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The proposed feasibility study is necessary to test if children and young adults will participate in and adhere to a 12-week, home-based, supervised progressive strength training exercise program and to obtain preliminary data that will subsequently allow us to determine the safety and impact of strength training in spinal muscular atrophy. Our pilot study will address 3 aims: (1) Ascertain the feasibility of, and potential barriers to, participation in and adherence to a 12-week home-based, supervised, progressive strength training exercise program in children and young adults aged 5-21 years with SMA types II and III; (2) Determine the safety and tolerability of progressive strength training in a pilot study sample of children and young adults with SMA types II and III; and (3) Determine candidate outcome measures.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Spinal muscular atrophy | Experimental | Children and adolescents with diagnosis of SMA type II or III. The intervention group (the only arm/group in this pilot study) receives a home-based, supervised, 12-week progressive strength-training program. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Progressive strength training | Other | the systematic increase in resistance weights |
|
| Measure | Description | Time Frame |
|---|---|---|
| Strength | Primary Outcome Measure was muscle strength. Strength was measured using a fixed myometry evaluation, quantitative muscle analysis (QMA). QMA utilizes a relative fixed point for the participant to exert effort. Each muscle of interest was tested using QMA. | 12 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Aga Lewelt, MD | University of Utah | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Utah | Salt Lake City | Utah | 84132 | United States |
This was a pilot study and all participants were in the treatment group
Participants were recruited from May 2010 until February 2012. Some participants were recruited during clinic visits and others contacted us after being informed of the study
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| ID | Title | Description |
|---|---|---|
| FG000 | Spinal Muscular Atrophy | Children and adolescents with diagnosis of SMA type II or III |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Spinal Muscular Atrophy | Children and adolescents with diagnosis of SMA type II or III |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Strength | Primary Outcome Measure was muscle strength. Strength was measured using a fixed myometry evaluation, quantitative muscle analysis (QMA). QMA utilizes a relative fixed point for the participant to exert effort. Each muscle of interest was tested using QMA. | Posted | Median | 95% Confidence Interval | kilograms | 12 weeks |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Spinal Muscular Atrophy | Children and adolescents with diagnosis of SMA type II or III |
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This was a pilot study with only one arm, the treatment arm. This is an inherent limitation.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Aga Lewelt MD, Principal Investigator | Department of Physical Medicine and Rehabilitation, School of Medicine, University of Utah | 801-518-4572 | aga@genetics.utah.edu |
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| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| D009468 | Neuromuscular Diseases |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
| 0 |
| 9 |
| 0 |
| 9 |
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| D019636 | Neurodegenerative Diseases |