Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy
| Status: | Completed |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 18 - 90 |
| Updated: | 3/31/2019 |
| Start Date: | June 8, 2011 |
| End Date: | February 28, 2018 |
Effect of Functional Exercise in Patients With Spinal and Bulbar Muscular Atrophy
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....
-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....
Objective: Spinal and bulbar muscular atrophy (SBMA), or Kennedy s disease, is a slowly
progressive, X-linked motor neuron disease for which there is currently no treatment. It is
caused by a mutation in the androgen receptor that results in a polyglutamine repeat
expansion. The role of exercise in motor neuron disease research is debated. Although SBMA is
believed to be primarily a neuronopathy, the potential effect of aberrant androgen receptor
activity on muscle leads exercise to be of uncertain utility in this population. Our
objective is to examine the safety and efficacy of exercise regimens in Kennedy s disease.
Study Population: We aim to enroll 80 men with genetically confirmed Kennedy s disease.
Design: This will be a randomized, evaluator blinded, trial with 25 subjects in each exercise
arm. Following informed consent, the subjects will undergo an initial medical and physical
evaluations followed by a series of neurological tests and blood work over a two-day
outpatient visit at the NIH. The subjects will provide blood work for analysis of hormonal
levels and assessment of any potential muscle damage. On the second day of their visit, the
subjects will be randomized and taught a series of either functional or stretching exercises
that they will engage in as part of the study and control arms, respectively. Following the
baseline visit to NIH, the subjects will be monitored throughout the study with telephone
contacts and other measures including video recording to monitor their progress and
compliance. The subjects will return to the NIH at the end of a 12 week period at which time
the physical and laboratory testing will be repeated. After the 12 week exercise period there
will be a four week period where we will follow-up with the participants by telephone and
e-mail. At the end of the trial all of the subjects will be informed of the study results and
given the opportunity to learn the exercises if they are found to be beneficial.
Outcome Measures: The primary outcome measure used will be the Adult Myopathy Assessment Tool
(AMAT). Secondary outcome measures will involve quantitative muscle analysis (QMA), the Timed
Up and Go (TUG) test, a quality of life measure (SF-36v2TM), adverse event questionnaires, a
Computerized Dynamic Posturography (CDP) assessment of balance, accelerometer measurements of
exercise effort, and progressive height sit-to-stand testing. Several exploratory biomarkers
that may be affected by exercise will be evaluated, including insulin-like growth factor-1
(IGF-1), insulin-like growth factor binding protein 3, testosterone, growth hormone, and
creatine kinase. Beck Depression Inventory (BDI) testing will also be used to determine if
the subjects mood is affected by exercise. Qualitative sub-study questions, international
index of erectile function, Block brief food questionnaire, Aggression Questionnaire,
Activities-specific Balance Confidence (ABC) scale, Fatigue Symptom Inventory, and physical
activity survey measures will also be employed.
progressive, X-linked motor neuron disease for which there is currently no treatment. It is
caused by a mutation in the androgen receptor that results in a polyglutamine repeat
expansion. The role of exercise in motor neuron disease research is debated. Although SBMA is
believed to be primarily a neuronopathy, the potential effect of aberrant androgen receptor
activity on muscle leads exercise to be of uncertain utility in this population. Our
objective is to examine the safety and efficacy of exercise regimens in Kennedy s disease.
Study Population: We aim to enroll 80 men with genetically confirmed Kennedy s disease.
Design: This will be a randomized, evaluator blinded, trial with 25 subjects in each exercise
arm. Following informed consent, the subjects will undergo an initial medical and physical
evaluations followed by a series of neurological tests and blood work over a two-day
outpatient visit at the NIH. The subjects will provide blood work for analysis of hormonal
levels and assessment of any potential muscle damage. On the second day of their visit, the
subjects will be randomized and taught a series of either functional or stretching exercises
that they will engage in as part of the study and control arms, respectively. Following the
baseline visit to NIH, the subjects will be monitored throughout the study with telephone
contacts and other measures including video recording to monitor their progress and
compliance. The subjects will return to the NIH at the end of a 12 week period at which time
the physical and laboratory testing will be repeated. After the 12 week exercise period there
will be a four week period where we will follow-up with the participants by telephone and
e-mail. At the end of the trial all of the subjects will be informed of the study results and
given the opportunity to learn the exercises if they are found to be beneficial.
Outcome Measures: The primary outcome measure used will be the Adult Myopathy Assessment Tool
(AMAT). Secondary outcome measures will involve quantitative muscle analysis (QMA), the Timed
Up and Go (TUG) test, a quality of life measure (SF-36v2TM), adverse event questionnaires, a
Computerized Dynamic Posturography (CDP) assessment of balance, accelerometer measurements of
exercise effort, and progressive height sit-to-stand testing. Several exploratory biomarkers
that may be affected by exercise will be evaluated, including insulin-like growth factor-1
(IGF-1), insulin-like growth factor binding protein 3, testosterone, growth hormone, and
creatine kinase. Beck Depression Inventory (BDI) testing will also be used to determine if
the subjects mood is affected by exercise. Qualitative sub-study questions, international
index of erectile function, Block brief food questionnaire, Aggression Questionnaire,
Activities-specific Balance Confidence (ABC) scale, Fatigue Symptom Inventory, and physical
activity survey measures will also be employed.
- INCLUSION CRITERIA:
1. Genetically confirmed SBMA.
2. Ambulatory and walk a distance of at least 50 feet with or without a walker.
3. Able to stand for 10 minutes without the use of any assistive devices.
4. Willing to travel to the NIH at the beginning and end of the study.
5. Willing to participate in telephone monitoring.
6. AMAT score of less than 41, but greater than 14.
7. Male.
8. Willing to participate in all aspects of trial design and follow-up.
9. Access to a computer with an internet connection
10. Able to do all of the exercises according to the standards of the study examiners
at the beginning and end of the study
11. Willing to forgo starting an additional exercise plan for the 12 week duration of
the study
12. Age greater than 18 years
EXCLUSION CRITERIA:
1. Medical condition which would preclude exercise such as COPD, congestive heart
failure, and cardiac arrhythmias.
2. Presence of an additional comorbid condition such as stroke, myopathy, or
radiculopathy which also results in weakness.
3. 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.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
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