A Study of NIDO-361 in Patients With SBMA

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)

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).

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)