Phase 2
N=54
A Study of NIDO-361 in Patients With SBMA
Spinal and Bulbar Muscular Atrophy · Kennedy's Disease
Bottom Line
View on ClinicalTrials.gov: NCT06411912 ↗Enrolled (actual)
54
Serious AEs
11.1%
Results posted
Mar 2026
Primary outcome: Primary: Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C] — 4.1402; 3.8580; -0.0871; -0.1042 cm³
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- NIDO-361 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Nido Biosciences, Inc.
- Primary completion
- Oct 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C] |
4.1402; 3.8580; -0.0871; -0.1042; -0.1363; -0.1511 | — |
| PRIMARY Number of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) |
25; 26; 9; 17; 3; 3 | — |
| PRIMARY Number of Participants Discontinuing Study Drug and Number of Participants' Deaths |
0; 1; 0; 0 | — |
| PRIMARY Total Number of Mild, Moderate, and Severe Adverse Events (AEs) Across All Participants |
65; 98; 27; 36; 2; 10 | — |
| SECONDARY Change From Baseline to Day 180 and Day 360 in Modified-SBMAFRS (m-SBMAFRS) |
15.9; 15.2; 0.7; 0.5; 1.2; 0.1 | — |
| SECONDARY Change From Baseline to Day 180 and Day 360 in Two-Minute Walk Test (2MWT) |
128.4; 112.8; -0.4; 4.5; -1.8; 3.3 | — |
| SECONDARY Change From Baseline to Day 180 and Day 360 in 6-Minute Walk Test (6MWT) |
363.3; 314.2; 1.5; 13.7; -4.6; 8.9 | — |
| SECONDARY Change From Baseline to Day 180 and Day 360 in Timed Up and Go (TUG) Test |
9.927; 11.859; 0.133; -0.044; -0.179; 0.126 | — |
| SECONDARY Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Non-Sedentary Behavior |
231.824; 262.087; -19.569; -39.777 | — |
| SECONDARY Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: 95th Percentile of 6-Minute Physical Activity Windows |
31293.3393; 30905.5742; -1517.7024; -1091.4469 | — |
| SECONDARY Change From Baseline to Treatment (Days 331 to 360) in Acitgraphy-Derived Measurement: Maximum Walking Bout Duration |
268.0350; 184.3490; 17.1187; -47.2082 | — |
| SECONDARY Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Peak 95th Percentile Cadence |
168.637; 166.144; -2.382; 2.382 | — |
| SECONDARY Change From Baseline to Treatment (Days 331 to 360) in Actigraphy Derived Measurement: Average Breathing Rate |
16.856; 16.428; 0.433; -0.278 | — |
| SECONDARY Change From Baseline to Day 180 and Day 360 in Grip Strength as Measured by Handheld Dynamometer (HHD) |
44.66; 40.21; -0.74; -2.00; -1.36; -2.02 | — |
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).
Eligibility Criteria
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)
Data sourced from ClinicalTrials.gov (NCT06411912). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.