Trial Outcomes & Findings for A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG) (NCT NCT03759366)

Last Updated: 2024-12-11

Results Overview

The QMG scoring system consists of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item). Each item is graded from 0 to 3, (0 = none, 1 = mild, 2 = moderate, and 3 = severe). The range of total QMG score is 0 to 39, with higher scores indicating more severe disease.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

12 participants

Primary outcome timeframe

Baseline, Week 26

Results posted on

2024-12-11

Participant Flow

The study included a Primary Evaluation Treatment Period of 26 weeks, an Extension Period of up to an additional 208 weeks, and a Follow-up Period of 8 weeks.

All participants were offered participation in the Extension Period of the study.

Participant milestones

Participant milestones
Measure	Eculizumab Participants received eculizumab by intravenous (IV) infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 milligrams (mg), based on the participant's current body weight.
Primary Evaluation Period (26 Weeks) STARTED	11
Primary Evaluation Period (26 Weeks) Received at Least 1 Dose of Study Drug	11
Primary Evaluation Period (26 Weeks) COMPLETED	10
Primary Evaluation Period (26 Weeks) NOT COMPLETED	1
Extension Period (Up to 208 Weeks) STARTED	10
Extension Period (Up to 208 Weeks) Received at Least 1 Dose of Study Drug	10
Extension Period (Up to 208 Weeks) COMPLETED	2
Extension Period (Up to 208 Weeks) NOT COMPLETED	8

Reasons for withdrawal

Reasons for withdrawal
Measure	Eculizumab Participants received eculizumab by intravenous (IV) infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 milligrams (mg), based on the participant's current body weight.
Primary Evaluation Period (26 Weeks) Physician Decision	1
Extension Period (Up to 208 Weeks) Physician Decision	1
Extension Period (Up to 208 Weeks) Lost to Follow-up	1
Extension Period (Up to 208 Weeks) Participant transitioned to Ultomiris	2
Extension Period (Up to 208 Weeks) Participant transitioned to Soliris	1
Extension Period (Up to 208 Weeks) Participant transferred to another study	1
Extension Period (Up to 208 Weeks) Withdrawal by Subject	2

Baseline Characteristics

A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eculizumab n=11 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Age, Continuous	14.8 years STANDARD_DEVIATION 1.78 • n=99 Participants
Sex: Female, Male Female	9 Participants n=99 Participants
Sex: Female, Male Male	2 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	3 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	5 Participants n=99 Participants
Race (NIH/OMB) White	2 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=99 Participants
Quantitative Myasthenia Gravis (QMG) Total Score	16.7 units on a scale STANDARD_DEVIATION 5.64 • n=99 Participants

PRIMARY outcome

Timeframe: Baseline, Week 26

Population: Modified Full Analysis Set: all participants 12 to \<18 years of age who received at least 1 dose of eculizumab. Here, 'Overall Number of Participants Analyzed' signifies those participants who were evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Change From Baseline in the QMG Total Score at Week 26 Regardless of Rescue Treatment	-6.1 units on a scale Standard Deviation 4.56 • Interval 4.56 to

SECONDARY outcome

Timeframe: Baseline, Week 26

The MG-ADL is an 8-point questionnaire that focuses on relevant symptoms and functional performance of activities of daily living in participants with myasthenia gravis (MG). The 8 items of the MG-ADL are derived from symptom-based components of the original 13-item QMG to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response is graded from 0 (normal) to 3 (most severe). The range of total MG-ADL score is 0 to 24, with higher scores indicating more severe disease.

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score at Week 26 Regardless of Rescue Treatment	-2.5 units on a scale Standard Deviation 1.78 • Interval 1.78 to

SECONDARY outcome

Timeframe: Week 26

The MG-ADL is an 8-point questionnaire that focuses on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MG-ADL are derived from symptom-based components of the original 13-item QMG to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response is graded from 0 (normal) to 3 (most severe). The range of total MG-ADL score is 0 to 24, with higher scores indicating more severe disease.

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Percentage of Participants With ≥3-Point Reduction in the MG-ADL Total Score With No Rescue Treatment	50.0 percentage of participants 95% Confidence Interval 18.7 • Interval 18.7 to 81.3

SECONDARY outcome

Timeframe: Week 26

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Percentage of Participants With ≥3-Point Reduction in the MG-ADL Total Score Regardless of Rescue Treatment	50.0 percentage of participants 95% Confidence Interval 18.7 • Interval 18.7 to 81.3

SECONDARY outcome

Timeframe: Week 26

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Percentage of Participants With ≥5-Point Reduction in the QMG Total Score With No Rescue Treatment	70.0 percentage of participants 95% Confidence Interval 34.8 • Interval 34.8 to 93.3

SECONDARY outcome

Timeframe: Week 26

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Percentage of Participants With ≥5-Point Reduction in the QMG Total Score Regardless of Rescue Treatment	70.0 percentage of participants 95% Confidence Interval 34.8 • Interval 34.8 to 93.3

SECONDARY outcome

Timeframe: Baseline, Week 26

The MGC is a validated assessment tool for measuring clinical status of participants with MG. The MGC assesses 10 important functional areas most frequently affected by MG: ocular (2 items), facial (1 item), bulbar (3 items), respiratory (1 item), axial (1 item), and gross motor (2 items). The scales are weighted for clinical significance that incorporates patient-reported outcomes. The MGC total score ranges from 0 to 50, with lower scores indicating less functional impairment and higher scores indicating greater functional impairment.

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Change From Baseline in the Myasthenia Gravis Composite (MGC) Scale Total Score at Week 26 Regardless of Rescue Treatment	-9.4 units on a scale Standard Deviation 5.91 • Interval 5.91 to

SECONDARY outcome

Timeframe: Baseline, Week 26

The EQ-5D-Y is a reliable and validated survey of health status in 5 areas: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression, each of which is completed by the participant for participants ≥12 years of age (at time of assessment) and completed by the participant's caregiver or with caregiver assistance for participant \<12 years of age. Each area has 3 levels: Level 1 (no problems), Level 2 (some problems), and Level 3 (extreme problems). The EQ visual analogue scale (VAS) records the participant's self-rated health on a vertical, 20-centimeter VAS where the endpoints are labelled 'Best imaginable health state, marked as 100' and 'Worst imaginable health state, marked as 0'.

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Change From Baseline in the European Quality of Life 5-Dimension Youth Version (EQ-5D-Y) Visual Analogue Scale (VAS) Score at Week 26 Regardless of Rescue Treatment	23.5 units on a scale Standard Deviation 23.34 • Interval 23.34 to

SECONDARY outcome

Timeframe: Baseline, Week 26

The Neuro-QoL Pediatric Fatigue questionnaire is a reliable and validated brief 11-item survey of fatigue, completed by the participant for participants ≥12 years of age (at time of assessment) and completed by the participant's caregiver or with caregiver assistance for participants \<12 years of age. Each item was scored on a scale of 1 to 5 (1=Not at all, 2=A little bit, 3=Somewhat, 4=Quite a bit, 5=Very much). Total score is the sum of each item's score and it ranges from 11 to 55. Higher scores indicate greater fatigue and greater impact of MG on activities.

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Change From Baseline in the Neurological Quality of Life-Fatigue Questionnaire (Neuro-QoL Pediatric Fatigue) Total Score at Week 26 Regardless of Rescue Treatment	-7.9 units on a scale Standard Deviation 7.37 • Interval 7.37 to

SECONDARY outcome

Timeframe: Week 26

Population: Modified Full Analysis Set: all participants 12 to \<18 years of age who received at least 1 dose of eculizumab. Here, 'Number Analyzed' and 'Overall Number of Participants Analyzed' signify those participants who were evaluable for this outcome measure.

The MG clinical state (improved, unchanged, and worse) was assessed using the MGFAPIS.

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Number of Participants in Each Category of the Myasthenia Gravis Foundation of America Post-Intervention Status (MGFAPIS) Regardless of Rescue Treatment at Week 26 Improved	10 Participants
Number of Participants in Each Category of the Myasthenia Gravis Foundation of America Post-Intervention Status (MGFAPIS) Regardless of Rescue Treatment at Week 26 Unchanged	0 Participants
Number of Participants in Each Category of the Myasthenia Gravis Foundation of America Post-Intervention Status (MGFAPIS) Regardless of Rescue Treatment at Week 26 Worse	0 Participants

SECONDARY outcome

Timeframe: Baseline up to Week 26

Population: Modified Full Analysis Set: all participants 12 to \<18 years of age who received at least 1 dose of eculizumab.

Rescue therapy (for example, high dose corticosteroid, plasma exchange, or intravenous immunoglobulin) was to be allowed when a participant experienced clinical deterioration. Clinical deterioration was defined as follows: Participants who experienced an MG crisis, which was defined as weakness due to MG that was severe enough to necessitate intubation or to delay extubation following surgery; or, Significant symptomatic worsening that required rescue medication in the opinion of the Investigator; or, Participants for whom the Investigator believed that the participants' health was in jeopardy if rescue therapy was not given.

Outcome measures

Outcome measures
Measure	Eculizumab n=11 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Percentage of Participants With Clinical Deteriorations, Myasthenic Crises, and Rescue Therapy Use Clinical Deterioration	9.1 percentage of participants
Percentage of Participants With Clinical Deteriorations, Myasthenic Crises, and Rescue Therapy Use MG Crisis	9.1 percentage of participants
Percentage of Participants With Clinical Deteriorations, Myasthenic Crises, and Rescue Therapy Use Requiring Rescue Therapy	9.1 percentage of participants

SECONDARY outcome

Timeframe: 24 hours postdose on Day 1; predose and 60 minutes postdose at Week 12; predose at Week 26

Population: Pharmacokinetic Analysis Set: all participants 12 to \<18 years of age who received at least 1 dose of eculizumab and who had PK data assessments during the study. Here, 'Number Analyzed' signifies those participants who were evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Eculizumab n=11 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Pharmacokinetics (PK): Serum Concentration Of Eculizumab Day 1, 24 hours postdose	359.6 micrograms (μg)/milliliter (mL) Standard Deviation 105.18
Pharmacokinetics (PK): Serum Concentration Of Eculizumab Week 12, Predose	382.8 micrograms (μg)/milliliter (mL) Standard Deviation 159.57
Pharmacokinetics (PK): Serum Concentration Of Eculizumab Week 12, 60 minutes postdose	910.5 micrograms (μg)/milliliter (mL) Standard Deviation 277.29
Pharmacokinetics (PK): Serum Concentration Of Eculizumab Week 26, Predose	433.9 micrograms (μg)/milliliter (mL) Standard Deviation 171.85

SECONDARY outcome

Timeframe: Baseline; 24 hours postdose on Day 1; predose and 60 minutes postdose at Week 12; predose at Week 26

Population: Pharmacodynamic Analysis Set: all participants 12 to \<18 years of age who received at least 1 dose of eculizumab and who had PD data assessments during the study. Here, 'Number Analyzed' signifies those participants who were evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Eculizumab n=11 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Pharmacodynamics (PD): Serum Free Complement Component 5 Concentrations Baseline	172.7 μg/mL Standard Deviation 34.52
Pharmacodynamics (PD): Serum Free Complement Component 5 Concentrations Day 1, 24 hours postdose	0.0 μg/mL Standard Deviation 0.01
Pharmacodynamics (PD): Serum Free Complement Component 5 Concentrations Week 12, Predose	0.0 μg/mL Standard Deviation 0.01
Pharmacodynamics (PD): Serum Free Complement Component 5 Concentrations Week 12, 60 minutes postdose	0.0 μg/mL Standard Deviation 0.01
Pharmacodynamics (PD): Serum Free Complement Component 5 Concentrations Week 26, Predose	0.0 μg/mL Standard Deviation 0.02

SECONDARY outcome

Timeframe: Baseline; 24 hours postdose on Day 1; predose and 60 minutes postdose at Week 12; predose at Week 26

Outcome measures

Outcome measures
Measure	Eculizumab n=10 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
PD: Percentage of Hemolysis (In Vitro Assay) Baseline	105.8 percentage of hemolysis Standard Deviation 14.15
PD: Percentage of Hemolysis (In Vitro Assay) Day 1, 24 hours postdose	1.1 percentage of hemolysis Standard Deviation 2.01
PD: Percentage of Hemolysis (In Vitro Assay) Week 12, Predose	1.8 percentage of hemolysis Standard Deviation 4.67
PD: Percentage of Hemolysis (In Vitro Assay) Week 12, 60 minutes postdose	0.2 percentage of hemolysis Standard Deviation 0.45
PD: Percentage of Hemolysis (In Vitro Assay) Week 26, Predose	0.5 percentage of hemolysis Standard Deviation 1.29

SECONDARY outcome

Timeframe: Baseline, Week 52

Outcome measures

Outcome measures
Measure	Eculizumab n=8 Participants Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
Change From Baseline in the QMG Total Score at Week 52 Regardless of Rescue Treatment	-4.9 units on a scale Standard Deviation 4.26 • Interval 4.26 to

Adverse Events

Eculizumab

Serious events: 4 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Eculizumab n=11 participants at risk Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
General disorders Pyrexia	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations Peritonsillar abscess	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Nervous system disorders Myasthenia gravis	9.1% 1/11 • Number of events 8 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Nervous system disorders Myasthenia gravis crisis	9.1% 1/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Psychiatric disorders Suicide attempt	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.

Other adverse events

Other adverse events
Measure	Eculizumab n=11 participants at risk Participants received eculizumab by IV infusion during the Primary Evaluation Treatment Period (26 weeks) and the Extension Period (up to 208 weeks). Dosing was initiated with a weekly weight-based induction regimen (Induction Phase) and, thereafter, participants were dosed every 2 weeks (Maintenance Phase). Eculizumab was administered at doses of 300, 600, 900, or 1200 mg, based on the participant's current body weight.
General disorders Fatigue	27.3% 3/11 • Number of events 5 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Infusion site extravasation	9.1% 1/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Injection site bruising	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Injection site pain	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Vaccination site pain	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations Nasopharyngitis	54.5% 6/11 • Number of events 10 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations Upper respiratory tract infection	27.3% 3/11 • Number of events 4 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations COVID-19	18.2% 2/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations Cellulitis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations Pharyngitis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations Viral infection	9.1% 1/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Musculoskeletal and connective tissue disorders Pain in extremity	27.3% 3/11 • Number of events 4 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Musculoskeletal and connective tissue disorders Costochondritis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Musculoskeletal and connective tissue disorders Muscle spasms	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Musculoskeletal and connective tissue disorders Muscle twitching	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Musculoskeletal and connective tissue disorders Musculoskeletal stiffness	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Musculoskeletal and connective tissue disorders Myalgia	18.2% 2/11 • Number of events 3 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Abdominal pain	27.3% 3/11 • Number of events 3 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Diarrhoea	18.2% 2/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Vomiting	18.2% 2/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Abdominal distension	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Abdominal pain upper	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Constipation	18.2% 2/11 • Number of events 3 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Flatulence	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Mouth ulceration	9.1% 1/11 • Number of events 21 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Nausea	9.1% 1/11 • Number of events 4 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Injury, poisoning and procedural complications Thermal burn	18.2% 2/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Injury, poisoning and procedural complications Arthropod bite	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Nervous system disorders Headache	36.4% 4/11 • Number of events 16 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Nervous system disorders Dizziness	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Nervous system disorders Tremor	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Metabolism and nutrition disorders Decreased appetite	18.2% 2/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Metabolism and nutrition disorders Ketosis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Metabolism and nutrition disorders Hypokalaemia	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Nasal congestion	18.2% 2/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Cough	9.1% 1/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	27.3% 3/11 • Number of events 4 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Sinus congestion	18.2% 2/11 • Number of events 4 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Acne	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Dry skin	9.1% 1/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Eczema	18.2% 2/11 • Number of events 8 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Pruritus	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Rash	18.2% 2/11 • Number of events 3 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Urticaria	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Blood and lymphatic system disorders Iron deficiency anaemia	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Blood and lymphatic system disorders Leukopenia	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Blood and lymphatic system disorders Lymphocytosis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Blood and lymphatic system disorders Monocytosis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Blood and lymphatic system disorders Neutropenia	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Cardiac disorders Palpitations	18.2% 2/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Investigations Glucose urine present	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Investigations Electrocardiogram PR prolongation	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Psychiatric disorders Behaviour disorder	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Psychiatric disorders Panic attack	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Ear and labyrinth disorders Ear pain	18.2% 2/11 • Number of events 3 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Eye disorders Eye pruritus	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Eye disorders Lacrimation increased	9.1% 1/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Eye disorders Ocular hyperaemia	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Product Issues Device malfunction	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Renal and urinary disorders Hypercalciuria	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Vascular disorders Poor venous access	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Pyrexia	27.3% 3/11 • Number of events 8 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Injury, poisoning and procedural complications Immunisation reaction	18.2% 2/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Ear and labyrinth disorders Cerumen impaction	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Eye disorders Visual impairment	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Abdominal discomfort	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Rectal haemorrhage	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Asthenia	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Chronic fatigue syndrome	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Gait disturbances	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Infusion site discharge	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
General disorders Infusion site swelling	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations Influenza	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Infections and infestations Viral upper respiratory tract infection	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Gastrointestinal disorders Stomatitis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Injury, poisoning and procedural complications Contusion	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Injury, poisoning and procedural complications Fall	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Injury, poisoning and procedural complications Head injury	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Investigations Alanine aminotransferase increased	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Investigations Aspartate aminotransferase increased	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Investigations Blood bicarbonate decreased	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Investigations Haemoglobin decreased	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Metabolism and nutrition disorders Dehydration	9.1% 1/11 • Number of events 2 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Metabolism and nutrition disorders Folate deficiency	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Metabolism and nutrition disorders Hyperuricaemia	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Metabolism and nutrition disorders Vitamin B12 deficiency	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Metabolism and nutrition disorders Vitamin D deficiency	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Musculoskeletal and connective tissue disorders Joint swelling	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Nervous system disorders Myasthenia gravis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Nervous system disorders Post-traumatic headache	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Psychiatric disorders Anxiety	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Dyspnoea	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Productive cough	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Respiratory disorder	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Respiratory, thoracic and mediastinal disorders Sputum discoloured	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Asteatosis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Hand dermatitis	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Skin and subcutaneous tissue disorders Rash pruritic	9.1% 1/11 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.
Reproductive system and breast disorders Dysmenorrhoea	11.1% 1/9 • Number of events 1 • From date of first dose (Day 1) through 8 weeks after last dose (4 years and 7 months). Safety analysis set included all participants who received at least 1 dose of eculizumab.

Additional Information

Alexion Europe SAS European Clinical Trial Information

Alexion Pharmaceuticals Inc.

Phone: 7 87148158

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place