Spinal Muscular Atrophy Clinical Trial
Official title:
The Role of Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)
Verified date | April 2024 |
Source | Ohio State University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
The primary objective of this research protocol is to study and follow the course of motor neuron loss in individuals with spinal muscular atrophy (SMA) using the electrophysiological technique of motor unit number estimation (MUNE). This study is based on the hypothesis that the electrophysiological technique of motor unit number estimation (MUNE) and compound muscle action potential (CMAP) provide sensitive indicators to assess the severity and progression of disease in adults with SMA.
Status | Active, not recruiting |
Enrollment | 140 |
Est. completion date | December 31, 2024 |
Est. primary completion date | December 31, 2024 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 17 Years to 70 Years |
Eligibility | Inclusion Criteria: - 1. Adults age 17 to 70 with SMA type 2 or 3. The diagnosis of SMA must be documented by the homozygous deletion of both SMN1 genes on standard genetic tests for the disorder, and the determination of type 2 SMA by history of ability to maintain a sitting position when placed, or type 3 by the ability to stand and walk independently. Wheelchair mobile patients will not be excluded from the study. 2. Interest in participating and the ability to travel to the study site on an agreed upon date on four occasions over a total interval of twenty-four months. 3. Be in good health with the exception of SMA. Assessments will be rescheduled for a later date in the event of any intercurrent illness that might affect performance on the assessment. Inclusion criteria for control subjects: 1. Healthy adults age 17 to 70 2. Interest in participating and the ability to travel to the study site Exclusion Criteria: - Patients unable to tolerate the assessment by virtue of associated medical conditions, respiratory failure with ventilator dependence, or an obligatory need for orthotics that cannot be removed during the evaluation. 2. Patients who are, in the investigator's opinion, mentally or legally incapacitated, preventing informed consent, or is unable to read and understand written material including the consent 3. Patients with a pacemaker or cardiac device Exclusion criteria for control subjects: 1. Subjects with any neurological disorder 2. Subjects with chronic medical illness that will interfere with their ability to tolerate the study 3. Subjects who are, in the investigator's opinion, mentally or legally incapacitated, preventing informed consent, or is unable to read and understand written material including the consent. |
Country | Name | City | State |
---|---|---|---|
United States | The Ohio State University Medical Center | Columbus | Ohio |
Lead Sponsor | Collaborator |
---|---|
Ohio State University | Cure SMA |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | MUNE | The primary objective of this proposal is to study and follow the course of motor neuron loss in adults with spinal muscular atrophy (SMA) using the electrophysiological technique of motor unit number estimation (MUNE). This study is based on the hypothesis that the electrophysiological technique of motor unit number estimation (MUNE) can provide sensitive indicator data to assess the severity and progression of disease in adults with SMA. | up to 15 years | |
Primary | CMAP | The primary objective of this proposal is to study and follow the course of motor neuron loss in adults with spinal muscular atrophy (SMA) using the electrophysiological technique of compound muscle action potential (CMAP). This study is based on the hypothesis that the electrophysiological technique of compound muscle action potential (CMAP) can provide sensitive indicator data to assess the severity and progression of disease in adults with SMA. | up to 15 years | |
Secondary | EIM | Secondary Objective is to see if electrical impedance myography (EIM) correlates to the electrophysiological testing (see Primary Outcome measures: MUNE and CMAP). | up to 15 years | |
Secondary | Nusinersen | Explore the changes in outcome measures in patients treated with nusinersen | up to 15 years | |
Secondary | Treatment insurance approval and safety | Explore the insurance approval process, patient acceptance, and side effects of the newly approved FDA treatment of nusinersen. | up to 15 years |
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