Trial Outcomes & Findings for Progressive Strength Training in Spinal Muscular Atrophy (NCT NCT01233817)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

12 participants

Primary outcome timeframe

12 weeks

Results posted on

2017-06-14

Participant Flow

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

This was a pilot study and all participants were in the treatment group

Participant milestones

Participant milestones
Measure	Spinal Muscular Atrophy Children and adolescents with diagnosis of SMA type II or III
Overall Study STARTED	12
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Spinal Muscular Atrophy Children and adolescents with diagnosis of SMA type II or III
Overall Study Lost to Follow-up	1
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Progressive Strength Training in Spinal Muscular Atrophy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Spinal Muscular Atrophy n=9 Participants Children and adolescents with diagnosis of SMA type II or III
Age, Categorical <=18 years	8 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Age, Continuous	9.56 years STANDARD_DEVIATION 3.94 • n=99 Participants
Sex: Female, Male Female	5 Participants n=99 Participants
Sex: Female, Male Male	4 Participants n=99 Participants
Region of Enrollment United States	9 participants n=99 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Spinal Muscular Atrophy n=9 Participants Children and adolescents with diagnosis of SMA type II or III
Strength	0.39 kilograms Interval -0.5 to 1.1

Adverse Events

Spinal Muscular Atrophy

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Aga Lewelt MD, Principal Investigator

Department of Physical Medicine and Rehabilitation, School of Medicine, University of Utah

Phone: 801-518-4572

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place