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Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01166022
Recruitment Status : Completed
First Posted : July 20, 2010
Last Update Posted : August 12, 2016
Information provided by (Responsible Party):
Darryl C. De Vivo, Columbia University

Brief 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. 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.

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Neuromuscular Disease Other: Exercise Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized, Controlled Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
Study Start Date : December 2010
Actual Primary Completion Date : August 2014
Actual Study Completion Date : August 2014

Arm Intervention/treatment
Experimental: Exercise
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.
Other: Exercise
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.

No Intervention: Typical Activity
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.

Primary Outcome Measures :
  1. Distance walked in the Six Minute Walk Test (6MWT). [ Time Frame: Up to 19 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   8 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  1. Weakness and hypotonia consistent with the clinical diagnosis of SMA type 3, i.e. having achieved the ability to walk independently
  2. Laboratory documentation of homozygous absence of SMN1 exon 7
  3. ability to walk at least 25 meters without assistance
  4. Aged 8 to 50 years at the time of enrollment
  5. Ability to tread the stationary cycle ergometer
  6. 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.

Exclusion Criteria:

  1. Inability to walk independently at least 25 meters
  2. 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
  3. 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
  4. Inability to meet study visit requirements, or cooperate reliably with functional testing and exercise protocol
  5. Coexisting medical conditions that contraindicate travel, testing or moderate intensity exercise
  6. Pregnant or breastfeeding women, or those intending to become pregnant during the course of the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01166022

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United States, New York
Columbia University SMA Clinical Research Center
New York, New York, United States, 10032
Sponsors and Collaborators
Columbia University
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Principal Investigator: Darryl C De Vivo, MD Columbia University
Additional Information:
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Responsible Party: Darryl C. De Vivo, Sidney Carter Professor of Neurology and Professor of Pediatrics, Columbia University Identifier: NCT01166022    
Other Study ID Numbers: AAAE8200
A-15886 ( Other Identifier: CU )
First Posted: July 20, 2010    Key Record Dates
Last Update Posted: August 12, 2016
Last Verified: August 2016
Keywords provided by Darryl C. De Vivo, Columbia University:
Spinal Muscular Atrophy
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Neuromuscular Diseases
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases