Progressive Strength Training in Spinal Muscular Atrophy

NCT01233817 | Completed Results DMC

• 1 other identifier: 39995
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Muscular Atrophy, Spinal

Keywords

Spinal muscular atrophy; Neuromuscular disorder; Progressive strength training; Children

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

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.

Other: Progressive strength training

Interventions

Progressive strength training OTHER

the systematic increase in resistance weights

Spinal muscular atrophy

Eligibility Criteria

• Age: 5 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Utah: lead

Study Sites (1)

University of Utah

Salt Lake City, Utah, 84132, United States

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal; Neuromuscular Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases

Limitations and Caveats

This was a pilot study with only one arm, the treatment arm. This is an inherent limitation.

Results Point of Contact

• Title: Aga Lewelt MD, Principal Investigator
• Organization: Department of Physical Medicine and Rehabilitation, School of Medicine, University of Utah

aga@genetics.utah.edu

801-518-4572

Study Officials

• Aga Lewelt, MD

  University of Utah

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 19, 2010
• First Posted: November 3, 2010
• Study Start: June 1, 2010
• Primary Completion: June 1, 2013
• Study Completion: June 1, 2013
• Last Updated: June 14, 2017
• Results First Posted: March 12, 2013

Record last verified: 2017-05

Locations

US (1)