A Study of NIDO-361 in Patients With SBMA

NCT06411912 | Completed Phase 2 Results DMC

• 1 other identifier: NIDO-361-002
• Study Type: interventional
• Target: 54
• Locations: 4 countries
• Sites: 5

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).

Conditions

Kennedy's Disease; Spinal and Bulbar Muscular Atrophy

Outcome Measures

Primary Outcomes (4)

• 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 C-muscles have a muscle fat content where more than 50% of the muscle tissue has been replaced by fat and most of the functional capacity is likely lost.

  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.

  Through study completion, an average of 1 year

• 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

  Adverse events were coded with MedDRA Dictionary Version 26.1.

  Through study completion, an average of 1 year

Secondary Outcomes (10)

• Change From Baseline to Day 180 and Day 360 in Modified-SBMAFRS (m-SBMAFRS)

  Baseline, Day 180, Day 360

• Change From Baseline to Day 180 and Day 360 in Two-Minute Walk Test (2MWT)

  Baseline, Day 180, Day 360

• Change From Baseline to Day 180 and Day 360 in 6-Minute Walk Test (6MWT)

  Baseline, Day 180, Day 360

• Change From Baseline to Day 180 and Day 360 in Timed Up and Go (TUG) Test

  Baseline, Day 180, Day 360

• Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Non-Sedentary Behavior

  Baseline, Treatment (Days 331 to 360)

Study Arms (2)

NIDO-361

EXPERIMENTAL

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

Drug: NIDO-361

Placebo

PLACEBO COMPARATOR

Participants receive matched dose placebo given orally once daily for 12 months.

Drug: Placebo

Interventions

NIDO-361 DRUG

Tablets containing 100mg of NIDO-361 for oral administration.

NIDO-361

Placebo DRUG

Placebo oral tablets

Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Ambulatory male
• Documented SBMA diagnosis confirmed by DNA genetic testing
• Able to complete six-minute walk test (6MWT)

You may not qualify if:

• 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)

Sponsors & Collaborators

• Nido Biosciences, Inc.: lead

Study Sites (5)

Rigshospitalet, Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center

Copenhagen, Denmark

Location

IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

Location

Azienda Ospedale Università di Padova

Padua, Italy

Location

Kyungpook National University Chilgok Hospital

Daegu, South Korea

Location

University of College London Hospital (UCLH)

London, United Kingdom

Location

MeSH Terms

Conditions

Bulbo-Spinal Atrophy, X-Linked

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Director
• Organization: Nido Biosciences, Inc.

clinicaltrials@nidobio.com

617-693-6819

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: May 8, 2024
• First Posted: May 13, 2024
• Study Start: March 1, 2024
• Primary Completion: October 22, 2025
• Study Completion: October 22, 2025
• Last Updated: March 3, 2026
• Results First Posted: March 3, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

IT (2); KR (1); DK (1); GB (1)