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OpenTrials
Completed

NCT Number: NCT02044029

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.

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.

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Key information

Conditions

Healthy Volunteer, Muscular Atrophy, Spinal Atrophy Muscular Atrophy Nervous System Diseases Neurologic Manifestations Neuromuscular Manifestations Pathological Conditions, Anatomical Pathological Conditions, Signs and Symptoms Signs and Symptoms

Age range

10 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Basel, 4005, Switzerland

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Differences in quantitative muscle MRI based on fat content and T2 values

    Time frame: At baseline, Week 12 and Week 24

  2. Disease progression, assessed through the Motor Function Measure test

    Time frame: At baseline, Week 12 and Week 24

Secondary outcomes

  1. Disease progression, assessed with the 6-minute Walk Test.

    Time frame: At baseline, Week 12 and Week 24

  2. Levels of Survival of Motor Neuron (SMN) RNA

    Time frame: At baseline and Week 24

  3. Levels of SMN proteins

    Time frame: At baseline and Week 24

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study 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

Important dates

Study start
2014
Primary completion
2015
Study completion
2015
First posted
Jan 23, 2014
Registry last updated
Nov 2, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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