2-minute walk test (2MWT) measures the distance traveled (meters) in 2 minutes.

6-minute walk test (6MWT) measures the distance traveled (meters) in 6 minutes.

The Timed Up and Go Test (TUG) is used to assess mobility and to provide an estimate on balance and risk of falling. Patients are asked to rise from a chair, walk 3 meters, turn around, return to the chair, and sit. The time that it takes to complete the task is recorded. Assistive devices, orthoses, and ankle braces are allowed.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

Grip strength (lbs) is averaged across three trials for the right and left hands separately using a handheld dynamometer (HHD).

Total of all reporting groups

Adverse events were coded with MedDRA Dictionary Version 26.1.

The safety analysis set was defined as all randomized patients, who received at least 1 dose of study treatment (placebo or NIDO-361). If a patient experienced more than 1 event in a given system organ class (SOC), that patient was counted once for the SOC. If a patient experienced more than 1 event with a given preferred term (PT), that patient was counted only once for that PT.

The modified Spinal and Bulbar Muscular Atrophy Functional Rating Scale (m-SBMAFRS) is calculated as the sum of the items for the truncal and lower-limb domains from the SBMAFRS, which is a validated functional rating scale developed from the Amyotrophic Lateral Sclerosis Functional Rating Scale. Each item has five responses options scoring 0 (worst) to 4 (normal). A higher score indicates better functioning. Total range is 0 (worst) to 30 (normal).

Patients, who received at least 1 dose of study treatment (placebo or NIDO-361) and had at least 1 post-baseline assessment of efficacy parameters, were considered included in analysis.

2-minute walk test (2MWT) measures the distance traveled (meters) in 2 minutes.

Patients, who received at least 1 dose of study treatment (placebo or NIDO-361) and had at least 1 post-baseline assessment of efficacy parameters, were considered included in analysis.

6-minute walk test (6MWT) measures the distance traveled (meters) in 6 minutes.

Patients, who received at least 1 dose of study treatment (placebo or NIDO-361) and had at least 1 post-baseline assessment of efficacy parameters, were considered included in analysis.

The Timed Up and Go Test (TUG) is used to assess mobility and to provide an estimate on balance and risk of falling. Patients are asked to rise from a chair, walk 3 meters, turn around, return to the chair, and sit. The time that it takes to complete the task is recorded. Assistive devices, orthoses, and ankle braces are allowed.

Patients, who received at least 1 dose of study treatment (placebo or NIDO-361) and had at least 1 post-baseline assessment of efficacy parameters, were considered included in analysis.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

A minimum daily wear time requirement of 16 hrs. was used to filter out days from the aggregation. Days that met the minimum wear requirement were considered valid days and days that did not meet this criterion were considered invalid days. For each time period, a minimum of 15 valid days were required for the time period to be considered valid. For valid time periods, the aggregation was performed over all valid days. The value for invalid time periods was set to missing.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

A minimum daily wear time requirement of 16 hrs. was used to filter out days from the aggregation. Days that met the minimum wear requirement were considered valid days and days that did not meet this criterion were considered invalid days. For each time period, a minimum of 15 valid days were required for the time period to be considered valid. For valid time periods, the aggregation was performed over all valid days. The value for invalid time periods was set to missing.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

A minimum daily wear time requirement of 16 hrs. was used to filter out days from the aggregation. Days that met the minimum wear requirement were considered valid days and days that did not meet this criterion were considered invalid days. For each time period, a minimum of 15 valid days were required for the time period to be considered valid. For valid time periods, the aggregation was performed over all valid days. The value for invalid time periods was set to missing.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

A minimum daily wear time requirement of 16 hrs. was used to filter out days from the aggregation. Days that met the minimum wear requirement were considered valid days and days that did not meet this criterion were considered invalid days. For each time period, a minimum of 15 valid days were required for the time period to be considered valid. For valid time periods, the aggregation was performed over all valid days. The value for invalid time periods was set to missing.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

A minimum daily wear time requirement of 16 hrs. was used to filter out days from the aggregation. Days that met the minimum wear requirement were considered valid days and days that did not meet this criterion were considered invalid days. For each time period, a minimum of 15 valid days were required for the time period to be considered valid. For valid time periods, the aggregation was performed over all valid days. The value for invalid time periods was set to missing.

Grip strength (lbs) is averaged across three trials for the right and left hands separately using a handheld dynamometer (HHD).

Patients, who received at least 1 dose of study treatment (placebo or NIDO-361) and had at least 1 post-baseline assessment of efficacy parameters, were considered included in analysis.

Adverse events were coded with MedDRA Dictionary Version 26.1.

The safety analysis set was defined as all randomized patients, who received at least 1 dose of study treatment (placebo or NIDO-361). If a patient experienced more than 1 event in a given system organ class (SOC), that patient was counted once for the SOC. If a patient experienced more than 1 event with a given preferred term (PT), that patient was counted only once for that PT.

Participants receive NIDO-361, 100 mg for at least 2 months and 200 mg for the remainder of the study.