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

Primary Purpose

Spinal Muscular Atrophy, Neuromuscular Disease

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Exercise

About this trial

This is an interventional treatment trial for Spinal Muscular Atrophy focused on measuring Spinal Muscular Atrophy, Exercise, Conditioning, Strengthening

Eligibility Criteria

8 Years - 50 Years (Child, Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

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 Criteria:

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.

Sites / Locations

Columbia University SMA Clinical Research Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Exercise

Typical Activity

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Distance walked in the Six Minute Walk Test (6MWT).

Secondary Outcome Measures

Full Information

NCT ID

NCT01166022

First Posted

July 14, 2010

Last Updated

August 11, 2016

Sponsor

Columbia University

1. Study Identification

Unique Protocol Identification Number

NCT01166022

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2016

Overall Recruitment Status

Completed

Study Start Date

December 2010 (undefined)

Primary Completion Date

August 2014 (Actual)

Study Completion Date

August 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Columbia University

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Spinal Muscular Atrophy, Neuromuscular Disease

Keywords

Spinal Muscular Atrophy, Exercise, Conditioning, Strengthening

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Parallel Assignment

Masking

Outcomes Assessor

Allocation

Randomized

Enrollment

14 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Exercise

Arm Type

Experimental

Arm Description

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.

Arm Title

Typical Activity

Arm Type

No Intervention

Arm Description

Intervention Type

Other

Intervention Name(s)

Exercise

Intervention Description

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.

Primary Outcome Measure Information:

Title

Distance walked in the Six Minute Walk Test (6MWT).

Time Frame

Up to 19 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

8 Years

Maximum Age & Unit of Time

50 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Criteria: 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Darryl C De Vivo, MD

Organizational Affiliation

Columbia University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Columbia University SMA Clinical Research Center

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

12. IPD Sharing Statement

Links:

URL

http://www.columbiasma.org

Description

Related Info

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

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