Status:
COMPLETED
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
Lead Sponsor:
Columbia University
Conditions:
Spinal Muscular Atrophy
Neuromuscular Disease
Eligibility:
All Genders
8-50 years
Phase:
NA
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 enrol...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
December 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2014
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT01166022
Start Date
December 1 2010
End Date
August 1 2014
Last Update
August 12 2016
Active Locations (1)
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1
Columbia University SMA Clinical Research Center
New York, New York, United States, 10032