Motor Neuron DiseasePhase 1/2NCT01369901
Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy
A Phase 1/2 study testing A functional exercise program and A stretching exercise program in people with Motor Neuron Disease, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 90 years, men only
- Conditions the study is for
- Motor Neuron Disease
- What is being tested?
- A functional exercise program and A stretching exercise program
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Jun 2011to 28 Feb 2018
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 90 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 12Show fewer
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: 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.
Read more
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....
Read the detailed description
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.
- Conditions
- Motor Neuron Disease
- Being tested
- A functional exercise program, A stretching exercise program
- Keywords
- Exercise, Spinal Bulbar Muscular Atrophy, Spinal and Bulbar Muscular Atrophy, SBMA, Motor Neuron Disease
- Medical subject headings
- Motor Neuron Disease, Motor Activity, Bulbo-Spinal Atrophy, X-Linked
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
A functional exercise programBehavioural
A stretching exercise programBehavioural
| Study group | Role | What they receive |
|---|---|---|
| Group A | Experimental | Functional exercise Receives: Behavioral: A functional exercise program |
| Group B | Active comparator | Stretching exercise Receives: Behavioral: A stretching exercise program |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Beck Depression Inventory (BDI) testing will also be used to determine if the subjects mood is affected by exercise.
To assess the effect of functional exercise on quality of life as measured by the SF36v2.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2011 (estimated)
- Study start8 Jun 2011
- Study completion28 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Motor Neuron DiseaseStudies of the same condition, open and under way.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)The sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Investigators
- Kenneth H Fischbeck, M.D., Principal investigator, National Institute of Neurological Disorders and Stroke (NINDS)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01369901
- Sponsor's protocol ID
- 110171
- Other IDs
- 11-N-0171
- Status last verified
- 28 Feb 2018
- First submitted
- 8 Jun 2011
- Last updated
- 17 Dec 2019
- Results posted
- No
Publications
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.
PubMed 4233749Harding 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.
PubMed 6890989Olney 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.
PubMed 2046924
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Functional Exercise in Patients With Spinal and Bulbar Muscular Atrophy
- ClinicalTrials.gov ID
- NCT01369901
- Acronym / sponsor ID
- 110171
- Conditions
- Motor Neuron Disease
- Keywords
- Exercise, Spinal Bulbar Muscular Atrophy, Spinal and Bulbar Muscular Atrophy, SBMA, Motor Neuron Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- 8 Jun 2011
- Study completion
- 28 Feb 2018 (actual)
- First posted
- 9 Jun 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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....
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A | Experimental | Behavioral: A functional exercise programFunctional exercise |
| Group B | Active comparator | Behavioral: A stretching exercise programStretching exercise |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
Key exclusion criteria (3 listed)
- 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.
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 |
|---|---|---|
| A functional exercise program | Behavioural | Group A |
| A stretching exercise program | Behavioural | Group B |
Primary outcome
Adult Myopathy Assessment Tool (AMAT)
7 secondary outcomes
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.
Beck Depression Inventory (BDI) testing will also be used to determine if the subjects mood is affected by exercise.
To assess the effect of functional exercise on quality of life as measured by the SF36v2.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2019.
- Background: PubMed 4233749Kennedy 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.
- Background: PubMed 6890989Harding 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.
- Background: PubMed 2046924Olney 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.
Related trials for Motor Neuron Disease
Active, not recruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
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.
Kenneth H Fischbeck, M.D. principal investigator
National Institute of Neurological Disorders and Stroke (NINDS)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing A functional exercise program and A stretching exercise program in people with Motor Neuron Disease, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
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 12 inclusion and 3 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 17 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Adult Myopathy Assessment Tool (AMAT). 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 NCT01369901, which also lists 3 linked publications.
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 17 Dec 2019.