Healthy Volunteer, Muscular Atrophy, SpinalNCT02044029

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.

An observational study of Healthy Volunteer, Muscular Atrophy, Spinal, run by Hoffmann-La Roche at 1 site in Switzerland.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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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. Read more about this study

Who is it for?: Ages 10 years and olderHealthy volunteers can take part
Conditions the study is for: Healthy Volunteer, Muscular Atrophy, Spinal
How many people: 38 participants
Where: 1 siteSwitzerland
Study dates: Jan 2014to Jul 2015
Phase: Observational

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 10 years and older
All sexes
Healthy volunteers accepted
Child, Adult, Older adult

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

Who may be able to join

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

Who cannot join

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.

Trial locations and contacts

1 site in 1 country.

Switzerland1 site

Study site
Basel, 4005

Other trials in Switzerland

About this study

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

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.

Conditions: Healthy Volunteer, Muscular Atrophy, Spinal
Medical subject headings: Muscular Atrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02044029
Study group	Role	What they receive
Age- and gender-matched controls		Not described
Patients with spinal muscular atrophy		Not described

What is being measured

The primary outcome is the main question the study is designed to answer.

Differences in quantitative muscle MRI based on fat content and T2 values
Measured: At baseline, Week 12 and Week 24
Disease progression, assessed through the Motor Function Measure test
Measured: At baseline, Week 12 and Week 24

Secondary outcomes (3)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 2014Jul 2015

First posted23 Jan 2014 (estimated)
Study startJan 2014
Primary completionJul 2015
Study completionJul 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Healthy Volunteer, Muscular Atrophy, SpinalStudies of the same condition, open and under way.
More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Case-control
Time perspective: Prospective
Enrolment: 38 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02044029
Sponsor's protocol ID: BE29037
Status last verified: Nov 2016
First submitted: 16 Jan 2014
Last updated: 2 Nov 2016
Results posted: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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