A Study of RVT-1401 in Myasthenia Gravis (MG) Patients | NCT03863080

Label	Type	Description	Intervention Names
Regimen A	Experimental	RVT-1401 680 mg weekly for 6 weeks + optional open-label extension (RVT-1401, 340 mg every 2 weeks for 6 weeks)	Drug: RVT-1401
Regimen B	Experimental	RVT-1401 340 mg weekly for 6 weeks + optional open-label extension (RVT-1401, 340 mg every 2 weeks for 6 weeks)	Drug: RVT-1401
Placebo	Placebo Comparator	Placebo for 6 weeks + optional open-label extension (RVT-1401, 340 mg every 2 weeks for 6 weeks)	Drug: RVT-1401 Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
RVT-1401	Drug	Subcutaneous administration of RVT-1401	Placebo Regimen A Regimen B

Measure	Description	Time Frame
Double-Blind Treatment Period: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition.	Up to Week 18
Open-Label Extension Period: Number of Participants Reporting AEs and SAEs	An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs are defined as those AEs that started or worsened in severity after the initiation of study drug administration. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition.	Up to Week 18
Double-Blind Treatment Period: Number of Participants With Clinically Significant Changes in Vital Signs	Vital signs including systolic blood pressure (SBP), diastolic blood pressure (DBP), pulse rate and temperature were measured after resting for at least 5 minutes in a semi-supine position.	Up to Week 7
Open-label Extension Period: Number of Participants With Clinically Significant Changes in Vital Signs

Measure	Description	Time Frame
Double-Blind Treatment Period: Area Under the Concentration-time Curve From Time 0 to 168 Hours (AUC0-168h) of RVT-1401	Blood samples were collected for the analysis of Pharmacokinetic parameter AUC (0-168h).	Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8

Facility	Status	City	State	ZIP	Country	Contacts
IMC/Diagnostic and Medical Clinic		Mobile	Alabama	36604	United States
Phoenix Neurological Associates

ID	Title	Description
FG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W),x 3 doses followed by a 6-week follow-up period where the participant did not receive any study treatment.

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Apr 10, 2019	Oct 5, 2023

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Double-Blind Treatment Period: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition.

Safety Analysis Set.

Posted

Count of Participants

Participants

Up to Week 18

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Units	Counts
Participants	OG0006 OG0015 OG0026

Title

Denominators

Categories

Any AE

Title	Measurements
	OG0005 OG0014 OG0025

Any SAE

Primary

Open-Label Extension Period: Number of Participants Reporting AEs and SAEs

An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs are defined as those AEs that started or worsened in severity after the initiation of study drug administration. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition.

Open-label Extension (OLE) Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Posted

Count of Participants

Participants

Up to Week 18

ID	Title	Description
OG000	Open-Label Extension Period: RVT-1401 340 mg Biweekly	Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Primary

Double-Blind Treatment Period: Number of Participants With Clinically Significant Changes in Vital Signs

Vital signs including systolic blood pressure (SBP), diastolic blood pressure (DBP), pulse rate and temperature were measured after resting for at least 5 minutes in a semi-supine position.

Safety Analysis Set.

Posted

Count of Participants

Participants

Up to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Primary

Open-label Extension Period: Number of Participants With Clinically Significant Changes in Vital Signs

Vital signs including SBP, DBP, pulse rate and temperature were measured after resting for at least 5 minutes in a semi-supine position.

OLE Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Posted

Count of Participants

Participants

Up to Week 18

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week

Primary

Double-blind Treatment Period: Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters

Clinical laboratory parameters included clinical chemistry, hematology and urinalysis.

Safety Analysis Set.

Posted

Count of Participants

Participants

Up to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Primary

Open-label Extension Period: Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters

Clinical laboratory parameters included clinical chemistry, hematology and urinalysis.

OLE Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Posted

Count of Participants

Participants

Up to Week 18

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week

Primary

Double-Blind Treatment Period: Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG)

Twelve-lead ECG was performed after 5 minutes of rest in the supine position.

Safety Analysis Set.

Posted

Count of Participants

Participants

Up to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Primary

Open-label Extension Period: Number of Participants With Clinically Significant Changes in ECG

Twelve-lead ECG was performed after 5 minutes of rest in the supine position.

OLE Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Posted

Count of Participants

Participants

Up to Week 18

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Primary

Double-blind Treatment Period: Percent Change From Baseline in Levels of Total Immunoglobulin G (IgG)

Serum samples were collected for the analysis of total immunoglobulin G. Percent change from Baseline was calculated as the mean value at the specified time frame (Week 7) minus the Baseline value, divided by the Baseline value x 100.

Full Analysis Set comprised of all randomized participants who received at least 1 dose of randomized study medication with at least 1 valid post-treatment value. Only those participants with data available at specified time points has been presented.

Posted

Least Squares Mean

Standard Error

Percent change

Baseline (Day 1) and Up to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Primary

Double-blind Treatment Period: Percent Change From Baseline in IgG Subclasses 1, 2, 3 and 4

Serum samples were collected for the analysis of IgG 1, 2, 3 and 4 levels. Percent change from Baseline was calculated as the mean value at the specified time frame (Week 7) minus the Baseline value, divided by the Baseline value x 100.

Full Analysis Set. Only those participants with data available at specified time points has been presented.

Posted

Least Squares Mean

Standard Error

Percent change

Baseline (Day 1) and Up to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week

Primary

Double-Blind Treatment Period: Percent Change From Baseline in Anti-acetylcholine Receptor Immunoglobulin G (Anti-AChR-IgG) at Week 7

Serum samples were collected for the analysis of Anti-AChR-IgG. Percent change from Baseline was calculated as the mean value at the specified time frame (Week 7) minus the Baseline value, divided by the Baseline value x 100.

Full Analysis Set. Only those participants with data available at definite timepoints has been presented.

Posted

Least Squares Mean

Standard Error

Percent change

Baseline (Day 1) and Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received an additional 3 doses of 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002

Secondary

Double-Blind Treatment Period: Area Under the Concentration-time Curve From Time 0 to 168 Hours (AUC0-168h) of RVT-1401

Blood samples were collected for the analysis of Pharmacokinetic parameter AUC (0-168h).

Pharmacokinetic (PK) Analysis Set comprised of all participants who underwent PK sampling and had evaluable concentration-time data for analysis. As PK analyses were not conducted, a PK Analysis Set was not presented.

Posted

Geometric Mean

Geometric Coefficient of Variation

Hours*microgram per milliliter

Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8

ID	Title	Description
OG000	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Secondary

Open-label Extension Period: AUC0-168h of RVT-1401

Pharmacokinetic parameters were not estimated because the sparse PK sampling schedule did not allow for accurate estimates of these parameters.

Pharmacokinetic Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Posted

Geometric Mean

Geometric Coefficient of Variation

Hours*microgram per milliliter

Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8, Week 9, Week 12 and Week 14

ID	Title	Description
OG000	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Secondary

Double-Blind Treatment Period: Maximum Concentration (Cmax) of RVT-1401

Blood samples were collected for the analysis of Pharmacokinetic parameter Cmax.

Pharmacokinetic Analysis Set.

Posted

Geometric Mean

Geometric Coefficient of Variation

micrograms per milliliter

Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8

ID	Title	Description
OG000	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Units	Counts

Secondary

Open-label Extension Period: Cmax of RVT-1401

Blood samples were collected for the analysis of Pharmacokinetic parameter Cmax.

Pharmacokinetic Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Posted

Geometric Mean

Geometric Coefficient of Variation

Micrograms per milliliter

Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8, Week 9, Week 12 and Week 14

ID	Title	Description
OG000	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Secondary

Double-Blind Treatment Period: Trough Concentrations (Ctrough) of RVT-1401

Blood samples were planned to be collected at indicated time points to measure the concentration of RVT-1401 pre-dose (Ctrough) as an assessment of the pharmacokinetic RVT-1401.

Pharmacokinetic Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Posted

Mean

Standard Deviation

Milligrams per liter

Pre-dose

ID	Title	Description
OG000	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Secondary

Open-label Extension Period: Ctrough of RVT-1401

Blood samples were planned to be collected at indicated time points to measure the concentration of RVT-1401 pre-dose (Ctrough) as an assessment of the pharmacokinetic RVT-1401.

Pharmacokinetic Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Posted

Mean

Standard Deviation

Milligrams per liter

Pre-dose

ID	Title	Description
OG000	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Units	Counts

Secondary

Double-blind Treatment Period: Change From Baseline in the Quantitative Myasthenia Gravis Score (QMG) Score

The QMG score is a physician-reported outcome measure was used to assess MG disease severity and pattern of deficits based on quantitative testing of affected muscle groups. The scale comprised of 13 test items that were graded on a scale of 0 (no myasthenic findings) to 3 (maximal myasthenic deficits). The total sum across all 13 items represents the QMG score. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Full Analysis Set. Only those participants with data available at specified time points has been represented (represented by n=X in the category titles).

Posted

Mean

Standard Deviation

Scores on a scale

Baseline (Day 1) and at Week 2, Week 3, Week 4, Week 5, Week 6 (Day 36) and Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week

Secondary

Double-Blind Treatment Period: Percentage of Participants With an Improvement/ Response on the QMG Score From Baseline

The response is defined as improvement from Baseline on the QMG score by => 3 points. The QMG score is a physician-reported outcome measure was used to assess MG disease severity and pattern of deficits based on quantitative testing of affected muscle groups. The scale comprised of 13 test items that were graded on a scale of 0 (no myasthenic findings) to 3 (maximal myasthenic deficits). The total sum across all 13 items represents the QMG score. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Full Analysis Set. Only those participants with data available at specified time points has been presented.

Posted

Number

Percentage of participants

Baseline (Day 1) to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week

Secondary

Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Score

The MG-ADL is an 8-item, participant-reported outcome measure that assessed Myasthenia Gravis symptoms and their effects on activities of daily living, with each response graded from 0 (normal) to 3 (most severe). The MG-ADL score was calculated by totaling the rating for each of the 8 items. Total MG-ADL scores range from 0 to 24 with higher scores indicating more severe disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Full Analysis Set. Only those participants with data available at specified time points has been presented.

Posted

Mean

Standard Deviation

Scores on a scale

Baseline (Day 1) and at Week 2, Week 3, Week 4, Week 5, Week 6 (Day 36), Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Secondary

Double-Blind Treatment Period: Percentage of Participants With an Improvement/ Response on the MG-ADL Score

The response was defined as improvement (decrease) from Baseline on the MG-ADL score by => 2 points. The MG-ADL is an 8-item, participant-reported outcome measure that assessed Myasthenia Gravis symptoms and their effects on activities of daily living, with each response graded from 0 (normal) to 3 (most severe). The MG-ADL score was calculated by totaling the rating for each of the 8 items. Total MG-ADL scores range from 0 to 24 with higher scores indicating more severe disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug.

Full Analysis Set. Only those participants with data available at specified time points has been presented.

Posted

Number

Percentage of participants

Baseline (Day 1) to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Secondary

Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Composite Score (MGC) Score

The MGC was developed by selecting the best performing items from 3 commonly used Myasthenia Gravis specific scales (QMG, Myasthenia Gravis manual muscle test, and MG-ADL) and is comprised of 10 functional domains: 3 ocular, 3 bulbar, 1 respiratory, 1 neck, and 2 limb items. The total score ranges from 0 (no myasthenic findings) to 50 (maximal myasthenic deficits). The scale measures symptoms and signs of MG in these domains incorporating both physician and participant-reported test items. Higher scores correlate with clinical worsening of the disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Full Analysis Set. Only those participants with data available at specified timepoints has been presented.

Posted

Mean

Standard Deviation

Scores on a scale

Baseline (Day 1) and at Week 2, Week 3, Week 4, Week 5, Week 6 (Day 36), Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week

Secondary

Double-Blind Treatment Period: Percentage of Participants With an Improvement on the MGC Score

The response was defined as improvement (decrease) from Baseline on the MGC score by => 3 points. The MGC was developed by selecting the best performing items from 3 commonly used Myasthenia Gravis specific scales (QMG, Myasthenia Gravis manual muscle test, and MG-ADL) and is comprised of 10 functional domains: 3 ocular, 3 bulbar, 1 respiratory, 1 neck, and 2 limb items. The total score ranges from 0 (no myasthenic findings) to 50 (maximal myasthenic deficits). The scale measures symptoms and signs of MG in these domains incorporating both physician and participant-reported test items. Higher scores correlate with clinical worsening of the disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug.

Full Analysis Set. Only those participants with data available at specified time points has been presented.

Posted

Number

Percentage of participants

Baseline (Day 1) to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week

Secondary

Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Quality of Life 15 Revised Score (MG-QOL 15r) Score

The MG-QOL15r is a participant-reported questionnaire designed to assess how a participant's Myasthenia Gravis affects different aspects related to their quality of life. The scale includes 15 items that are graded on a scale of 0 to2; the total across is the sum of all 15 items and represents the MG-QOL15r score. The range of the MG-QOL15r score is 0 - 30. Higher scores indicate worse outcomes. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Full Analysis Set. Only those participants with data available at specified time points has been presented.

Posted

Mean

Standard Deviation

Scores on a scale

Baseline (Day 1) and at Week 4 and Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.

Secondary

Double-Blind Treatment Period: Number of Participants Reporting Confirmed Positive Anti-RVT-1401 Antibodies

The serum levels of anti-RVT-1401 antibodies were determined. All samples that were potentially positive were analyzed with the confirmation assay where presence of anti-RVT-1401 was confirmed; the therapeutic antibody was used to compete with the analytical responses of anti-drug antibody (ADA) to assess specificity of screened positive samples.

Full Analysis Set.

Posted

Count of Participants

Participants

Up to Week 7

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week

Secondary

Open-Label Extension Period: Number of Participants Reporting Confirmed Positive Anti-RVT-1401 Antibodies

The serum levels of anti-RVT-1401 antibodies were determined. All samples that were potentially positive were analyzed with the confirmation assay where presence of anti-RVT-1401 was confirmed; the therapeutic antibody was used to compete with the analytical responses of ADA to assess specificity of screened positive samples.

Full Analysis Set.

Posted

Count of Participants

Participants

Up to Week 18

ID	Title	Description
OG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
OG002	Double-blind Treatment Period: RVT-1401 680 mg/Week

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment. Participants	6	0	6	5	6
EG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment. Participants	5	0	5	4	5
EG002	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment. Participants	6	1	6	5	6
EG003	Open-label Extension Period: RVT-1401 340 mg Biweekly	Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	15	1	15	10	15

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Herpes simplex	Infections and infestations	MedDRA 21.1	Non-systematic Assessment		EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG0030 affected15 at risk
Myasthenia gravis	Nervous system disorders	MedDRA 21.1	Non-systematic Assessment		EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Gingival pain	Gastrointestinal disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG0030 affected15 at risk
Anal incontinence	Gastrointestinal disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Constipation	Gastrointestinal disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Diarrhoea	Gastrointestinal disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Nausea	Gastrointestinal disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Injection site erythema	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0012 affected5 at risk EG0021 affected6 at risk EG003
Injection site bruising	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0011 affected5 at risk EG0020 affected6 at risk EG003
Injection site haemorrhage	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003
Injection site swelling	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0011 affected5 at risk EG0020 affected6 at risk EG003
Vessel puncture site bruise	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003
Asthenia	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Injection site pain	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Facial pain	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Fatigue	General disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Upper respiratory tract infection	Infections and infestations	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0011 affected5 at risk EG0020 affected6 at risk EG003
Urinary tract infection	Infections and infestations	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0011 affected5 at risk EG0020 affected6 at risk EG003
Viral upper respiratory tract infection	Infections and infestations	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003
Tonsillitis	Infections and infestations	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003
Fall	Injury, poisoning and procedural complications	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Red blood cell count increased	Investigations	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0011 affected5 at risk EG0020 affected6 at risk EG003
White blood cells urine positive	Investigations	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0011 affected5 at risk EG0020 affected6 at risk EG003
Total complement activity decreased	Investigations	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Decreased appetite	Metabolism and nutrition disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003
Muscle spasms	Musculoskeletal and connective tissue disorders	MedDRA 21.1	Non-systematic Assessment	EG0002 affected6 at risk EG0011 affected5 at risk EG0020 affected6 at risk EG003
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003
Muscular weakness	Musculoskeletal and connective tissue disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Dizziness	Nervous system disorders	MedDRA 21.1	Non-systematic Assessment	EG0001 affected6 at risk EG0010 affected5 at risk EG0022 affected6 at risk EG003
Headache	Nervous system disorders	MedDRA 21.1	Non-systematic Assessment	EG0003 affected6 at risk EG0011 affected5 at risk EG0020 affected6 at risk EG003
Myasthenia gravis	Nervous system disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Ataxia	Nervous system disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Rash	Skin and subcutaneous tissue disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003
Upper-airway cough syndrome	Respiratory, thoracic and mediastinal disorders	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0020 affected6 at risk EG003
Herpes simplex	Infections and infestations	MedDRA 21.1	Non-systematic Assessment	EG0000 affected6 at risk EG0010 affected5 at risk EG0021 affected6 at risk EG003

ID	Term
D020361	Paraneoplastic Syndromes, Nervous System
D009423	Nervous System Neoplasms
D009371	Neoplasms by Site
D009369	Neoplasms
D010257	Paraneoplastic Syndromes
D020274	Autoimmune Diseases of the Nervous System
D009422	Nervous System Diseases
D019636	Neurodegenerative Diseases
D020511	Neuromuscular Junction Diseases
D009468	Neuromuscular Diseases
D001327	Autoimmune Diseases
D007154	Immune System Diseases

ID	Title	Description
BG000	Double-blind Treatment Period: Placebo	Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
BG001	Double-blind Treatment Period: RVT-1401 340 mg/Week	Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment. Participants
BG002	Double-blind Treatment Period: RVT-1401 680 mg/Week	Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
BG003	Total	Total of all reporting groups