Spinal and Bulbar Muscular AtrophyPhase 2PIONEER KDNCT06411912

A Study of NIDO-361 in Patients With SBMA

A Phase 2 study testing NIDO-361 in people with Spinal and Bulbar Muscular Atrophy and Kennedy's Disease, run by Nido Biosciences, Inc. at 5 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

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). Read more about this study

Who is it for?: Ages 18 to 70 years, men only
Conditions the study is for: Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
What is being tested?: NIDO-361
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 54 participants
Where: 5 sitesin 4 countries
Study dates: 1 Mar 2024to 22 Oct 2025
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 70 years
Men only
No healthy volunteers
Adult, Older adult

Who may be able to join

Ambulatory male
Documented SBMA diagnosis confirmed by DNA genetic testing
Able to complete six-minute walk test (6MWT)

Who cannot join

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)

Trial locations and contacts

5 sites in 4 countries.

Country

City, postcode or hospital

5 sites in 4 countries

Italy2 sites

IRCCS Istituto Neurologico Carlo Besta
Milan
Azienda Ospedale Università di Padova
Padova

Other trials in Italy

Denmark1 site

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

Other trials in Denmark

South Korea1 site

Kyungpook National University Chilgok Hospital
Daegu

Other trials in South Korea

United Kingdom1 site

University of College London Hospital (UCLH)
London

Other trials in United Kingdom

About this study

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

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).

Conditions: Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
Being tested: NIDO-361, Placebo
Medical subject headings: Bulbo-Spinal Atrophy, X-Linked

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT06411912
Study group	Role	What they receive
NIDO-361	Experimental	Participants receive NIDO-361, 100 mg for at least 2 months and 200 mg for the remainder of the study. Receives: Drug: NIDO-361
Placebo	Placebo comparator	Participants receive matched dose placebo given orally once daily for 12 months. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C]
Analysis of thigh included adductors, hamstrings, and quadriceps and total (whole-body) included muscles in the rotator cuff, arm, torso, and leg muscle regions (38 muscles in total). Muscles were categorized according to their fat content as follows: A-muscles were defined as muscle fat fraction (MFF) < 50% and muscle fat infiltration (MFI) < 10%. A-muscles have a low or slightly elevated fat content to a level that can be present in individuals without a neuromuscular disease. B-muscles were defined as MFF < 50% and MFI ≥ 10%. B-muscles have a muscle fat content at a level where disease involvement is likely. C-muscles were defined as MFF ≥ 50%. C-muscles have a muscle fat content where more than 50% of the muscle tissue has been replaced by fat and most of the functional capacity is likely lost.
Measured: Baseline, Day 180, Days 360-374 (Last Visit)
Number of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)
Adverse events were coded with MedDRA Dictionary Version 26.1.
Measured: Through study completion, an average of 1 year
Number of Participants Discontinuing Study Drug and Number of Participants' Deaths
Measured: Through study completion, an average of 1 year
Total Number of Mild, Moderate, and Severe Adverse Events (AEs) Across All Participants
Adverse events were coded with MedDRA Dictionary Version 26.1.
Measured: Through study completion, an average of 1 year

Secondary outcomes (10)

Change From Baseline to Day 180 and Day 360 in Modified-SBMAFRS (m-SBMAFRS)
The modified Spinal and Bulbar Muscular Atrophy Functional Rating Scale (m-SBMAFRS) is calculated as the sum of the items for the truncal and lower-limb domains from the SBMAFRS, which is a validated functional rating scale developed from the Amyotrophic Lateral Sclerosis Functional Rating Scale. Each item has five responses options scoring 0 (worst) to 4 (normal). A higher score indicates better functioning. Total range is 0 (worst) to 30 (normal).
Measured: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in Two-Minute Walk Test (2MWT)
2-minute walk test (2MWT) measures the distance traveled (meters) in 2 minutes.
Measured: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in 6-Minute Walk Test (6MWT)
6-minute walk test (6MWT) measures the distance traveled (meters) in 6 minutes.
Measured: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in Timed Up and Go (TUG) Test
The Timed Up and Go Test (TUG) is used to assess mobility and to provide an estimate on balance and risk of falling. Patients are asked to rise from a chair, walk 3 meters, turn around, return to the chair, and sit. The time that it takes to complete the task is recorded. Assistive devices, orthoses, and ankle braces are allowed.
Measured: Baseline, Day 180, Day 360
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Non-Sedentary Behavior
The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.
Measured: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: 95th Percentile of 6-Minute Physical Activity Windows
The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.
Measured: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Acitgraphy-Derived Measurement: Maximum Walking Bout Duration
The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.
Measured: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Peak 95th Percentile Cadence
The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.
Measured: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy Derived Measurement: Average Breathing Rate
The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.
Measured: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Day 180 and Day 360 in Grip Strength as Measured by Handheld Dynamometer (HHD)
Grip strength (lbs) is averaged across three trials for the right and left hands separately using a handheld dynamometer (HHD).
Measured: Baseline, Day 180, Day 360

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Mar 202422 Oct 2025

First posted13 May 2024
Study start1 Mar 2024
Primary completion22 Oct 2025
Study completion22 Oct 2025

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Spinal and Bulbar Muscular AtrophyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: Participants will receive NIDO-361, 100 mg for at least 2 months and 200 mg for the remainder of the study or placebo administered orally once daily for 12 months.
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 54 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06411912
Sponsor's protocol ID: NIDO-361-002
Acronym: PIONEER KD
Status last verified: Mar 2026
First submitted: 8 May 2024
Last updated: 3 Mar 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 3 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
ClinicalTrials.gov ID: NCT06411912
Acronym / sponsor ID: PIONEER KD / NIDO-361-002
Conditions: Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 54 participants (actual)
Lead sponsor: Nido Biosciences, Inc.Industry (pharma, biotech, devices)
Study start: 1 Mar 2024 (actual)
Primary completion: 22 Oct 2025 (actual)
Study completion: 22 Oct 2025 (actual)
First posted: 13 May 2024 (actual)
Results: Posted 3 Mar 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2
Model description: Participants will receive NIDO-361, 100 mg for at least 2 months and 200 mg for the remainder of the study or placebo administered orally once daily for 12 months.

Arm	Type	Receives
NIDO-361	Experimental	Drug: NIDO-361Participants receive NIDO-361, 100 mg for at least 2 months and 200 mg for the remainder of the study.
Placebo	Placebo comparator	Drug: PlaceboParticipants receive matched dose placebo given orally once daily for 12 months.

Who may qualify

Ages: 18 to 70 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

Ambulatory male
Documented SBMA diagnosis confirmed by DNA genetic testing
Able to complete six-minute walk test (6MWT)

Key exclusion criteria (2 listed)

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)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
NIDO-361Tablets containing 100mg of NIDO-361 for oral administration.	Drug	NIDO-361
PlaceboPlacebo oral tablets	Drug	Placebo

Primary outcome

Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C]
Time frame: Baseline, Day 180, Days 360-374 (Last Visit)
Analysis of thigh included adductors, hamstrings, and quadriceps and total (whole-body) included muscles in the rotator cuff, arm, torso, and leg muscle regions (38 muscles in total). Muscles were categorized according to their fat content as follows: A-muscles were defined as muscle fat fraction (MFF) < 50% and muscle fat infiltration (MFI) < 10%. A-muscles have a low or slightly elevated fat content to a level that can be present in individuals without a neuromuscular disease. B-muscles were defined as MFF < 50% and MFI ≥ 10%. B-muscles have a muscle fat content at a level where disease involvement is likely. C-muscles were defined as MFF ≥ 50%. C-muscles have a muscle fat content where more than 50% of the muscle tissue has been replaced by fat and most of the functional capacity is likely lost.
Number of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)
Time frame: Through study completion, an average of 1 year
Adverse events were coded with MedDRA Dictionary Version 26.1.
Number of Participants Discontinuing Study Drug and Number of Participants' Deaths
Time frame: Through study completion, an average of 1 year
Total Number of Mild, Moderate, and Severe Adverse Events (AEs) Across All Participants
Time frame: Through study completion, an average of 1 year
Adverse events were coded with MedDRA Dictionary Version 26.1.

10 secondary outcomes

Change From Baseline to Day 180 and Day 360 in Modified-SBMAFRS (m-SBMAFRS)
Time frame: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in Two-Minute Walk Test (2MWT)
Time frame: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in 6-Minute Walk Test (6MWT)
Time frame: Baseline, Day 180, Day 360
Change From Baseline to Day 180 and Day 360 in Timed Up and Go (TUG) Test
Time frame: Baseline, Day 180, Day 360
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Non-Sedentary Behavior
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: 95th Percentile of 6-Minute Physical Activity Windows
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Acitgraphy-Derived Measurement: Maximum Walking Bout Duration
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Peak 95th Percentile Cadence
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Treatment (Days 331 to 360) in Actigraphy Derived Measurement: Average Breathing Rate
Time frame: Baseline, Treatment (Days 331 to 360)
Change From Baseline to Day 180 and Day 360 in Grip Strength as Measured by Handheld Dynamometer (HHD)
Time frame: Baseline, Day 180, Day 360

Results and safety

Results posted to the registry on 3 Mar 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	26	26
NIDO-361	28	27

Primary outcome: Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C]

Time frame: Baseline, Day 180, Days 360-374 (Last Visit). Mean (Standard Deviation). Unit: cm³

Measure	Placebo n = 26	NIDO-361 n = 28
Thigh LMV [A+B+C] - Baseline	4.1402 (1.7780)	3.8580 (1.5878)
Thigh LMV [A+B+C] - Day 180	-0.0871 (0.1354)	-0.1042 (0.1496)
Thigh LMV [A+B+C] - Last Visit	-0.1363 (0.1648)	-0.1511 (0.2453)
Total LMV [A+B+C] - Baseline	9.8473 (3.4210)	9.1409 (3.4097)
Total LMV [A+B+C] - Day 180	-0.2723 (0.4268)	-0.2853 (0.3296)
Total LMV [A+B+C] - Last Visit	-0.3120 (0.4020)	-0.4147 (0.6160)

Population analysed: All randomized patients with at least one post baseline MRI assessment were included in this analysis. Only muscles assessed with no major quality issues were included in the analysis.

Primary outcome: Number of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)

Time frame: Through study completion, an average of 1 year. Number. Unit: participants

Measure	Placebo n = 26	NIDO-361 n = 28
Number of Participants with AEs: Any Treatment-Emergent Adverse Events (TEAEs)	25	26
Number of Participants with Drug-Related AEs: Any Treatment-Emergent Adverse Events (TEAEs)	9	17
Number of Participants with SAEs: Any Treatment-Emergent Adverse Events (TEAEs)	3	3
Number of Participants with Drug-Related SAEs: Any Treatment-Emergent Adverse Events (TEAEs)	0	1

Population analysed: The safety analysis set was defined as all randomized patients, who received at least 1 dose of study treatment (placebo or NIDO-361). If a patient experienced more than 1 event in a given system organ class (SOC), that patient was counted once for the SOC. If a patient experienced more than 1 event with a given preferred term (PT), that patient was counted only once for that PT.

Primary outcome: Number of Participants Discontinuing Study Drug and Number of Participants' Deaths

Time frame: Through study completion, an average of 1 year. Number. Unit: participants

Measure	Placebo n = 26	NIDO-361 n = 28
Number of Participants Discontinuing Study Drug: Any TEAE Leading to Study Drug Discontinutation	0	1
Number of Participants' Deaths: Any TEAE Leading to Study Drug Discontinutation	0	0

Primary outcome: Total Number of Mild, Moderate, and Severe Adverse Events (AEs) Across All Participants

Time frame: Through study completion, an average of 1 year. Number. Unit: adverse events

Measure	Placebo n = 26	NIDO-361 n = 28
Mild (Grade 1) AEs: Any Grade 3 or Above TEAEs	65	98
Moderate (Grade 2) AEs: Any Grade 3 or Above TEAEs	27	36
Severe (Grade 3) AEs: Any Grade 3 or Above TEAEs	2	10

10 secondary outcomes are reported in the full results.

Adverse events

Time frame: From enrollment until end of study (up to 1 year). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	3 of 26 (11.5%)	25 of 26 (96.2%)	0 of 26
NIDO-361	3 of 28 (10.7%)	26 of 28 (92.9%)	0 of 28

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

5 sites in 4 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing NIDO-361 in people with Spinal and Bulbar Muscular Atrophy and Kennedy's Disease, run by Nido Biosciences, Inc. at 5 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in Italy, Denmark, South Korea, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 3 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C] (time frame: Baseline, Day 180, Days 360-374 (Last Visit)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06411912.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2026.