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

• ClinicalTrials.gov Identifier: NCT03759366
• Recruitment Status: Completed
• First Posted: November 30, 2018
• Results First Posted: August 25, 2022
• Last Update Posted: December 18, 2023
• Sponsor: Alexion Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Alexion Pharmaceuticals, Inc.

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Conditions: Myasthenia Gravis; Myasthenia Gravis, Juvenile Form; Myasthenia Gravis, Generalized
• Intervention: Drug: Eculizumab
• Enrollment: 11

Participant Flow

Recruitment Details	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.
Pre-assignment Details	Interim results at data cut-off date 06 January 2022 has been reported. Final results will be posted after study completion.

Arm/Group Title	Eculizumab
Arm/Group Description	Participants received eculizumab weekly 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.
Period Title: Primary Evaluation Period (26 Weeks)
Started	11
Completed	10
Not Completed	1
Reason Not Completed
Ongoing during the Primary Evaluation Period	1
Period Title: Extension Period (Up to 208 Weeks)
Started	10
Completed	0
Not Completed	10
Reason Not Completed
Ongoing during the Extension Period	10

Baseline Characteristics

Arm/Group Title		Eculizumab
Arm/Group Description		Participants received eculizumab weekly 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.
Overall Number of Baseline Participants		11
Baseline Analysis Population Description		Full analysis set included all participants who received at least 1 dose of eculizumab.
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	11 participants
		14.8 (1.78)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	11 participants
	Female	9 81.8%
	Male	2 18.2%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	11 participants
	Hispanic or Latino	2 18.2%
	Not Hispanic or Latino	9 81.8%
	Unknown or Not Reported	0 0.0%
Race/Ethnicity, Customized; Measure Type: Count of Participants; Unit of measure: Participants
Race	Number Analyzed	11 participants
	Asian	3 27.3%
	Black or African American	5 45.5%
	White	2 18.2%
	Other	1 9.1%
QMG Total Score [1]; Mean (Standard Deviation); Unit of measure: Units on a scale
	Number Analyzed	11 participants
		16.7 (5.64)
		[1] Measure Description: 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 score indicating most severe disease.

Outcome Measures

1. Primary Outcome

Title	Change From Baseline in the QMG Total Score at Week 26 Regardless of Rescue Treatment
Description	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 score indicating more severe disease.
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Modified full analysis set (mFAS) included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Mean (Standard Deviation); Unit of Measure: units on a scale
	-6.1 (4.56)

2. Secondary Outcome

Title	Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score at Week 26 Regardless of Rescue Treatment
Description	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 score indicating more severe disease.
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Mean (Standard Deviation); Unit of Measure: units on a scale
	-2.5 (1.78)

3. Secondary Outcome

Title	Percentage of Participants With ≥3-Point Reduction in the MG-ADL Total Score With No Rescue Treatment
Description	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 score indicating more severe disease.
Time Frame	Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	50.0; (18.7 to 81.3)

4. Secondary Outcome

Title	Percentage of Participants With ≥3-Point Reduction in the MG-ADL Total Score Regardless of Rescue Treatment
Description	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 score indicating more severe disease.
Time Frame	Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	50.0; (18.7 to 81.3)

5. Secondary Outcome

Title	Percentage of Participants With ≥5-Point Reduction in the QMG Total Score With No Rescue Treatment
Description	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 score indicating more severe disease.
Time Frame	Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	70.0; (34.8 to 93.3)

6. Secondary Outcome

Title	Percentage of Participants With ≥5-Point Reduction in the QMG Total Score Regardless of Rescue Treatment
Description	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 score indicating more severe disease.
Time Frame	Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	70.0; (34.8 to 93.3)

7. Secondary Outcome

Title	Change From Baseline in the Myasthenia Gravis Composite (MGC) Scale Total Score at Week 26 Regardless of Rescue Treatment
Description	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.
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	9
Mean (Standard Deviation); Unit of Measure: units on a scale
	-9.6 (6.25)

8. Secondary Outcome

Title	Change From Baseline in the European Quality of Life 5-Dimension Youth Version (EQ-5D-Y) Scale Score at Week 26 Regardless of Rescue Treatment
Description	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 cm VAS where the endpoints are labelled 'Best imaginable health state, marked as 100' and 'Worst imaginable health state, marked as 0'.
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Mean (Standard Deviation); Unit of Measure: units on a scale
	23.5 (23.34)

9. Secondary Outcome

Title	Change From Baseline in the Neurological Quality of Life-Fatigue Questionnaire (Neuro-QoL Pediatric Fatigue) Total Score at Week 26 Regardless of Rescue Treatment
Description	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.
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Mean (Standard Deviation); Unit of Measure: units on a scale
	-7.9 (7.37)

10. Secondary Outcome

Title	Number of Participants in Each Category of the Myasthenia Gravis Foundation of America Post-Intervention Status (MGFAPIS) Regardless of Rescue Treatment at Week 26
Description	The MG clinical state (improved, unchanged, and worse) was assessed using the MGFAPIS.
Time Frame	Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab. Overall number of participants analyzed = participants evaluable for this outcome measure.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	10
Measure Type: Count of Participants; Unit of Measure: Participants
Improved	10 100.0%
Unchanged	0 0.0%
Worse	0 0.0%

11. Secondary Outcome

Title	Percentage of Participants With Clinical Deteriorations, Myasthenic Crises, and Rescue Therapy Use
Description	Rescue therapy (for example, high dose corticosteroid, plasma exchange [PE], or intravenous immunoglobulin [IVIg]) 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.
Time Frame	Baseline up to Week 26

Outcome Measure Data

Analysis Population Description

mFAS included participants 12 to <18 years of age who received at least 1 dose of eculizumab.

Arm/Group Title	Eculizumab
Arm/Group Description:	Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed	11
Measure Type: Number; Unit of Measure: percentage of participants
Clinical Deterioration	9.1
MG Crisis	9.1
Requiring Rescue Therapy	9.1

12. Secondary Outcome

Title	Pharmacokinetics (PK): Serum Concentration Of Eculizumab
Description	[Not Specified]
Time Frame	24 hours postdose on Day 1; predose and 60 minutes postdose at Week 12; predose at Week 26

Outcome Measure Data

Analysis Population Description

PK analysis set included participants who had PK data assessments during this study. Here, number analyzed = participants evaluable at specified timepoint.

Arm/Group Title		Eculizumab
Arm/Group Description:		Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed		11
Mean (Standard Deviation); Unit of Measure: micrograms (μg)/milliliter (mL)
Day 1, 24 hours postdose (n = 11)	Number Analyzed	11 participants
		359.6 (105.18)
Week 12, Predose	Number Analyzed	10 participants
		382.8 (159.57)
Week 12, 60 minutes postdose	Number Analyzed	11 participants
		910.5 (277.29)
Week 26, Predose	Number Analyzed	9 participants
		433.9 (171.85)

13. Secondary Outcome

Title	Pharmacodynamics (PD): Serum Free Complement Component 5 (C5) Concentrations
Description	[Not Specified]
Time Frame	Baseline; 24 hours postdose on Day 1; predose and 60 minutes postdose at Week 12; predose at Week 26

Outcome Measure Data

Analysis Population Description

PD analysis set included participants who had PD data assessments during this study. Here, number analyzed = participants evaluable at specified timepoint.

Arm/Group Title		Eculizumab
Arm/Group Description:		Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed		11
Mean (Standard Deviation); Unit of Measure: μg/mL
Baseline	Number Analyzed	11 participants
		172.7 (34.52)
Day 1, 24 hours postdose	Number Analyzed	11 participants
		0.0 (0.01)
Week 12, Predose	Number Analyzed	11 participants
		0.0 (0.01)
Week 12, 60 minutes postdose	Number Analyzed	11 participants
		0.0 (0.01)
Week 26, Predose	Number Analyzed	10 participants
		0.0 (0.02)

14. Secondary Outcome

Title	PD: Percentage of Hemolysis (In Vitro Assay)
Description	[Not Specified]
Time Frame	Baseline; 24 hours postdose on Day 1; predose and 60 minutes postdose at Week 12; predose at Week 26

Outcome Measure Data

Analysis Population Description

PD analysis set included participants who had PD data assessments during this study. Here, 'Overall number of participants analyzed' = participants evaluable for this endpoint. Number analyzed = participants evaluable at specified timepoint.

Arm/Group Title		Eculizumab
Arm/Group Description:		Participants received eculizumab weekly 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.
Overall Number of Participants Analyzed		10
Mean (Standard Deviation); Unit of Measure: percentage of hemolysis
Baseline	Number Analyzed	10 participants
		105.8 (14.15)
Day 1, 24 hours postdose	Number Analyzed	10 participants
		1.1 (2.01)
Week 12, Predose	Number Analyzed	10 participants
		1.8 (4.67)
Week 12, 60 minutes postdose	Number Analyzed	10 participants
		0.2 (0.45)
Week 26, Predose	Number Analyzed	9 participants
		0.5 (1.29)

Adverse Events

Time Frame	Baseline up to the data cut-off date 6 January 2022 (up to approximately 3 years)
Adverse Event Reporting Description	Safety analysis set included all participants who received at least 1 dose of eculizumab.
Arm/Group Title	Eculizumab
Arm/Group Description	Participants received eculizumab weekly 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.
All-Cause Mortality
	Eculizumab
	Affected / at Risk (%)
Total	0/11 (0.00%)
Serious Adverse Events
	Eculizumab
	Affected / at Risk (%)	# Events
Total	3/11 (27.27%)
General disorders
Pyrexia † 1	1/11 (9.09%)	1
Infections and infestations
Peritonsillar abscess † 1	1/11 (9.09%)	1
Nervous system disorders
Myasthenia gravis † 1	1/11 (9.09%)	3
Myasthenia gravis crisis † 1	1/11 (9.09%)	1
1 Term from vocabulary, MedDRA 24.1; † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Eculizumab
	Affected / at Risk (%)	# Events
Total	11/11 (100.00%)
Blood and lymphatic system disorders
Iron deficiency anaemia † 1	1/11 (9.09%)	1
Leukopenia † 1	1/11 (9.09%)	1
Lymphocytosis † 1	1/11 (9.09%)	1
Monocytosis † 1	1/11 (9.09%)	1
Neutropenia † 1	1/11 (9.09%)	1
Cardiac disorders
Palpitations † 1	2/11 (18.18%)	2
Ear and labyrinth disorders
Ear pain † 1	1/11 (9.09%)	1
Eye disorders
Eye pruritus † 1	1/11 (9.09%)	1
Lacrimation increased † 1	1/11 (9.09%)	1
Ocular hyperaemia † 1	1/11 (9.09%)	1
Gastrointestinal disorders
Abdominal pain † 1	2/11 (18.18%)	2
Diarrhoea † 1	2/11 (18.18%)	2
Vomiting † 1	2/11 (18.18%)	2
Abdominal distension † 1	1/11 (9.09%)	1
Abdominal pain upper † 1	1/11 (9.09%)	1
Constipation † 1	1/11 (9.09%)	2
Flatulence † 1	1/11 (9.09%)	1
Mouth ulceration † 1	1/11 (9.09%)	15
Nausea † 1	1/11 (9.09%)	1
General disorders
Fatigue † 1	2/11 (18.18%)	3
Infusion site extravasation † 1	1/11 (9.09%)	2
Injection site bruising † 1	1/11 (9.09%)	1
Injection site pain † 1	1/11 (9.09%)	1
Vaccination site pain † 1	1/11 (9.09%)	1
Pyrexia † 1	2/11 (18.18%)	4
Infections and infestations
Nasopharyngitis † 1	4/11 (36.36%)	5
Upper respiratory tract infection † 1	2/11 (18.18%)	2
COVID-19 † 1	1/11 (9.09%)	1
Cellulitis † 1	1/11 (9.09%)	1
Pharyngitis † 1	1/11 (9.09%)	1
Post viral fatigue syndrome † 1	1/11 (9.09%)	1
Viral infection † 1	1/11 (9.09%)	2
Injury, poisoning and procedural complications
Thermal burn † 1	2/11 (18.18%)	2
Vaccination complication † 1	2/11 (18.18%)	2
Arthropod bite † 1	1/11 (9.09%)	1
Investigations
Glucose urine present † 1	1/11 (9.09%)	1
Electrocardiogram PR prolongation † 1	1/11 (9.09%)	1
Metabolism and nutrition disorders
Decreased appetite † 1	2/11 (18.18%)	2
Ketosis † 1	1/11 (9.09%)	1
Hypokalaemia † 1	1/11 (9.09%)	1
Musculoskeletal and connective tissue disorders
Pain in extremity † 1	3/11 (27.27%)	4
Costochondritis † 1	1/11 (9.09%)	1
Muscle spasms † 1	1/11 (9.09%)	1
Muscle twitching † 1	1/11 (9.09%)	1
Musculoskeletal stiffness † 1	1/11 (9.09%)	1
Myalgia † 1	1/11 (9.09%)	1
Nervous system disorders
Headache † 1	4/11 (36.36%)	12
Dizziness † 1	1/11 (9.09%)	1
Tremor † 1	1/11 (9.09%)	1
Product Issues
Device malfunction † 1	1/11 (9.09%)	1
Psychiatric disorders
Behaviour disorder † 1	1/11 (9.09%)	1
Panic attack † 1	1/11 (9.09%)	1
Renal and urinary disorders
Hypercalciuria † 1	1/11 (9.09%)	1
Respiratory, thoracic and mediastinal disorders
Nasal congestion † 1	2/11 (18.18%)	2
Cough † 1	1/11 (9.09%)	1
Oropharyngeal pain † 1	1/11 (9.09%)	1
Sinus congestion † 1	1/11 (9.09%)	1
Skin and subcutaneous tissue disorders
Acne † 1	1/11 (9.09%)	1
Dry skin † 1	1/11 (9.09%)	2
Eczema † 1	1/11 (9.09%)	7
Pruritus † 1	1/11 (9.09%)	1
Rash † 1	1/11 (9.09%)	1
Urticaria † 1	1/11 (9.09%)	1
Vascular disorders
Poor venous access † 1	1/11 (9.09%)	1
1 Term from vocabulary, MedDRA 24.1; † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals Inc.
• Phone: +1.855.752.2356
• EMail: clinicaltrials@alexion.com

• Responsible Party: Alexion Pharmaceuticals, Inc.
• ClinicalTrials.gov Identifier: NCT03759366
• Other Study ID Numbers: ECU-MG-303
• First Submitted: November 26, 2018
• First Posted: November 30, 2018
• Results First Submitted: August 1, 2022
• Results First Posted: August 25, 2022
• Last Update Posted: December 18, 2023