A Study of NIDO-361 in Patients With SBMA

Spinal and Bulbar Muscular Atrophy Clinical Trial

— PIONEER KD  

Official title:

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06411912
• Other study ID #: NIDO-361-002
• Status: Recruiting
• Phase: Phase 2
• Start date: March 1, 2024
• Est. completion date: August 1, 2025

Study information

• Verified date: May 2024
• Source: Nido Biosciences, Inc.
• Contact: Nido Biosciences
• Phone: 617-693-6819
• Email: ClinicalTrials[@]nidobio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 54
• Est. completion date: August 1, 2025
• Est. primary completion date: August 1, 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 70 Years

Eligibility

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)

Related Conditions & MeSH terms

• Atrophy
• Bulbo-Spinal Atrophy, X-Linked
• Kennedy's Disease
• Muscular Atrophy
• Spinal and Bulbar Muscular Atrophy

Intervention

Drug:
• NIDO-361
Tablets containing 100mg of NIDO-361 for oral administration.
• Placebo
Placebo oral tablets

Locations

Country	Name	City	State
Denmark	Rigshospitalet, Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center	Copenhagen
Italy	IRCCS Istituto Neurologico Carlo Besta	Milano
Korea, Republic of	Kyungpook National University Chilgok Hospital	Daegu
United Kingdom	University of College London Hospital (UCLH)	London

Sponsors (1)

Lead Sponsor	Collaborator
Nido Biosciences, Inc.

Countries where clinical trial is conducted

Denmark,  Italy,  Korea, Republic of,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in thigh and total lean muscle volume as assessed by whole-body MRI		Through study completion, an average of 1 year
Primary	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

External Links

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