A Study of NIDO-361 in Patients With SBMA (PIONEER KD)

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).

Study Overview

• Status: Completed
• Conditions: Spinal and Bulbar Muscular Atrophy; Kennedy's Disease
• Intervention / Treatment: Drug: Placebo; Drug: NIDO-361

• Study Type: Interventional
• Enrollment (Actual): 54
• Phase: Phase 2

Contacts and Locations

Study Locations

• Denmark
  • Copenhagen, Denmark
    • Rigshospitalet, Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center
• Italy
  • Milan, Italy
    • IRCCS Istituto Neurologico Carlo Besta
  • Padua, Italy
    • Azienda Ospedale Universita Di Padova
• South Korea
  • Daegu, South Korea
    • Kyungpook National University Chilgok Hospital
• United Kingdom
  • London, United Kingdom
    • University of College London Hospital (UCLH)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NIDO-361; Participants receive NIDO-361, 100 mg for at least 2 months and 200 mg for the remainder of the study.	Drug: NIDO-361; Tablets containing 100mg of NIDO-361 for oral administration.
Placebo Comparator: Placebo; Participants receive matched dose placebo given orally once daily for 12 months.	Drug: Placebo; Placebo oral tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	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.	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.	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.	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.	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.	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.	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.	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.	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).	Baseline, Day 180, Day 360

Collaborators and Investigators

• Sponsor: Nido Biosciences, Inc.

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2024
• Primary Completion (Actual): October 22, 2025
• Study Completion (Actual): October 22, 2025

Study Registration Dates

• First Submitted: May 8, 2024
• First Submitted That Met QC Criteria: May 10, 2024
• First Posted (Actual): May 13, 2024

Study Record Updates

• Last Update Posted (Actual): March 3, 2026
• Last Update Submitted That Met QC Criteria: March 1, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Genetic Diseases, X-Linked; Spinal Cord Diseases; Motor Neuron Disease; Muscular Atrophy, Spinal; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Bulbo-Spinal Atrophy, X-Linked
• Other Study ID Numbers: NIDO-361-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No