Spinal Muscular AtrophyNCT01166022
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
A study testing Exercise in people with Spinal Muscular Atrophy and Neuromuscular Disease, run by Columbia University at 1 site in United States.
Trial summary
This study is being conducted to test whether exercise can be effectively used as an intervention to treat Spinal Muscular Atrophy (SMA). In order to answer this question, the investigators will enroll 14 subjects with SMA between ages 8 and 50 and ask them to complete an 18 month training schedule. Read more about this study
- Who is it for?
- Ages 8 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Spinal Muscular Atrophy, Neuromuscular Disease
- What is being tested?
- Exercise
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2010to Aug 2014
- Phase
- Interventional
- 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 8 to 50 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Weakness and hypotonia consistent with the clinical diagnosis of SMA type 3, i.e. having achieved the ability to walk independently
- Laboratory documentation of homozygous absence of SMN1 exon 7
- ability to walk at least 25 meters without assistance
- Aged 8 to 50 years at the time of enrollment
- Ability to tread the stationary cycle ergometer
- Written informed consent of patient (if ≥ 18 years of age) or parents/guardian (if < 18 years of age), and assent for participants who are of minor age.
Who cannot join
- Inability to walk independently at least 25 meters
- Any acute co-morbid condition interfering with study participation in the judgment of the investigators within 7 days of enrollment including bacterial infection, viral infectious process, food poisoning, temperature > 99.0ºF, need for acute treatment or observation due to any other reason, as judged by the investigator; patient can be included after resolution of the acute event
- Use of investigational medications intended for the treatment of SMA including riluzole, valproic acid, hydroxyurea, oral use of albuterol, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, or agents with known or presumed histone deacetylase (HDAC) inhibition, within 30 days prior to study entry. After signing informed consent, participants may discontinue prohibited medications and will then qualify for a screening visit after the 30 day wash-out period
- Inability to meet study visit requirements, or cooperate reliably with functional testing and exercise protocol
- Coexisting medical conditions that contraindicate travel, testing or moderate intensity exercise
- Pregnant or breastfeeding women, or those intending to become pregnant during the course of the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University SMA Clinical Research Center
New York, New York, 10032
About this study
Official title: Randomized, Controlled Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
This study is being conducted to test whether exercise can be effectively used as an intervention to treat Spinal Muscular Atrophy (SMA). In order to answer this question, the investigators will enroll 14 subjects with SMA between ages 8 and 50 and ask them to complete an 18 month training schedule. At some points subjects will be asked to closely follow a specific training regimen and at other points they may be asked to exercise in the same manner they do normally. The exercises they will be asked to perform include biking on a stationary cycle and lifting hand weights. Subjects will be asked to come in to the clinic seven times over the course of the study to perform tests. These tests include motor function measures, a physical exam, questionnaires, a exercise capacity test which involves riding a stationary bicycle, and test where the subject is asked to walk as far as they can in six minutes. The main goal of the study is to see if the subjects who participate in the exercise protocol have larger increases in the distance they can walk in six minutes than those who do not.
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Disease
- Being tested
- Exercise
- Keywords
- Spinal Muscular Atrophy, Exercise, Conditioning, Strengthening
- Medical subject headings
- Muscular Atrophy, Spinal, Neuromuscular Diseases, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExerciseOther
Muscle strengthening program using hand weights and resistance bands in combination with a home based cycle ergometry. This home-based exercise program will be performed up to 5 times weekly. Each session will consist of up to 30 minutes of cycling and 30 minutes of strengthening.
| Study group | Role | What they receive |
|---|---|---|
| Exercise | Experimental | Muscle strengthening program using weights and resistance bands in combination with a home based cycle ergometry program. The home-based exercise program will be performed up to 5 times weekly. Receives: Other: Exercise |
| Typical Activity | No intervention | Subjects in this group will be asked to maintain their typical daily activity. Those assigned to this arm will be given the opportunity to join the intervention arm seven months after their screening visit. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Distance walked in the Six Minute Walk Test (6MWT).
Measured: Up to 19 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2010 (estimated)
- Study startDec 2010
- Primary completionAug 2014
- Study completionAug 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Darryl C. De Vivo, Columbia University
- Investigators
- Darryl C De Vivo, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01166022
- Sponsor's protocol ID
- AAAE8200
- Other IDs
- A-15886
- Status last verified
- Aug 2016
- First submitted
- 14 Jul 2010
- Last updated
- 12 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Controlled Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
- ClinicalTrials.gov ID
- NCT01166022
- Acronym / sponsor ID
- AAAE8200
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Disease
- Keywords
- Spinal Muscular Atrophy, Exercise, Conditioning, Strengthening
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Study start
- Dec 2010
- Primary completion
- Aug 2014 (actual)
- Study completion
- Aug 2014 (actual)
- First posted
- 20 Jul 2010 (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.
This study is being conducted to test whether exercise can be effectively used as an intervention to treat Spinal Muscular Atrophy (SMA). In order to answer this question, the investigators will enroll 14 subjects with SMA between ages 8 and 50 and ask them to complete an 18 month training schedule. At some points subjects will be asked to closely follow a specific training regimen and at other points they may be asked to exercise in the same manner they do normally. The exercises they will be asked to perform include biking on a stationary cycle and lifting hand weights. Subjects will be asked to come in to the clinic seven times over the course of the study to perform tests. These tests include motor function measures, a physical exam, questionnaires, a exercise capacity test which involves riding a stationary bicycle, and test where the subject is asked to walk as far as they can in six minutes. The main goal of the study is to see if the subjects who participate in the exercise protocol have larger increases in the distance they can walk in six minutes than those who do not.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exercise | Experimental | Other: ExerciseMuscle strengthening program using weights and resistance bands in combination with a home based cycle ergometry program. The home-based exercise program will be performed up to 5 times weekly. |
| Typical Activity | No intervention | –Subjects in this group will be asked to maintain their typical daily activity. Those assigned to this arm will be given the opportunity to join the intervention arm seven months after their screening visit. |
Who may qualify
- Ages
- 8 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Weakness and hypotonia consistent with the clinical diagnosis of SMA type 3, i.e. having achieved the ability to walk independently
- Laboratory documentation of homozygous absence of SMN1 exon 7
- ability to walk at least 25 meters without assistance
- Aged 8 to 50 years at the time of enrollment
- Ability to tread the stationary cycle ergometer
Key exclusion criteria (6 listed)
- Inability to walk independently at least 25 meters
- Any acute co-morbid condition interfering with study participation in the judgment of the investigators within 7 days of enrollment including bacterial infection, viral infectious process, food poisoning, temperature > 99.0ºF, need for acute treatment or observation due to any other reason, as judged by the investigator; patient can be included after resolution of the acute event
- Use of investigational medications intended for the treatment of SMA including riluzole, valproic acid, hydroxyurea, oral use of albuterol, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, or agents with known or presumed histone deacetylase (HDAC) inhibition, within 30 days prior to study entry. After signing informed consent, participants may discontinue prohibited medications and will then qualify for a screening visit after the 30 day wash-out period
- Inability to meet study visit requirements, or cooperate reliably with functional testing and exercise protocol
- Coexisting medical conditions that contraindicate travel, testing or moderate intensity exercise
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 |
|---|---|---|
| ExerciseMuscle strengthening program using hand weights and resistance bands in combination with a home based cycle ergometry. This home-based exercise program will be performed up to 5 times weekly. Each session will consist of up to 30 minutes of cycling and 30 minutes of strengthening. | Other | Exercise |
Primary outcome
Distance walked in the Six Minute Walk Test (6MWT).
Time frame: Up to 19 months
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 12 Aug 2016.
- Related InfoLink provided by the sponsor.
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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.
Darryl C De Vivo, MD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Exercise in people with Spinal Muscular Atrophy and Neuromuscular Disease, run by Columbia University 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 6 inclusion and 6 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 12 Aug 2016. 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.
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?
Distance walked in the Six Minute Walk Test (6MWT). (time frame: Up to 19 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Aug 2016.