Adults With SMA Treated With Nusinersen
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|ClinicalTrials.gov Identifier: NCT04591678|
Recruitment Status : Active, not recruiting
First Posted : October 19, 2020
Last Update Posted : June 9, 2021
|Condition or disease||Intervention/treatment|
|Spinal Muscular Atrophy||Drug: nusinersen|
This is a single center, 22-month observational study of nusinersen treatment in adult patients with SMA. There will be a total of five visits. All subjects will be evaluated at a screening visit no more than four weeks before starting their standard of care nusinersen treatment to determine their eligibility for participation. Eligible patients will complete their standard induction intrathecal nusinersen treatment on day 1, 15, 29 and 60 followed by maintenance doses every four months. Subjects will be reevaluated after completing the loading doses at 2 months followed by every four-month evaluations for the total duration of the treatment of 22 months.
Currently, there is no data published on patients receiving this treatment over the age of 18. Without published data collected from research, it is difficult for adult patients to get approval from their insurance companies to receive this treatment. This study hopes to collect data, in order to make this treatment option easier for patients with SMA to access and in general learn how well nusinersen is working in adult patients with SMA overtime.
|Study Type :||Observational|
|Estimated Enrollment :||15 participants|
|Official Title:||Characterizing Longitudinal Outcomes in Adults With SMA Treated With Nusinersen|
|Actual Study Start Date :||October 1, 2018|
|Actual Primary Completion Date :||May 14, 2021|
|Estimated Study Completion Date :||June 30, 2021|
SMA nusinersen adult cohort
The nusinersen treatment will be given as standard of care. The treatment (which is NOT research, but the standard care) will be given by an injection into the cerebrospinal fluid (fluid in your spine) through a needle inserted into your lower back. Participants will receive a 12 mg (5 mL) dose during each administration/injection, which will occur on the following days: 1 (baseline), 15, 29, and 60. Following the 60 day treatment, participants will receive treatment every 4 months (6, 10, 14 etc.).
After the 60 day, 6 month, 10 month, 14 month, 18 month and 22 month treatments the study team will see each participant afterwards to collect information to evaluate your general health, function and response to the treatment for the study.
SPINRAZA (nusinersen) is FDA approved to treat spinal muscular atrophy (SMA) through intrathecal injection.
Other Name: SPINRAZA
- Muscle strength [ Time Frame: 22 months ]To evaluate the effect of nusinersen treatment on muscle strength in ambulatory SMA adults
- Change in Six Minute Walk Test [ Time Frame: 22 months ]6 Minute Walk Test (6WMT)
- Change in Hammersmith Functional Motor Scale Expanded [ Time Frame: 22 months ]Hammersmith Functional Motor Scale Expanded (HFMSE)
- Change in SMA Functional Rating Scale [ Time Frame: 22 months ]modified SMA Functional Rating Scale(SMA-FRS)
- Change in Forced Vital Capacity [ Time Frame: 22 months ]Forced Vital Capacity (FVC)
- Change in Negative Inspiratory Force [ Time Frame: 22 months ]Negative Inspiratory Force (NIF)
- Change in lean muscle mass [ Time Frame: 22 months ]Muscle mass measured using Dual-Energy X-Ray Absorptiometry (DXA)
- Change in Quality of Life [ Time Frame: 22 months ]Quality of life will be measured using 36-Item Short Form Survey (SF-36)
- Change in Ulnar and Peroneal muscle measures [ Time Frame: 22 months ]Compound Muscle Action Potential Amplitude (CMAP) will be recorded
- Change in Motor Unit Number Estimation (MUNE) [ Time Frame: 22 months ]Ulnar nerve (Recording at the Abductor Digiti Minimi muscle) MUNE scores will be recorded
- Number of participants that experience Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 22 months ]Number of participants who experience adverse outcomes
Biospecimen Retention: Samples Without DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04591678
|United States, Ohio|
|The Ohio State University|
|Columbus, Ohio, United States, 43210|
|Principal Investigator:||Bakri Elsheikh, MD||Ohio State University|