New drug shows promise for rare Muscle-Wasting disease
NCT ID NCT06411912
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested an experimental drug called NIDO-361 in 54 men with spinal and bulbar muscular atrophy (SBMA), also known as Kennedy's disease. The goal was to see if the drug is safe and can help maintain or improve muscle mass. Participants received either the drug or a placebo, and researchers measured changes in muscle volume and tracked any side effects over about a year.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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54 people
The number who actually took part.
- Started
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Mar 2024
- Finished
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Oct 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 70 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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)
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Azienda Ospedale Università di Padova
Padova, Italy
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IRCCS Istituto Neurologico Carlo Besta
Milan, Italy
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Kyungpook National University Chilgok Hospital
Daegu, South Korea
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Rigshospitalet, Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center
Copenhagen, Denmark
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University of College London Hospital (UCLH)
London, United Kingdom
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Other studies related to the condition(s) this trial covers.