819
—
Change From Baseline In Proteinuria At Week 28
Week 28
5301.75 mg/day
Standard Deviation 2984.445
5186.80 mg/day
Standard Deviation 4069.279
—
Change From Baseline In Proteinuria At Week 28
Change from Baseline
302.00 mg/day
Standard Deviation 764.211
182.20 mg/day
Standard Deviation 994.558
—

SECONDARY outcome

Timeframe: Baseline, Week 28

Population: 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 Week 28.

Outcome measures

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=4 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=5 Participants
Placebo was administered TID during the 6-month treatment period.
Total
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Percent Change From Baseline In Proteinuria At Week 28
12.5 percent change
Standard Deviation 31.11
-10.4 percent change
Standard Deviation 31.35
—

SECONDARY outcome

Timeframe: 6 months

Population: 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 6-month blinded treatment period, with eGFR as the dependent variable and time as the independent variable.

Outcome measures

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=6 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=7 Participants
Placebo was administered TID during the 6-month treatment period.
Total
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To 6 Months
-2.26840 mL/min/1.73 m^2 per month
Standard Deviation 1.790814
-1.45421 mL/min/1.73 m^2 per month
Standard Deviation 2.228395
—

SECONDARY outcome

Timeframe: 12 months

Population: 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 during the open-label extension period with eGFR as the dependent variable and time as the independent variable.

Outcome measures

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=5 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=4 Participants
Placebo was administered TID during the 6-month treatment period.
Total
n=9 Participants
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Slope Of Estimated Glomerular Filtration Rate (eGFR) After Open-label Danicopan Treatment
-1.02556 mL/min/1.73 m^2 per month
Standard Deviation 1.389128
-1.84935 mL/min/1.73 m^2 per month
Standard Deviation 3.506810
-1.39169 mL/min/1.73 m^2 per month
Standard Deviation 2.401039

SECONDARY outcome

Timeframe: Baseline, Week 28

Population: 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 Week 28 is presented.

Outcome measures

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=6 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=7 Participants
Placebo was administered TID during the 6-month treatment period.
Total
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Change From Baseline In eGFR At Week 28
Baseline
79.890 mL/min/1.73 m^2
Standard Deviation 44.4571
68.381 mL/min/1.73 m^2
Standard Deviation 36.7249
—
Change From Baseline In eGFR At Week 28
Week 28
67.543 mL/min/1.73 m^2
Standard Deviation 47.8062
50.874 mL/min/1.73 m^2
Standard Deviation 15.0517
—
Change From Baseline In eGFR At Week 28
Change from Baseline
-12.347 mL/min/1.73 m^2
Standard Deviation 10.8063
-8.700 mL/min/1.73 m^2
Standard Deviation 16.0990
—

SECONDARY outcome

Timeframe: Baseline, Week 28

Population: 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 ≥ 20% increase from baseline in eGFR.

Outcome measures

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=6 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=5 Participants
Placebo was administered TID during the 6-month treatment period.
Total
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Participants With Significant Improvement In eGFR Relative To Baseline At Week 28
0 participants
0 participants
—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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 ≥ 20% increase from baseline in eGFR.

Outcome measures

Outcome measures
Measure
Danicopan (Double-blind Treatment Period)
n=5 Participants
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=5 Participants
Placebo was administered TID during the 6-month treatment period.
Total
All participants who received danicopan or placebo during the 6-month treatment period followed by danicopan 200 mg TID in the extension period.
Participants With Significant Improvement In eGFR Relative To Baseline At Week 52
0 participants
0 participants
—

Adverse Events

Danicopan (Double-blind Treatment Period)

Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths

Placebo (Double-blind Treatment Period)

Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths

Danicopan (Open-label Extension Period)

Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Danicopan (Double-blind Treatment Period)
n=6 participants at risk
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=7 participants at risk
Placebo was administered TID during the 6-month treatment period.
Danicopan (Open-label Extension Period)
n=12 participants at risk
All participants who received danicopan or placebo in the treatment period were to receive danicopan 200 mg TID during the open-label extension period.
Renal and urinary disorders
Acute kidney injury
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.

Other adverse events

Other adverse events
Measure
Danicopan (Double-blind Treatment Period)
n=6 participants at risk
Danicopan was administered at a starting dose of 100 mg TID for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.
Placebo (Double-blind Treatment Period)
n=7 participants at risk
Placebo was administered TID during the 6-month treatment period.
Danicopan (Open-label Extension Period)
n=12 participants at risk
All participants who received danicopan or placebo in the treatment period were to receive danicopan 200 mg TID during the open-label extension period.
Injury, poisoning and procedural complications
Skin laceration
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Injury, poisoning and procedural complications
Joint injury
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Vascular disorders
Haematoma
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Vascular disorders
Orthostatic hypotension
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Vascular disorders
Pallor
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Social circumstances
Pregnancy of partner
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Respiratory, thoracic and mediastinal disorders
Paranasal sinus discomfort
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
General disorders
Pyrexia
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
General disorders
Feeling cold
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
General disorders
Oedema peripheral
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
General disorders
Pain
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
General disorders
Face oedema
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Psychiatric disorders
Nightmare
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Psychiatric disorders
Anxiety
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Psychiatric disorders
Insomnia
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Reproductive system and breast disorders
Perineal pain
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Injury, poisoning and procedural complications
Contusion
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
28.6%
2/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Injury, poisoning and procedural complications
Hand fracture
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Investigations
Blood creatinine increased
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Respiratory, thoracic and mediastinal disorders
Nasal congestion
33.3%
2/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
33.3%
2/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Respiratory, thoracic and mediastinal disorders
Cough
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Respiratory, thoracic and mediastinal disorders
Epistaxis
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Respiratory, thoracic and mediastinal disorders
Lower respiratory tract congestion
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Nervous system disorders
Headache
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Nervous system disorders
Dizziness
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Nervous system disorders
Dizziness postural
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Nervous system disorders
Lethargy
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Nervous system disorders
Memory impairment
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Nervous system disorders
Hypoaesthesia
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Nervous system disorders
Migraine
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Eye disorders
Eye pain
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Eye disorders
Vision blurred
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Gastrointestinal disorders
Nausea
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
28.6%
2/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Gastrointestinal disorders
Vomiting
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
28.6%
2/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Gastrointestinal disorders
Diarrhoea
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
16.7%
2/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Renal and urinary disorders
Acute kidney injury
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Skin and subcutaneous tissue disorders
Pruritus
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Skin and subcutaneous tissue disorders
Macule
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Arthralgia
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Joint effusion
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Muscle spasms
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Muscular weakness
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Arthropathy
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Neck pain
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Musculoskeletal and connective tissue disorders
Pain in extremity
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Endocrine disorders
Hypothyroidism
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Metabolism and nutrition disorders
Gout
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Infections and infestations
Viral upper respiratory tract infection
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Infections and infestations
Gastroenteritis
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Infections and infestations
Pharyngitis
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
14.3%
1/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Infections and infestations
Nasopharyngitis
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
16.7%
2/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Infections and infestations
Upper respiratory tract infection
0.00%
0/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
8.3%
1/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
Vascular disorders
Dizziness
16.7%
1/6 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/7 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.
0.00%
0/12 • Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.

Additional Information

Alexion Pharmaceuticals Inc.

Alexion Pharmaceuticals Inc.

Phone: 1 855-752-2356

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place