A Study of NIDO-361 in Patients With SBMA

NCT ID: NCT06411912

Last Updated: 2025-10-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

54 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-01

Study Completion Date

2025-10-22

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Spinal and Bulbar Muscular Atrophy Kennedy's Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Patients will be randomly assigned to receive NIDO-361 or placebo at the recommended Phase 2 dose (RP2D) of 100 mg once daily. Patients will receive daily oral doses of NIDO-361 or placebo for 12 months.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

NIDO-361

Participants receive 100 mg of NIDO-361 given orally once daily.

Group Type EXPERIMENTAL

NIDO-361

Intervention Type DRUG

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

Placebo

Participants receive matched dose placebo given orally once daily.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo oral tablets

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

NIDO-361

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

Intervention Type DRUG

Placebo

Placebo oral tablets

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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)
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Nido Biosciences, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

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

Copenhagen, , Denmark

Site Status

IRCCS Istituto Neurologico Carlo Besta

Milan, , Italy

Site Status

Azienda Ospedale Università di Padova

Padua, , Italy

Site Status

Kyungpook National University Chilgok Hospital

Daegu, , South Korea

Site Status

University of College London Hospital (UCLH)

London, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Denmark Italy South Korea United Kingdom

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

NIDO-361-002

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

A Trial of Hydroxyurea in Spinal Muscular Atrophy
NCT00485511 COMPLETED PHASE2/PHASE3
Spinraza in Adult Spinal Muscular Atrophy
NCT03709784 ACTIVE_NOT_RECRUITING