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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Darryl C. De Vivo, Columbia University
ClinicalTrials.gov Identifier:
NCT01166022
First received: July 14, 2010
Last updated: July 29, 2013
Last verified: July 2013
  Purpose

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 Intervention
Spinal Muscular Atrophy
Neuromuscular Disease
Other: Exercise

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized, Controlled Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)

Resource links provided by NLM:


Further study details as provided by Columbia University:

Primary Outcome Measures:
  • Distance walked in the Six Minute Walk Test (6MWT). [ Time Frame: Up to 19 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 14
Study Start Date: November 2010
Estimated Study Completion Date: August 2014
Primary Completion Date: February 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   8 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01166022

Locations
United States, New York
Columbia University SMA Clinical Research Center
New York, New York, United States, 10032
Sponsors and Collaborators
Columbia University
Investigators
Principal Investigator: Darryl C De Vivo, MD Columbia University
  More Information

Additional Information:
No publications provided

Responsible Party: Darryl C. De Vivo, Sidney Carter Professor of Neurology and Professor of Pediatrics, Columbia University
ClinicalTrials.gov Identifier: NCT01166022     History of Changes
Other Study ID Numbers: AAAE8200, A-15886
Study First Received: July 14, 2010
Last Updated: July 29, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Columbia University:
Spinal Muscular Atrophy
Exercise
Conditioning
Strengthening

Additional relevant MeSH terms:
Atrophy
Muscular Atrophy
Muscular Atrophy, Spinal
Neuromuscular Diseases
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases

ClinicalTrials.gov processed this record on September 22, 2014