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.

Healthy Volunteer, Muscular Atrophy, Spinal Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02044029
• Other study ID #: BE29037
• Status: Completed
• Phase: N/A
• First received: January 16, 2014
• Last updated: November 1, 2016
• Start date: January 2014
• Est. completion date: July 2015

Study information

• Verified date: November 2016
• Source: Hoffmann-La Roche
• Contact: n/a
• Is FDA regulated: No
• Health authority: N/A: N/A
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 38
• Est. completion date: July 2015
• Est. primary completion date: July 2015
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 10 Years and older

Eligibility

Inclusion Criteria:

• Male and female subjects, aged >/= 10 years at screening

• For SMA patients: Confirmed clinical diagnosis of 5q-autosomal recessive SMA, ambulant at time of screening, and prefereably without spinal cord fixation

Exclusion Criteria:

• Previous (3 months or less) or concomitant participation in any other therapeutic trial

• Known or suspected cancer

• Other chronic disease or inadequate renal, liver, or heart function

• Contraindications for MRI scans, including but not limited to: claustrophobia, pacemaker, artifical heart valves, cochlear implants, presence of foreign metal objects in the body, intracranial vascular clips, etc. Any contraindications to MRI found on a standard radiography scan.

Study Design

Observational Model: Case Control, Time Perspective: Prospective

Related Conditions & MeSH terms

• Atrophy
• Disease Progression
• Healthy Volunteer, Muscular Atrophy, Spinal
• Muscular Atrophy
• Muscular Atrophy, Spinal

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Differences in quantitative muscle MRI based on fat content and T2 values		At baseline, Week 12 and Week 24	No
Primary	Disease progression, assessed through the Motor Function Measure test		At baseline, Week 12 and Week 24	No
Secondary	Disease progression, assessed with the 6-minute Walk Test.		At baseline, Week 12 and Week 24	No
Secondary	Levels of Survival of Motor Neuron (SMN) RNA		At baseline and Week 24	No
Secondary	Levels of SMN proteins		At baseline and Week 24	No