Kennedy's Disease, Spinal and Bulbar Muscular Atrophy
Conditions
Brief summary
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).
Interventions
DRUGNIDO-361
Tablets containing 100mg of NIDO-361 for oral administration.
DRUGPlacebo
Placebo oral tablets
Sponsors
Nido Biosciences, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
Participants will receive NIDO-361, 100 mg for at least 2 months and 200 mg for the remainder of the study or placebo administered orally once daily for 12 months.
Eligibility
Sex/Gender
MALE
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Ambulatory male * Documented SBMA diagnosis confirmed by DNA genetic testing * Able to complete six-minute walk test (6MWT)
Exclusion criteria
* Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, immunologic, malignant, metabolic, psychiatric, or other condition that, in the opinion of the Investigator, precludes the participant's safe participation in the study or would interfere with the study assessments * Inability to undergo MRI (mild sedation may be allowed)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C] | Baseline, Day 180, Days 360-374 (Last Visit) | 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 C-muscles have a muscle fat content where more than 50% of the muscle tissue has been replaced by fat and most of the functional capacity is likely lost. |
| Number of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) | Through study completion, an average of 1 year | Adverse events were coded with MedDRA Dictionary Version 26.1. |
| Number of Participants Discontinuing Study Drug and Number of Participants' Deaths | Through study completion, an average of 1 year | — |
| Total Number of Mild, Moderate, and Severe Adverse Events (AEs) Across All Participants | Through study completion, an average of 1 year | Adverse events were coded with MedDRA Dictionary Version 26.1. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline to Day 180 and Day 360 in Modified-SBMAFRS (m-SBMAFRS) | Baseline, Day 180, Day 360 | 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). |
| Change From Baseline to Day 180 and Day 360 in Two-Minute Walk Test (2MWT) | Baseline, Day 180, Day 360 | 2-minute walk test (2MWT) measures the distance traveled (meters) in 2 minutes. |
| Change From Baseline to Day 180 and Day 360 in 6-Minute Walk Test (6MWT) | Baseline, Day 180, Day 360 | 6-minute walk test (6MWT) measures the distance traveled (meters) in 6 minutes. |
| Change From Baseline to Day 180 and Day 360 in Timed Up and Go (TUG) Test | Baseline, Day 180, Day 360 | 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. |
| Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Non-Sedentary Behavior | Baseline, Treatment (Days 331 to 360) | 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. |
| Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: 95th Percentile of 6-Minute Physical Activity Windows | Baseline, Treatment (Days 331 to 360) | 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. |
| Change From Baseline to Treatment (Days 331 to 360) in Acitgraphy-Derived Measurement: Maximum Walking Bout Duration | Baseline, Treatment (Days 331 to 360) | 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. |
| Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Peak 95th Percentile Cadence | Baseline, Treatment (Days 331 to 360) | 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. |
| Change From Baseline to Treatment (Days 331 to 360) in Actigraphy Derived Measurement: Average Breathing Rate | Baseline, Treatment (Days 331 to 360) | 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. |
| Change From Baseline to Day 180 and Day 360 in Grip Strength as Measured by Handheld Dynamometer (HHD) | Baseline, Day 180, Day 360 | Grip strength (lbs) is averaged across three trials for the right and left hands separately using a handheld dynamometer (HHD). |
Countries
Denmark, Italy, South Korea, United Kingdom
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Customized Age Group at Informed Consent 18 to 44 years | 6 Participants |
| Age, Customized Age Group at Informed Consent 45 to 70 years | 24 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 27 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 11 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 18 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 26 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 26 | 0 / 28 |
| other Total, other adverse events | 25 / 26 | 26 / 28 |
| serious Total, serious adverse events | 3 / 26 | 3 / 28 |
Outcome results
None listed