Actively Recruiting

Age: 18Years - 120Years
MALE
Healthy Volunteers
NCT04944940

Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

Led by National Institute of Neurological Disorders and Stroke (NINDS) · Updated on 2026-03-11

70

Participants Needed

1

Research Sites

278 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Background: SBMA is an inherited chronic disease. It affects males in mid to late adulthood. It causes slowly progressive weakness of muscles and hand tremors. Researchers want to learn more about the effects of SBMA. Objective: To identify measurements that change over time in SBMA, including tests of muscle strength and function, as well as measurements of muscle and fat size. Eligibility: Men over the age of 18 both with and without a history of SBMA. Design: Participants will have a medical history, physical exam, and blood and urine tests. They will have neuromuscular ultrasound. They will have a lumbar puncture to obtain spinal fluid. For this, a needle will be inserted into the spinal canal in the lower back. Participants will have muscle strength and function tests. These tests may include pushing, pulling, rising from a chair and sitting back down, and/or walking. During these tests, they may wear an accelerometer (activity tracker) on their wrist. Participants will get an activity tracker to wear on their wrist for 10 days at home every 3 months. Participants with SBMA will also have lower limb magnetic resonance imaging (MRI) and optional whole-body MRI. They will have lung function tests. They will have speech and swallow tests. They will complete questionnaires. They may have optional body scans to measure bone density and lean body mass. They may have optional muscle biopsies. For biopsies, a needle will be used to take a small piece of muscle from the leg. Participants with SBMA will have 5 study visits over 2 years (every 6 months). Participants without SBMA will have 1 study visit.

CONDITIONS

Official Title

Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

Who Can Participate

Age: 18Years - 120Years
MALE
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Male, age 18 years or older
  • Willingness to comply with all study procedures and available for the study duration
  • For SBMA group: genetically confirmed SBMA
  • Ability to understand and sign informed consent
  • Ability to travel to the NIH Clinical Center
  • For healthy controls: no history of SBMA, neuromuscular disorder, or facial palsy
Not Eligible

You will not qualify if you...

  • Female or younger than 18 years
  • Lack of consent capacity
  • Contraindications to MRI such as non-removable metal devices or claustrophobia
  • Non-ambulatory status
  • Use of androgen reducing agents within past two years
  • For lumbar puncture: bleeding disorders, abnormal clotting tests, or use of anticoagulants
  • For muscle biopsy: advanced muscle wasting precluding biopsy, recent use of aspirin or NSAIDs
  • For whole-body MRI: prior androgen reducing treatments, inability to complete strength or walk tests, or planned major lifestyle changes
  • For healthy controls: bleeding disorders, abnormal clotting tests, or use of anticoagulants

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 1 location

1

National Institutes of Health Clinical Center

Bethesda, Maryland, United States, 20892

Actively Recruiting

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Research Team

A

Angela D Kokkinis, R.N.

CONTACT

C

Christopher Grunseich, M.D.

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

2

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Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA) | DecenTrialz