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.

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

38 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-01-31

Study Completion Date

2015-07-31

Brief Summary

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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.

Detailed Description

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Conditions

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Healthy Volunteer, Muscular Atrophy, Spinal

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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Age- and gender-matched controls

No interventions assigned to this group

Patients with spinal muscular atrophy

No interventions assigned to this group

Eligibility Criteria

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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.

Minimum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Basel, , Switzerland

Site Status

Countries

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Switzerland

Other Identifiers

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BE29037

Identifier Type: -

Identifier Source: org_study_id