Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis

• ClinicalTrials.gov Identifier: NCT03315130
• Recruitment Status: Completed
• First Posted: October 19, 2017
• Results First Posted: June 27, 2022
• Last Update Posted: July 27, 2022
• Sponsor: Ra Pharmaceuticals
• Information provided by (Responsible Party): UCB Pharma ( Ra Pharmaceuticals )

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Generalized Myasthenia Gravis
• Interventions: Drug: zilucoplan (RA101495); Drug: Placebo
• Enrollment: 45

Participant Flow

Recruitment Details	The study started to enroll participants in October 2017 and concluded in November 2020.
Pre-assignment Details	The Participant flow refers to the Intent-to-treat Set for the main portion and Safety Set for the extension portion.

Arm/Group Title	RA101495 0.1 mg/kg	RA101495 0.3 mg/kg	Placebo	RA101495 0.1 mg/kg (Before Switch) to 0.3 mg/kg (After Switch)
Arm/Group Description	Participants received RA101495 0.1 milligram/kilogram (mg/kg) as a subcutaneous injection once daily for 12 weeks in the main portion. Participants who completed main portion and were eligible to enter in the extension portion, continued receiving RA101495 0.1 mg/kg up to 48 weeks (until protocol amendment v3.0) in the extension portion (EP).	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. At the end of the treatment period in the main portion, participants received the same dose of study drug in the extension portion until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for generalized myasthenia gravis (gMG). In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. At the end of the treatment period in the main portion, participants were randomized to receive either RA101495 0.1 mg/kg (up to 48 weeks) or RA101495 0.3 mg/kg (up to 122 weeks) in the extension portion, as a subcutaneous injection once daily.	Following implementation of protocol amendment v3.0, and upon appropriate reconsent, all study participants ongoing in the extension portion who had previously received the RA101495 0.1mg/kg/day dose switched to receive the RA101495 0.3mg/kg/day until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for gMG. In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).
Period Title: Main Portion (12 Weeks)
Started	15	15	15	0
Completed	15	13	15	0
Not Completed	0	2	0	0
Reason Not Completed
Subject withdrew consent	0	1	0	0
Lost to Follow-up	0	1	0	0
Period Title: EP:Before Switch to 0.3 mg/kg(Week13-48)
Started	22 [1]	21 [2]	0	0
Completed	21	21	0	0
Not Completed	1	0	0	0
Reason Not Completed
Subject withdrew consent	1	0	0	0
[1] (15 [who received RA101495 0.1 mg/kg] + 7 [crossover from placebo]); [2] (14 [who received RA101495 0.3 mg/kg + 7 [crossover from placebo] + 1 [Subject who withdrew consent in Main Portion rejoined])
Period Title: EP:After Switch to 0.3 mg/kg(Week48-122)
Started	0	21	0	21
Completed	0	17	0	20
Not Completed	0	4	0	1
Reason Not Completed
Withdrawal by Subject	0	1	0	0
Subject withdrew consent	0	1	0	0
Death	0	2	0	1

Baseline Characteristics

Arm/Group Title		RA101495 0.1 mg/kg	RA101495 0.3 mg/kg	Placebo	Total
Arm/Group Description		Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants who completed main portion and were eligible to enter in the extension period, continued receiving RA101495 0.1 mg/kg up to 48 weeks (until protocol amendment v3.0) in the extension portion.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. At the end of the treatment period in the main portion, participants received the same dose of study drug in the extension portion until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for generalized myasthenia gravis (gMG). In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. At the end of the treatment period in the main portion, participants were randomized to receive either RA101495 0.1 mg/kg (up to 48 weeks) or RA101495 0.3 mg/kg (up to 122 weeks) in the extension portion, as a subcutaneous injection once daily.	Total of all reporting groups
Overall Number of Baseline Participants		15	15	15	45
Baseline Analysis Population Description		Intent-to-Treat (ITT) population included all randomized participants.
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	15 participants	15 participants	15 participants	45 participants
	<=18 years	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Between 18 and 65 years	14 93.3%	11 73.3%	13 86.7%	38 84.4%
	>=65 years	1 6.7%	4 26.7%	2 13.3%	7 15.6%
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	15 participants	15 participants	15 participants	45 participants
		45.5 (15.6)	54.5 (14.9)	48.4 (15.7)	49.5 (15.5)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	15 participants	15 participants	15 participants	45 participants
	Female	8 53.3%	5 33.3%	11 73.3%	24 53.3%
	Male	7 46.7%	10 66.7%	4 26.7%	21 46.7%
Race/Ethnicity, Customized; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	15 participants	15 participants	15 participants	45 participants
Asian		0 0.0%	1 6.7%	1 6.7%	2 4.4%
Black or African American		2 13.3%	3 20.0%	2 13.3%	7 15.6%
White		13 86.7%	11 73.3%	12 80.0%	36 80.0%

Outcome Measures

1. Primary Outcome

Title	Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score
Description	The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for myasthenia gravis (MG). The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.
Time Frame	From Baseline to Week 12

Outcome Measure Data

Analysis Population Description

The modified ITT (mITT) population included all participants in the ITT population who had received at least 1 dose of study drug.

Arm/Group Title	RA101495 0.1 mg/kg (mITT)	RA101495 0.3 mg/kg (mITT)	Placebo (mITT)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.
Overall Number of Participants Analyzed	15	14	15
Least Squares Mean (Standard Error); Unit of Measure: score on a scale
	-5.5 (1.2)	-6.0 (1.2)	-3.2 (1.2)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.1 mg/kg (mITT), Placebo (mITT)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	=0.0941
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-2.3
	Confidence Interval	(2-Sided) 80%; -4.5 to -0.1
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 1.7
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.3 mg/kg (mITT), Placebo (mITT)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	=0.0538
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-2.8
	Confidence Interval	(2-Sided) 80%; -5.1 to -0.6
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 1.7
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

2. Secondary Outcome

Title	Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale
Description	The MG-ADL is a brief 8-item survey designed to evaluate MG symptom severity. The scale consists of 8 items each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the 8 individual scores with range of 0-24. Higher scores are associated with more severe symptoms of MG.
Time Frame	From Baseline to Week 12

Outcome Measure Data

Analysis Population Description

The mITT population included all participants in the ITT population who had received at least 1 dose of study drug.

Arm/Group Title	RA101495 0.1 mg/kg (mITT)	RA101495 0.3 mg/kg (mITT)	Placebo (mITT)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.
Overall Number of Participants Analyzed	15	14	15
Least Squares Mean (Standard Error); Unit of Measure: score on a scale
	-3.3 (0.9)	-3.4 (0.9)	-1.1 (0.9)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.1 mg/kg (mITT), Placebo (mITT)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	=0.0470
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-2.2
	Confidence Interval	(2-Sided) 80%; -3.9 to -0.5
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 1.3
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.3 mg/kg (mITT), Placebo (mITT)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	=0.0392
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-2.3
	Confidence Interval	(2-Sided) 80%; -4.0 to -0.6
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 1.3
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

3. Secondary Outcome

Title	Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life 15r (MG-QOL15r) Survey
Description	The MG-QOL15r is a 15-item survey that was designed to assess quality of life in participants with MG. The survey consisted of 15 questions and the corresponding responses were each scored on a 0-2 point scale (0=Not much at all, 1=Somewhat, 2=Very Much). The total score is the sum of the 15 individual item scores with a range of 0-30. Higher scores indicate more severe impact of the disease on aspects of the participant's life.
Time Frame	From Baseline to Week 12

Outcome Measure Data

Analysis Population Description

The mITT population included all participants in the ITT population who had received at least 1 dose of study drug.

Arm/Group Title	RA101495 0.1 mg/kg (mITT)	RA101495 0.3 mg/kg (mITT)	Placebo (mITT)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.
Overall Number of Participants Analyzed	15	14	15
Least Squares Mean (Standard Error); Unit of Measure: score on a scale
	-7.4 (1.7)	-5.9 (1.7)	-2.1 (1.7)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.1 mg/kg (mITT), Placebo (mITT)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	=0.0170
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-5.3
	Confidence Interval	(2-Sided) 80%; -8.4 to -2.1
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 2.4
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.3 mg/kg (mITT), Placebo (mITT)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	=0.0624
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-3.7
	Confidence Interval	(2-Sided) 80%; -6.9 to -0.6
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 2.4
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

4. Secondary Outcome

Title	Main Portion: Change From Baseline to Week 12 in the MG Composite Scale Total Score
Description	The MG Composite is a 10-item scale that has been used to measure the clinical status of participants with MG, in order to evaluate treatment response. It consists of 10 items which included ptosis (score range=0 to 3), double vision on lateral gaze left/right/both (score range=0 to 4), eye closure (score range=0 to 2), talking (score range=0 to 6), chewing (score range=0 to 6), swallowing (score range=0 to 6), breathing (score range=0 to 9), neck flexion or extension (score range=0 to 4), shoulder abduction (score range=0 to 5) and hip flexion (score range= 0 to 5). The total score is the sum of the 10 individual scores with a range of 0-50. Higher scores in the MG Composite indicate more severe impairment due to the disease.
Time Frame	From Baseline to Week 12

Outcome Measure Data

Analysis Population Description

The mITT population included all participants in the ITT population who had received at least 1 dose of study drug.

Arm/Group Title	RA101495 0.1 mg/kg (mITT)	RA101495 0.3 mg/kg (mITT)	Placebo (mITT)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.
Overall Number of Participants Analyzed	15	14	15
Least Squares Mean (Standard Error); Unit of Measure: score on a scale
	-5.3 (1.5)	-7.4 (1.6)	-3.3 (1.6)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.1 mg/kg (mITT), Placebo (mITT)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	=0.1866
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-2.0
	Confidence Interval	(2-Sided) 80%; -4.9 to 0.9
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 2.2
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.3 mg/kg (mITT), Placebo (mITT)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	=0.0391
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-4.1
	Confidence Interval	(2-Sided) 80%; -7.0 to -1.1
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 2.2
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

5. Secondary Outcome

Title	Main Portion: Percentage of Participants With >= 3-point Reduction in QMG Total Score at Week 12
Description	The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.
Time Frame	Week 12

Outcome Measure Data

Analysis Population Description

The mITT population included all participants in the ITT population who had received at least 1 dose of study drug.

Arm/Group Title	RA101495 0.1 mg/kg (mITT)	RA101495 0.3 mg/kg (mITT)	Placebo (mITT)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.
Overall Number of Participants Analyzed	15	14	15
Measure Type: Number; Unit of Measure: percentage of participants
	66.7	71.4	53.3

6. Secondary Outcome

Title	Main Portion: Percentage of Participants Who Required Rescue Therapy Over the 12-week Treatment Period
Description	Percentage of participants who used at least 1 dose of rescue medication were reported.
Time Frame	Up to Week 12

Outcome Measure Data

Analysis Population Description

The mITT population included all participants in the ITT population who had received at least 1 dose of study drug.

Arm/Group Title	RA101495 0.1 mg/kg (mITT)	RA101495 0.3 mg/kg (mITT)	Placebo (mITT)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.
Overall Number of Participants Analyzed	15	14	15
Measure Type: Number; Unit of Measure: percentage of participants
	6.7	0	20.0

7. Secondary Outcome

Title	Main Portion: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Description	An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A serious adverse event (SAE) is any untoward medical occurrence that at any dose resulted in death, life-threatening, significant or persistent disability/incapacity, congenital anomaly/birth defect, important medical event, initial inpatient hospitalization or prolongation of hospitalization.
Time Frame	From Baseline to Week 12

Outcome Measure Data

Analysis Population Description

The safety population included all participants who had received at least 1 dose of study drug (i.e., mITT Population), with participants to be analyzed based on the actual treatment received.

Arm/Group Title	RA101495 0.1 mg/kg (SS)	RA101495 0.3 mg/kg (SS)	Placebo (SS)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the safety set (SS) population which included all participants who had received at least 1 dose of study drug (i.e., mITT Population), with participants to be analyzed based on the actual treatment received.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the SS population which included all participants who had received at least 1 dose of study drug (i.e., mITT Population), with participants to be analyzed based on the actual treatment received.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the SS population which included all participants who had received at least 1 dose of study drug (i.e., mITT Population), with participants to be analyzed based on the actual treatment received.
Overall Number of Participants Analyzed	15	14	15
Measure Type: Count of Participants; Unit of Measure: Participants
AEs	15 100.0%	12 85.7%	14 93.3%
SAEs	0 0.0%	5 35.7%	3 20.0%

8. Secondary Outcome

Title	Main Portion: Change From Baseline in Sheep Red Blood Cell (sRBC) Lysis Assay at Week 12 (Pre-dose)
Description	A BioTek ELx800 automated microplate reader is used to measure the optical density at 415 nanometer (nm) of human plasma samples to calculate the percent lysis of sheep erythrocytes that has occurred as a result of total complement activity. The measure of complement activity is determined by the degree of hemolysis of the erythrocytes.
Time Frame	Baseline and Week 12 (Pre-dose)

Outcome Measure Data

Analysis Population Description

The pharmacodynamic (PD) population included all participants in mITT Population who had at least 1 evaluable PD assessment.

Arm/Group Title	RA101495 0.1 mg/kg (PD)	RA101495 0.3 mg/kg (PD)	Placebo (PD)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.
Overall Number of Participants Analyzed	15	14	15
Least Squares Mean (Standard Error); Unit of Measure: percent lysis of sheep erythrocytes
	-81.822 (2.469)	-94.914 (2.908)	0.775 (2.571)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.1 mg/kg (PD), Placebo (PD)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-82.597
	Confidence Interval	(2-Sided) 80%; -87.249 to -77.946
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 3.563
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.3 mg/kg (PD), Placebo (PD)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	-95.689
	Confidence Interval	(2-Sided) 80%; -100.794 to -90.585
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 3.910
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

9. Secondary Outcome

Title	Main Portion: Change From Baseline in C5 Levels at Week 12 (Pre-dose)
Description	Blood samples were collected from participants to assess Complement Component 5C levels.
Time Frame	Baseline and Week 12 (Pre-dose)

Outcome Measure Data

Analysis Population Description

The PD population included all participants in mITT Population who had at least 1 evaluable PD assessment.

Arm/Group Title	RA101495 0.1 mg/kg (PD)	RA101495 0.3 mg/kg (PD)	Placebo (PD)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.	Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.
Overall Number of Participants Analyzed	15	14	15
Least Squares Mean (Standard Error); Unit of Measure: micrograms/milliliter (ug/mL)
	57.349 (7.057)	54.302 (6.804)	-2.774 (6.237)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.1 mg/kg (PD), Placebo (PD)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	60.123
	Confidence Interval	(2-Sided) 80%; 47.839 to 72.408
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 9.394
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	RA101495 0.3 mg/kg (PD), Placebo (PD)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	1-sided p-value is presented for each active treatment arm compared to the Placebo treatment arm.
	Method	ANCOVA
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	57.076
	Confidence Interval	(2-Sided) 80%; 44.955 to 69.197
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 9.269
	Estimation Comments	LS mean difference and associated confidence interval are presented for each active treatment arm compared to the Placebo treatment arm.

10. Secondary Outcome

Title	Main Portion: Plasma Concentration of RA101495 and Its Major Metabolites
Description	RA102758 and RA103488 are the metabolites of RA101495.
Time Frame	1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12

Outcome Measure Data

Analysis Population Description

The pharmacokinetic (PK) population included all participants in mITT population who had at least 1 evaluable PK assessment. Here "Number Analyzed" signifies number of participants who were evaluable at specified time points. 1 Participant (who was randomized to Placebo) mistakenly received Zilucoplan on Day 1 following a dispensing error (which was documented as a minor PD). Due to data confidentiality reasons, the results have not been reported for the placebo arm.

Arm/Group Title		RA101495 0.1 mg/kg (PK)	RA101495 0.3 mg/kg (PK)
Arm/Group Description:		Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.
Overall Number of Participants Analyzed		15	14
Mean (Standard Deviation); Unit of Measure: nanograms/milliliter (ng/mL)
RA101495- Day 1: 1 hour postdose	Number Analyzed	14 participants	14 participants
		652.136 (354.652)	2272.257 (1246.128)
RA101495- Day 1: 3 hours postdose	Number Analyzed	15 participants	14 participants
		1691.267 (469.766)	4176.000 (1211.166)
RA101495- Day 1: 6 hours postdose	Number Analyzed	14 participants	13 participants
		1925.357 (330.335)	4636.000 (809.804)
RA101495- Week 1: Pre-dose	Number Analyzed	14 participants	14 participants
		4571.214 (719.566)	9223.786 (2365.168)
RA101495- Week 2: Pre-dose	Number Analyzed	15 participants	13 participants
		4914.200 (743.215)	10222.846 (1662.869)
RA101495- Week 4: Pre-dose	Number Analyzed	14 participants	12 participants
		5100.286 (859.767)	10265.917 (1604.004)
RA101495- Week 8: Pre-dose	Number Analyzed	15 participants	12 participants
		5222.067 (688.427)	10075.000 (1872.400)
RA101495- Week 12: Pre-dose	Number Analyzed	15 participants	13 participants
		5281.333 (1122.372)	10284.846 (1771.586)
RA102758- Day 1: 1 hour postdose	Number Analyzed	14 participants	14 participants
		NA [1] (NA)	NA [2] (NA)
RA102758- Day 1: 3 hours postdose	Number Analyzed	15 participants	14 participants
		NA [2] (NA)	NA [2] (NA)
RA102758- Day 1: 6 hours postdose	Number Analyzed	14 participants	13 participants
		NA [2] (NA)	NA [2] (NA)
RA102758- Week 1: Pre-dose	Number Analyzed	14 participants	14 participants
		252.693 (78.909)	849.743 (273.388)
RA102758- Week 2: Pre-dose	Number Analyzed	15 participants	13 participants
		408.847 (94.773)	1473.431 (440.496)
RA102758- Week 4: Pre-dose	Number Analyzed	14 participants	12 participants
		462.486 (180.098)	1567.692 (557.745)
RA102758- Week 8: Pre-dose	Number Analyzed	15 participants	12 participants
		474.073 (157.549)	1432.808 (530.377)
RA102758- Week 12: Pre-dose	Number Analyzed	15 participants	13 participants
		465.665 (195.227)	1459.062 (451.013)
RA103488- Day 1: 1 hour postdose	Number Analyzed	14 participants	14 participants
		NA [2] (NA)	NA [2] (NA)
RA103488- Day 1: 3 hours postdose	Number Analyzed	15 participants	14 participants
		NA [2] (NA)	NA [2] (NA)
RA103488- Day 1: 6 hours postdose	Number Analyzed	14 participants	13 participants
		NA [2] (NA)	85.832 (257.664)
RA103488- Week 1: Pre-dose	Number Analyzed	14 participants	14 participants
		356.186 (162.209)	838.907 (255.419)
RA103488- Week 2: Pre-dose	Number Analyzed	15 participants	13 participants
		501.847 (218.467)	1135.731 (270.004)
RA103488- Week 4: Pre-dose	Number Analyzed	14 participants	12 participants
		536.266 (275.013)	1191.783 (251.853)
RA103488- Week 8: Pre-dose	Number Analyzed	15 participants	12 participants
		615.740 (229.359)	1234.708 (201.055)
RA103488- Week 12: Pre-dose	Number Analyzed	15 participants	13 participants
		621.813 (241.354)	1307.492 (331.553)

[1]

Mean and Standard Deviation are only calculated if at least 2/3 of the individual data points are above lower limit of quantification (LLOQ).

[2]

Mean and Standard Deviation are only calculated if at least 2/3 of the individual data points are above LLOQ.

11. Secondary Outcome

Title	Main Portion: Maximum Plasma Concentration (Cmax) on Day 1
Description	Cmax is defined as the maximum observed plasma concentration.
Time Frame	Pre-dose, 1, 3 and 6 hours postdose on Day 1

Outcome Measure Data

Analysis Population Description

The PK Population included all participants in mITT population who had at least 1 evaluable PK assessment. 1 Participant (who was randomized to Placebo) mistakenly received Zilucoplan on Day 1 following a dispensing error (which was documented as a minor PD). Due to data confidentiality reasons, the results have not been reported for the placebo arm.

Arm/Group Title	RA101495 0.1 mg/kg (PK)	RA101495 0.3 mg/kg (PK)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.
Overall Number of Participants Analyzed	15	14
Mean (Standard Deviation); Unit of Measure: ng/mL
	1945.467 (407.273)	4858.643 (1004.457)

12. Secondary Outcome

Title	Main Portion: Time Corresponding to Cmax (Tmax) on Day 1
Description	Tmax is defined as the time to observe maximum plasma concentration.
Time Frame	Pre-dose, 1, 3 and 6 hours postdose on Day 1

Outcome Measure Data

Analysis Population Description

The PK Population included all participants in mITT population who had at least 1 evaluable PK assessment. 1 Participant (who was randomized to Placebo) mistakenly received Zilucoplan on Day 1 following a dispensing error (which was documented as a minor PD). Due to data confidentiality reasons, the results have not been reported for the placebo arm.

Arm/Group Title	RA101495 0.1 mg/kg (PK)	RA101495 0.3 mg/kg (PK)
Arm/Group Description:	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.
Overall Number of Participants Analyzed	15	14
Median (Full Range); Unit of Measure: hours
	4.550; (2.95 to 5.50)	4.700; (2.62 to 5.92)

13. Secondary Outcome

Title	Main Portion: Metabolites (RA102758 and RA103488) to Parent Ratio
Description	RA102758 and RA103488 are the metabolites of RA101495.
Time Frame	Pre-dose, 1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12

Outcome Measure Data

Analysis Population Description

The PK Population included all participants in mITT population who had at least 1 evaluable PK assessment. Here "Number Analyzed" signifies number of participants who were evaluable at specified time points. 1 Participant (who was randomized to Placebo) mistakenly received Zilucoplan on Day 1 following a dispensing error (which was documented as a minor PD). Due to data confidentiality reasons, the results have not been reported for the placebo arm.

Arm/Group Title		RA101495 0.1 mg/kg (PK)	RA101495 0.3 mg/kg (PK)
Arm/Group Description:		Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.	Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.
Overall Number of Participants Analyzed		15	14
Mean (Standard Deviation); Unit of Measure: ratio
RA102758/RA101495- Day 1: Pre-dose	Number Analyzed	14 participants	14 participants
		NA [1] (NA)	NA [1] (NA)
RA102758/RA101495- Day 1: 1 hour postdose	Number Analyzed	14 participants	14 participants
		0.000 (0.000)	0.000 (0.000)
RA102758/RA101495- Day 1: 3 hours postdose	Number Analyzed	15 participants	14 participants
		0.000 (0.000)	0.000 (0.000)
RA102758/RA101495- Day 1: 6 hours postdose	Number Analyzed	14 participants	13 participants
		0.000 (0.000)	0.013 (0.044)
RA102758/RA101495- Week 1: Pre-dose	Number Analyzed	14 participants	14 participants
		0.121 (0.033)	0.202 (0.038)
RA102758/RA101495- Week 2: Pre-dose	Number Analyzed	15 participants	13 participants
		0.182 (0.038)	0.307 (0.057)
RA102758/RA101495- Week 4: Pre-dose	Number Analyzed	13 participants	12 participants
		0.197 (0.072)	0.323 (0.072)
RA102758/RA101495- Week 8: Pre-dose	Number Analyzed	15 participants	12 participants
		0.195 (0.057)	0.301 (0.070)
RA102758/RA101495- Week 12: Pre-dose	Number Analyzed	15 participants	13 participants
		0.184 (0.065)	0.303 (0.061)
RA103488/RA101495- Day 1: Pre-dose	Number Analyzed	14 participants	14 participants
		NA [1] (NA)	NA [1] (NA)
RA103488/RA101495- Day 1: 1 hour postdose	Number Analyzed	14 participants	14 participants
		0.000 (0.000)	0.000 (0.000)
RA103488/RA101495- Day 1: 3 hours postdose	Number Analyzed	15 participants	14 participants
		0.000 (0.000)	0.001 (0.001)
RA103488/RA101495- Day 1: 6 hours postdose	Number Analyzed	14 participants	13 participants
		0.000 (0.000)	0.015 (0.045)
RA103488/RA101495- Week 1: Pre-dose	Number Analyzed	14 participants	14 participants
		0.079 (0.037)	0.099 (0.044)
RA103488/RA101495- Week 2: Pre-dose	Number Analyzed	15 participants	13 participants
		0.104 (0.044)	0.114 (0.037)
RA103488/RA101495- Week 4: Pre-dose	Number Analyzed	13 participants	12 participants
		0.106 (0.046)	0.119 (0.038)
RA103488/RA101495- Week 8: Pre-dose	Number Analyzed	15 participants	12 participants
		0.118 (0.044)	0.127 (0.040)
RA103488/RA101495- Week 12: Pre-dose	Number Analyzed	15 participants	13 participants
		0.116 (0.035)	0.131 (0.041)

[1]

Mean and Standard Deviation are only calculated if at least 2/3 of the individual data points are above LLOQ.

Adverse Events

Time Frame	From Baseline up to Week 122
Adverse Event Reporting Description	Treatment emergent adverse events (TEAEs) are defined as an AE that occurs after a treatment start date or an AE that increases in severity after treatment start date. In this study, AEs were planned to be reported for Main portion and Main + Extension portion only.
Arm/Group Title	Main Portion: RA101495 0.1 mg/kg		Main Portion: RA101495 0.3 mg/kg		Main Portion: Placebo		Main and Extension Portion: RA101495 0.1 mg/kg Before Switch to 0.3 mg/kg		Main and Extension Portion: RA101495 0.1 mg/kg After Switch to 0.3 mg/kg		Main and Extension Portion: RA101495 0.3 mg/kg
Arm/Group Description	Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion.		Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion		Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion.		Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants who completed main portion and were eligible to enter in the extension period, continued receiving RA101495 0.1 mg/kg up to 48 weeks (until protocol amendment v3.0) in the extension portion.		Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants who completed main portion and were eligible to enter in the extension period, continued receiving RA101495 0.1 mg/kg up to Week 48 (until protocol amendment v3.0). Following implementation of protocol amendment v3.0, and upon appropriate reconsent, all study participants ongoing in the extension portion who had previously received the RA101495 0.1mg/kg/day dose switched to receive the RA101495 0.3mg/kg/day until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for gMG. In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).		Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. At the end of the treatment period in the main portion, participants received the same dose of study drug in the extension portion until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for gMG. In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).
All-Cause Mortality
	Main Portion: RA101495 0.1 mg/kg		Main Portion: RA101495 0.3 mg/kg		Main Portion: Placebo		Main and Extension Portion: RA101495 0.1 mg/kg Before Switch to 0.3 mg/kg		Main and Extension Portion: RA101495 0.1 mg/kg After Switch to 0.3 mg/kg		Main and Extension Portion: RA101495 0.3 mg/kg
	Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)
Total	0/15 (0.00%)		0/14 (0.00%)		0/15 (0.00%)		1/22 (4.55%)		0/21 (0.00%)		2/21 (9.52%)
Serious Adverse Events
	Main Portion: RA101495 0.1 mg/kg		Main Portion: RA101495 0.3 mg/kg		Main Portion: Placebo		Main and Extension Portion: RA101495 0.1 mg/kg Before Switch to 0.3 mg/kg		Main and Extension Portion: RA101495 0.1 mg/kg After Switch to 0.3 mg/kg		Main and Extension Portion: RA101495 0.3 mg/kg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/15 (0.00%)		5/14 (35.71%)		3/15 (20.00%)		5/22 (22.73%)		4/21 (19.05%)		11/21 (52.38%)
Cardiac disorders
Atrial fibrillation * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	2
Atrial flutter * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Bradycardia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	1/21 (4.76%)	1	0/21 (0.00%)	0
Cardiac arrest * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Myocardial infarction * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Gastrointestinal disorders
Pancreatic cyst * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Pancreatitis acute * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Abdominal pain * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	1/21 (4.76%)	1	0/21 (0.00%)	0
Gastric ulcer * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	1/21 (4.76%)	1	0/21 (0.00%)	0
Intra-abdominal fluid collection * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
General disorders
Systemic inflammatory response syndrome * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Non-cardiac chest pain * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Hepatobiliary disorders
Cholecystitis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Infections and infestations
Cellulitis * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Abdominal abscess * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Diverticulitis * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Pneumonia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	2	1/21 (4.76%)	1	1/21 (4.76%)	1
Pancreas infection * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Gastrointestinal infection * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Liver abscess * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Sepsis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	1/21 (4.76%)	1	0/21 (0.00%)	0
Staphylococcal bacteraemia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	1/21 (4.76%)	1
COVID-19 * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	1/21 (4.76%)	1	0/21 (0.00%)	0
Injury, poisoning and procedural complications
Post procedural complication * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Postoperative hypertension * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Spinal fracture * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Investigations
Blood culture positive * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Fibrin D dimer increased * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Musculoskeletal and connective tissue disorders
Back pain * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Rapidly progressive osteoarthritis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Musculoskeletal chest pain * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pancreatic carcinoma * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Nervous system disorders
Worsening of Myasthenia Gravis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	3/15 (20.00%)	3	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Syncope * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Renal and urinary disorders
Nephrolithiasis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	1/21 (4.76%)	2	0/21 (0.00%)	0
Ureterolithiasis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	1/21 (4.76%)	1	0/21 (0.00%)	0
Respiratory, thoracic and mediastinal disorders
Respiratory failure * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
1 Term from vocabulary, MedDRA v24.0; * Indicates events were collected by non-systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Main Portion: RA101495 0.1 mg/kg		Main Portion: RA101495 0.3 mg/kg		Main Portion: Placebo		Main and Extension Portion: RA101495 0.1 mg/kg Before Switch to 0.3 mg/kg		Main and Extension Portion: RA101495 0.1 mg/kg After Switch to 0.3 mg/kg		Main and Extension Portion: RA101495 0.3 mg/kg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	15/15 (100.00%)		12/14 (85.71%)		14/15 (93.33%)		22/22 (100.00%)		17/21 (80.95%)		21/21 (100.00%)
Blood and lymphatic system disorders
Iron deficiency anaemia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	1/22 (4.55%)	2	1/21 (4.76%)	1	1/21 (4.76%)	1
Anaemia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Increased tendency to bruise * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	2	0/21 (0.00%)	0	0/21 (0.00%)	0
Lymphadenopathy * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	2	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Cardiac disorders
Palpitations * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	2	0/21 (0.00%)	0	1/21 (4.76%)	1
Ear and labyrinth disorders
Vertigo * 1	0/15 (0.00%)	0	1/14 (7.14%)	2	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	3
Eye disorders
Cataract * 1	1/15 (6.67%)	2	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	2	1/21 (4.76%)	2	2/21 (9.52%)	2
Eyelid ptosis * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	2	1/21 (4.76%)	1	0/21 (0.00%)	0
Gastrointestinal disorders
Nausea * 1	2/15 (13.33%)	3	0/14 (0.00%)	0	0/15 (0.00%)	0	4/22 (18.18%)	7	1/21 (4.76%)	1	5/21 (23.81%)	8
Vomiting * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	3	2/21 (9.52%)	2	3/21 (14.29%)	4
Abdominal discomfort * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	1/22 (4.55%)	1	1/21 (4.76%)	1	1/21 (4.76%)	1
Abdominal pain upper * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	1/22 (4.55%)	1	0/21 (0.00%)	0	2/21 (9.52%)	2
Diarrhoea * 1	1/15 (6.67%)	1	1/14 (7.14%)	1	1/15 (6.67%)	1	2/22 (9.09%)	2	0/21 (0.00%)	0	5/21 (23.81%)	6
Constipation * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	2	0/21 (0.00%)	0	1/21 (4.76%)	1
Dysphagia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Dry mouth * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Lip dry * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Abdominal distension * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Mouth ulceration * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
General disorders
Injection site bruising * 1	2/15 (13.33%)	2	0/14 (0.00%)	0	2/15 (13.33%)	2	6/22 (27.27%)	13	0/21 (0.00%)	0	3/21 (14.29%)	3
Injection site scab * 1	3/15 (20.00%)	4	0/14 (0.00%)	0	0/15 (0.00%)	0	3/22 (13.64%)	4	0/21 (0.00%)	0	0/21 (0.00%)	0
Injection site nodule * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	2/21 (9.52%)	2	1/21 (4.76%)	1
Oedema peripheral * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	1/22 (4.55%)	1	1/21 (4.76%)	1	3/21 (14.29%)	3
Fatigue * 1	2/15 (13.33%)	2	1/14 (7.14%)	1	1/15 (6.67%)	1	2/22 (9.09%)	2	1/21 (4.76%)	1	2/21 (9.52%)	3
Influenza like illness * 1	1/15 (6.67%)	2	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	3	0/21 (0.00%)	0	1/21 (4.76%)	2
Chills * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	1/21 (4.76%)	1	1/21 (4.76%)	1
Pyrexia * 1	1/15 (6.67%)	2	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	3	1/21 (4.76%)	1	2/21 (9.52%)	2
Injection site pain * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	2
Injection site discomfort * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	3	0/21 (0.00%)	0	0/21 (0.00%)	0
Injection site haemorrhage * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Peripheral swelling * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	2	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Swelling face * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Pain * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	1/21 (4.76%)	1
Hepatobiliary disorders
Biliary colic * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Infections and infestations
Nasopharyngitis * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	6/22 (27.27%)	9	5/21 (23.81%)	6	8/21 (38.10%)	8
Upper respiratory tract infection * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	3/22 (13.64%)	3	2/21 (9.52%)	3	4/21 (19.05%)	5
Urinary tract infection * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	3/22 (13.64%)	3	2/21 (9.52%)	2	1/21 (4.76%)	2
Sinusitis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	2	0/21 (0.00%)	0	3/21 (14.29%)	3
Pharyngitis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Viral upper respiratory tract infection * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Viral infection * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Bronchitis * 1	1/15 (6.67%)	1	1/14 (7.14%)	1	0/15 (0.00%)	0	3/22 (13.64%)	3	0/21 (0.00%)	0	2/21 (9.52%)	2
Pneumonia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Herpes zoster * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Localised infection * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	1/21 (4.76%)	1
Escherichia urinary tract infection * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Vaginal infection * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	2	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	3
Injury, poisoning and procedural complications
Contusion * 1	1/15 (6.67%)	1	1/14 (7.14%)	1	1/15 (6.67%)	1	2/22 (9.09%)	3	0/21 (0.00%)	0	1/21 (4.76%)	1
Animal bite * 1	1/15 (6.67%)	1	1/14 (7.14%)	1	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	1/21 (4.76%)	1
Fall * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	2/21 (9.52%)	2	0/21 (0.00%)	0
Foot fracture * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	2
Limb injury * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Burns second degree * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Investigations
Amylase increased * 1	0/15 (0.00%)	0	2/14 (14.29%)	2	1/15 (6.67%)	1	1/22 (4.55%)	1	0/21 (0.00%)	0	2/21 (9.52%)	2
Lipase increased * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	2/15 (13.33%)	3	2/22 (9.09%)	2	0/21 (0.00%)	0	1/21 (4.76%)	1
Weight decreased * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Urine analysis abnormal * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Metabolism and nutrition disorders
Dehydration * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	1/15 (6.67%)	1	1/22 (4.55%)	1	0/21 (0.00%)	0	2/21 (9.52%)	2
Hyperglycaemia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Glucose tolerance impaired * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Hypokalaemia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	2	0/21 (0.00%)	0	0/21 (0.00%)	0
Weight fluctuation * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Hypoglycaemia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Musculoskeletal and connective tissue disorders
Muscle spasms * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	4/21 (19.05%)	4
Myalgia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	1/21 (4.76%)	1	4/21 (19.05%)	4
Muscular weakness * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	1/15 (6.67%)	1	1/22 (4.55%)	2	1/21 (4.76%)	1	1/21 (4.76%)	1
Muscle twitching * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	2/21 (9.52%)	2
Back pain * 1	1/15 (6.67%)	1	1/14 (7.14%)	1	1/15 (6.67%)	1	2/22 (9.09%)	4	0/21 (0.00%)	0	5/21 (23.81%)	6
Pain in extremity * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	2/21 (9.52%)	2	1/21 (4.76%)	3
Arthralgia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	2/22 (9.09%)	2	1/21 (4.76%)	1	3/21 (14.29%)	5
Tendonitis * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	1/21 (4.76%)	1	1/21 (4.76%)	1
Musculoskeletal chest pain * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Neck pain * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Joint swelling * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	2/15 (13.33%)	2	1/22 (4.55%)	1	1/21 (4.76%)	1	0/21 (0.00%)	0
Bursitis * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma * 1	0/15 (0.00%)	0	2/14 (14.29%)	2	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Basal cell carcinoma * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Nervous system disorders
Headache * 1	6/15 (40.00%)	6	3/14 (21.43%)	3	4/15 (26.67%)	5	10/22 (45.45%)	13	3/21 (14.29%)	4	4/21 (19.05%)	10
Dizziness * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	4/15 (26.67%)	4	4/22 (18.18%)	4	3/21 (14.29%)	3	3/21 (14.29%)	3
Paraesthesia * 1	1/15 (6.67%)	1	1/14 (7.14%)	1	0/15 (0.00%)	0	1/22 (4.55%)	3	0/21 (0.00%)	0	3/21 (14.29%)	3
Hypoaesthesia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	3	0/21 (0.00%)	0	2/21 (9.52%)	2
Worsening of Myasthenia Gravis * 1	2/15 (13.33%)	2	1/14 (7.14%)	1	2/15 (13.33%)	2	4/22 (18.18%)	6	0/21 (0.00%)	0	3/21 (14.29%)	3
Migraine with aura * 1	0/15 (0.00%)	0	1/14 (7.14%)	4	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	4
Somnolence * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Tremor * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Migraine * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	2/22 (9.09%)	2	0/21 (0.00%)	0	2/21 (9.52%)	2
Psychiatric disorders
Insomnia * 1	1/15 (6.67%)	1	1/14 (7.14%)	1	1/15 (6.67%)	1	2/22 (9.09%)	2	1/21 (4.76%)	1	1/21 (4.76%)	1
Depression * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	3/21 (14.29%)	3
Initial insomnia * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Sleep disorder * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Renal and urinary disorders
Nephrolithiasis * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	2/22 (9.09%)	2	0/21 (0.00%)	0	3/21 (14.29%)	3
Respiratory, thoracic and mediastinal disorders
Cough * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	3/22 (13.64%)	3	1/21 (4.76%)	1	5/21 (23.81%)	7
Dyspnoea * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	2/15 (13.33%)	2	1/22 (4.55%)	2	2/21 (9.52%)	2	1/21 (4.76%)	2
Nasal congestion * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	1/22 (4.55%)	2	0/21 (0.00%)	0	2/21 (9.52%)	2
Sleep apnoea syndrome * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Sinus congestion * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Pleurisy * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Skin and subcutaneous tissue disorders
Dermatitis contact * 1	1/15 (6.67%)	1	2/14 (14.29%)	2	1/15 (6.67%)	1	2/22 (9.09%)	4	0/21 (0.00%)	0	3/21 (14.29%)	3
Rash * 1	1/15 (6.67%)	1	1/14 (7.14%)	2	0/15 (0.00%)	0	1/22 (4.55%)	2	0/21 (0.00%)	0	2/21 (9.52%)	8
Miliaria * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	3/21 (14.29%)	3
Pruritus * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	2/15 (13.33%)	2	1/22 (4.55%)	1	0/21 (0.00%)	0	1/21 (4.76%)	1
Cold sweat * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Nail discolouration * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Nail disorder * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Onychoclasis * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Actinic keratosis * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
Erythema * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Skin exfoliation * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	0/22 (0.00%)	0	0/21 (0.00%)	0	0/21 (0.00%)	0
Skin mass * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	0/21 (0.00%)	0	0/21 (0.00%)	0
Vascular disorders
Hot flush * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	1/15 (6.67%)	1	1/22 (4.55%)	1	0/21 (0.00%)	0	1/21 (4.76%)	1
Cyanosis * 1	0/15 (0.00%)	0	1/14 (7.14%)	1	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	1/21 (4.76%)	1
Peripheral venous disease * 1	1/15 (6.67%)	1	0/14 (0.00%)	0	0/15 (0.00%)	0	1/22 (4.55%)	1	1/21 (4.76%)	1	0/21 (0.00%)	0
Hypotension * 1	0/15 (0.00%)	0	0/14 (0.00%)	0	0/15 (0.00%)	0	0/22 (0.00%)	0	0/21 (0.00%)	0	2/21 (9.52%)	2
1 Term from vocabulary, MedDRA v24.0; * Indicates events were collected by non-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: UCB
• Organization: Cares
• Phone: 001-844-599-2273
• EMail: UCBCares@ucb.com

Publications of Results:

Howard JF Jr, Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, Benatar M, Duda PW, MacDougall JE, Farzaneh-Far R, Kaminski HJ; Zilucoplan MG Study Group; Barohn R, Dimachkie M, Pasnoor M, Farmakidis C, Liu T, Colgan S, Benatar MG, Bertorini T, Pillai R, Henegar R, Bromberg M, Gibson S, Janecki T, Freimer M, Elsheikh B, Matisak P, Genge A, Guidon A, David W, Habib AA, Mathew V, Mozaffar T, Hinton JL, Hewitt W, Barnett D, Sullivan P, Ho D, Howard JF Jr, Traub RE, Chopra M, Kaminski HJ, Aly R, Bayat E, Abu-Rub M, Khan S, Lange D, Holzberg S, Khatri B, Lindman E, Olapo T, Sershon LM, Lisak RP, Bernitsas E, Jia K, Malik R, Lewis-Collins TD, Nicolle M, Nowak RJ, Sharma A, Roy B, Nye J, Pulley M, Berger A, Shabbir Y, Sachdev A, Patterson K, Siddiqi Z, Sivak M, Bratton J, Small G, Kohli A, Fetter M, Vu T, Lam L, Harvey B, Wolfe GI, Silvestri N, Patrick K, Zakalik K, Duda PW, MacDougall J, Farzaneh-Far R, Pontius A, Hoarty M. Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial. JAMA Neurol. 2020 May 1;77(5):582-592. doi: 10.1001/jamaneurol.2019.5125.

• Responsible Party: UCB Pharma ( Ra Pharmaceuticals )
• ClinicalTrials.gov Identifier: NCT03315130
• Other Study ID Numbers: RA101495-02.201
• First Submitted: October 16, 2017
• First Posted: October 19, 2017
• Results First Submitted: May 31, 2022
• Results First Posted: June 27, 2022
• Last Update Posted: July 27, 2022