A Study of NIDO-361 in Patients With SBMA

Phase 2CompletedNCT06411912

Nido Biosciences, Inc.54 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Spinal and Bulbar Muscular Atrophy Kennedy's Disease

Interventions

NIDO-361DRUG

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

PlaceboDRUG

Placebo oral tablets

Eligibility

Sex: MALEMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

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)

Locations (5)

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

Copenhagen, Denmark

IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

Azienda Ospedale Università di Padova

Padua, Italy

Kyungpook National University Chilgok Hospital

Daegu, South Korea

Outcomes

Primary Outcomes

Change from baseline in thigh and total lean muscle volume as assessed by whole-body MRI

Time frame: Through study completion, an average of 1 year

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.

Time frame: Through study completion, an average of 1 year

Data from ClinicalTrials.gov

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