Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy

NCT01369901 | Completed Phase 1

• 2 other identifiers: 11017111-N-0171
• Study Type: interventional
• Target: 61
• Locations: 1 country
• Sites: 1

Brief Summary

Background:

• Spinal and bulbar muscular atrophy (SBMA) is an inherited disorder that affects men. People with SBMA often have weakness throughout the body, including the muscles they use for swallowing, breathing, and speaking. We do not know if exercise helps or harms people with SBMA. Objective:
• To see if a 12-week program of either functional exercise or stretching exercises will improve strength, function, or quality of life in people with SBMA Eligibility:
• Participants will be men 18 years of age or older who have genetic confirmation of SBMA.
• They must be able to walk at least 50 feet with or without an assistive device such as a cane or a walker and stand for 10 minutes without using an assistive device.
• They must have access to a computer with an Internet connection. Design:
• At the first visit to NIH (2 days), participants will have a medical history taken and undergo a physical exam. They will also have blood tests and an EKG, and complete questionnaires about mood, health, and exercise. Tests of muscle strength, balance, and endurance will also be done.
• Participants who qualify for the study will receive instruction about either strengthening or stretching exercises. They will do these exercises at home one to three times a week for 12 weeks.
• They will wear a small activity monitor while they exercise and record their exercise in a diary.
• At the end of 12 weeks, participants will return to the NIH for 2 days. They will undergo the same tests as they had on the first visit.
• Participants will receive follow-up phone calls and e-mails during the study and for 4 weeks after the last visit....

Conditions

Motor Neuron Disease

Keywords

Exercise; Spinal Bulbar Muscular Atrophy; Spinal and Bulbar Muscular Atrophy; SBMA; Motor Neuron Disease

Outcome Measures

Primary Outcomes (1)

• Adult Myopathy Assessment Tool (AMAT)

Secondary Outcomes (7)

• To assess the effect of functional exercise on muscle strength as measured by quantitative muscle analysis (QMA), and the Timed up and go test (TUG), and progressive height sit-to-stand.

• Several exploratory biomarkers that may be affected by exercise will be evaluated, including insulin-like growth factor-1 (IGF-1), testosterone, growth hormone, and creatine kinase.

• To assess the ability of patients to maintain exercise compliance as measured by accelerometer measurements.

• To assess the effect of functional exercise on balance as measured by Computerized Dynamic Posturography (CDP) evaluation.

• To assess the effect of functional exercise on adverse outcomes as measured by an adverse event questionnaire.

Study Arms (2)

Group A

EXPERIMENTAL

Functional exercise

Behavioral: A functional exercise program

Group B

ACTIVE COMPARATOR

Stretching exercise

Behavioral: A stretching exercise program

Interventions

A functional exercise program BEHAVIORAL

Group A

A stretching exercise program BEHAVIORAL

Group B

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Genetically confirmed SBMA.
• Ambulatory and walk a distance of at least 50 feet with or without a walker.
• Able to stand for 10 minutes without the use of any assistive devices.
• Willing to travel to the NIH at the beginning and end of the study.
• Willing to participate in telephone monitoring.
• AMAT score of less than 41, but greater than 14.
• Male.
• Willing to participate in all aspects of trial design and follow-up.
• Access to a computer with an internet connection
• Able to do all of the exercises according to the standards of the study examiners at the beginning and end of the study
• Willing to forgo starting an additional exercise plan for the 12 week duration of the study
• Age greater than 18 years

You may not qualify if:

• Medical condition which would preclude exercise such as COPD, congestive heart failure, and cardiac arrhythmias.
• Presence of an additional comorbid condition such as stroke, myopathy, or radiculopathy which also results in weakness.
• Beginning a separate exercise program involving at least two weekly sessions of 20 minutes of exercise each within two months of the start of the trial.

Sponsors & Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Kennedy WR, Alter M, Sung JH. Progressive proximal spinal and bulbar muscular atrophy of late onset. A sex-linked recessive trait. Neurology. 1968 Jul;18(7):671-80. doi: 10.1212/wnl.18.7.671. No abstract available.

  PMID: 4233749 BACKGROUND

• Harding AE, Thomas PK, Baraitser M, Bradbury PG, Morgan-Hughes JA, Ponsford JR. X-linked recessive bulbospinal neuronopathy: a report of ten cases. J Neurol Neurosurg Psychiatry. 1982 Nov;45(11):1012-9. doi: 10.1136/jnnp.45.11.1012.

  PMID: 6890989 BACKGROUND

• Olney RK, Aminoff MJ, So YT. Clinical and electrodiagnostic features of X-linked recessive bulbospinal neuronopathy. Neurology. 1991 Jun;41(6):823-8. doi: 10.1212/wnl.41.6.823.

  PMID: 2046924 BACKGROUND

MeSH Terms

Conditions

Motor Neuron Disease; Motor Activity; Bulbo-Spinal Atrophy, X-Linked

Condition Hierarchy (Ancestors)

Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Behavior; Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Kenneth H Fischbeck, M.D.

  National Institute of Neurological Disorders and Stroke (NINDS)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NIH

Study Record Dates

• First Submitted: June 8, 2011
• First Posted: June 9, 2011
• Study Start: June 8, 2011
• Study Completion: February 28, 2018
• Last Updated: December 17, 2019

Record last verified: 2018-02-28

Locations

US (1)