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.

November 1, 2016 updated by: Hoffmann-La Roche

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

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

38

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Ambulant patients with spinal muscular atrophy (SMA) and age- and gender-matched healthy volunteers.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Age- and gender-matched controls
Patients with spinal muscular atrophy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Differences in quantitative muscle MRI based on fat content and T2 values
Time Frame: At baseline, Week 12 and Week 24
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
At baseline, Week 12 and Week 24

Secondary Outcome Measures

Outcome Measure
Time Frame
Disease progression, assessed with the 6-minute Walk Test.
Time Frame: At baseline, Week 12 and Week 24
At baseline, Week 12 and Week 24
Levels of Survival of Motor Neuron (SMN) RNA
Time Frame: At baseline and Week 24
At baseline and Week 24
Levels of SMN proteins
Time Frame: At baseline and Week 24
At baseline and Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hoffmann-La Roche

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2014

Primary Completion (Actual)

July 1, 2015

Study Completion (Actual)

July 1, 2015

Study Registration Dates

First Submitted

January 16, 2014

First Submitted That Met QC Criteria

January 21, 2014

First Posted (Estimate)

January 23, 2014

Study Record Updates

Last Update Posted (Estimate)

November 2, 2016

Last Update Submitted That Met QC Criteria

November 1, 2016

Last Verified

November 1, 2016

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BE29037

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Healthy Volunteer, Muscular Atrophy, Spinal

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