Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.
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|ClinicalTrials.gov Identifier: NCT02044029|
Recruitment Status : Completed
First Posted : January 23, 2014
Last Update Posted : November 2, 2016
This non-drug, single center, 24-week, longitudinal study in ambulant spinal muscular atrophy (SMA) patients and in age- and gender-matched healthy volunteers will assess the detection of disease progression by magnetic resonance imaging (MRI) and the Muscle Function Measure (MFM) test.
Each participant will be evaluated in three testing sessions: at baseline, at Week 12 and at Week 24. Both patients and volunteers will undergo MRI scans. Patients will additionally undergo testing of motor function and have blood samples taken for Survival of the Motor Neuron (SMN) genes, proteins and mRNA analysis.
|Condition or disease|
|Healthy Volunteer, Muscular Atrophy, Spinal|
|Study Type :||Observational|
|Actual Enrollment :||38 participants|
|Observational Model:||Case Control|
|Official Title:||A SINGLE CENTER, LONGITUDINAL, NON-DRUG STUDY TO ASSESS THE ABILITY OF MUSCLE IMAGING AND OF MOTOR FUNCTION MEASURE (MFM) TO DETECT CHANGES IN DISEASE PROGRESSION IN AMBULANT SPINAL MUSCULAR ATROPHY (SMA) PATIENTS AS COMPARED TO AGE-MATCHED HEALTHY CONTROLS|
|Study Start Date :||January 2014|
|Actual Primary Completion Date :||July 2015|
|Actual Study Completion Date :||July 2015|
|Age- and gender-matched controls|
|Patients with spinal muscular atrophy|
- Differences in quantitative muscle MRI based on fat content and T2 values [ Time Frame: At baseline, Week 12 and Week 24 ]
- Disease progression, assessed through the Motor Function Measure test [ Time Frame: At baseline, Week 12 and Week 24 ]
- Disease progression, assessed with the 6-minute Walk Test. [ Time Frame: At baseline, Week 12 and Week 24 ]
- Levels of Survival of Motor Neuron (SMN) RNA [ Time Frame: At baseline and Week 24 ]
- Levels of SMN proteins [ Time Frame: At baseline and Week 24 ]
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02044029
|Basel, Switzerland, 4005|
|Study Director:||Clinical Trials||Hoffmann-La Roche|