Muscular Atrophy, Spinal
Conditions
Keywords
Spinal muscular atrophy, Neuromuscular disorder, Progressive strength training, Children
Brief summary
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.
Interventions
the systematic increase in resistance weights
Sponsors
University of Utah
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 21 Years
Healthy volunteers
No
Inclusion criteria
* age 5-21 years * diagnosis of SMA type II or III * at least antigravity strength in elbow flexors * ability to follow commands and stay on task * place of residence within a 60-minute or 60-mile drive of the University of Utah
Exclusion criteria
* planned surgery or out-of-town trips during the proposed intervention period * inability to travel to study center for testing * neurological diagnosis other than SMA
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Strength | 12 weeks | 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. |
Countries
United States
Participant flow
Recruitment details
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
Pre-assignment details
This was a pilot study and all participants were in the treatment group
Participants by arm
| Arm | Count |
|---|---|
| Spinal Muscular Atrophy Children and adolescents with diagnosis of SMA type II or III | 9 |
| Total | 9 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Withdrawal by Subject | 2 |
Baseline characteristics
| Characteristic | Spinal Muscular Atrophy |
|---|---|
| Age, Categorical <=18 years | 8 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Age, Continuous | 9.56 years STANDARD_DEVIATION 3.94 |
| Region of Enrollment United States | 9 participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 9 |
| serious Total, serious adverse events | 0 / 9 |
Outcome results
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.
Time frame: 12 weeks
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Spinal Muscular Atrophy | Strength | 0.39 kilograms |