The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
54
Rigshospitalet, Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center
Copenhagen, Denmark
IRCCS Istituto Neurologico Carlo Besta
Milan, Italy
Azienda Ospedale Università di Padova
Padova, Italy
Kyungpook National University Chilgok Hospital
Daegu, South Korea
Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C]
Analysis of thigh included adductors, hamstrings, and quadriceps and total (whole-body) included muscles in the rotator cuff, arm, torso, and leg muscle regions (38 muscles in total). Muscles were categorized according to their fat content as follows: A-muscles were defined as muscle fat fraction (MFF) \< 50% and muscle fat infiltration (MFI) \< 10%. A-muscles have a low or slightly elevated fat content to a level that can be present in individuals without a neuromuscular disease. B-muscles were defined as MFF \< 50% and MFI ≥ 10%. B-muscles have a muscle fat content at a level where disease involvement is likely. C-muscles were defined as MFF ≥ 50%. C-muscles have a muscle fat content where more than 50% of the muscle tissue has been replaced by fat and most of the functional capacity is likely lost.
Time frame: Baseline, Day 180, Days 360-374 (Last Visit)
Number of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)
Adverse events were coded with MedDRA Dictionary Version 26.1.
Time frame: Through study completion, an average of 1 year
Number of Participants Discontinuing Study Drug and Number of Participants' Deaths
Time frame: Through study completion, an average of 1 year
Total Number of Mild, Moderate, and Severe Adverse Events (AEs) Across All Participants
Adverse events were coded with MedDRA Dictionary Version 26.1.
Time frame: Through study completion, an average of 1 year
Change From Baseline to Day 180 and Day 360 in Modified-SBMAFRS (m-SBMAFRS)
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).
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University of College London Hospital (UCLH)
London, United Kingdom
Time frame: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in Two-Minute Walk Test (2MWT)
2-minute walk test (2MWT) measures the distance traveled (meters) in 2 minutes.
Time frame: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in 6-Minute Walk Test (6MWT)
6-minute walk test (6MWT) measures the distance traveled (meters) in 6 minutes.
Time frame: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in Timed Up and Go (TUG) Test
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.
Time frame: Baseline, Day 180, Day 360
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Non-Sedentary Behavior
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.
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: 95th Percentile of 6-Minute Physical Activity Windows
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.
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Acitgraphy-Derived Measurement: Maximum Walking Bout Duration
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.
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Peak 95th Percentile Cadence
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.
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy Derived Measurement: Average Breathing Rate
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.
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Day 180 and Day 360 in Grip Strength as Measured by Handheld Dynamometer (HHD)
Grip strength (lbs) is averaged across three trials for the right and left hands separately using a handheld dynamometer (HHD).
Time frame: Baseline, Day 180, Day 360