A Study of NIDO-361 in Patients With SBMA

Phase 2

Active, not recruiting

• Conditions: Spinal and Bulbar Muscular Atrophy; Kennedy's Disease
• Interventions: Drug: NIDO-361; Drug: Placebo

• Registration Number: NCT06411912
• Lead Sponsor: Nido Biosciences, Inc.

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2024-03-01
• Study First Submitted: 2024-05-08
• Study First Submitted QC: 2024-05-10
• Study First Posted: 2024-05-13
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-23
• Last Update Posted: 2024-10-26
• Primary Completion: 2025-10-01
• Study Completion: 2025-10-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 54

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NIDO-361	NIDO-361	Participants receive 100 mg of NIDO-361 given orally once daily.
Placebo	Placebo	Participants receive matched dose placebo given orally once daily.

Primary Outcome Measures

Name	Time	Method
Number of patients with adverse events or serious adverse events. Number of patients discontinuing study and number of deaths. Number of mild, moderate, and severe adverse events.	Through study completion, an average of 1 year
Change from baseline in thigh and total lean muscle volume as assessed by whole-body MRI	Through study completion, an average of 1 year

Trial Locations

Locations (5)

Rigshospitalet, Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center; 🇩🇰 Copenhagen, Denmark

IRCCS Istituto Neurologico Carlo Besta; 🇮🇹 Milano, Italy

Azienda Ospedale Università di Padova; 🇮🇹 Padova, Italy

Kyungpook National University Chilgok Hospital; 🇰🇷 Daegu, Korea, Republic of

University of College London Hospital (UCLH); 🇬🇧 London, United Kingdom