Adults With SMA Treated With Nusinersen

NCT04591678 | Completed FDA Drug

• 1 other identifier: 2018H0311
• Study Type: observational
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single center, 22-month observational study of nusinersen treatment in adult patients with spinal muscular atrophy (SMA). There will be a total of seven visits. Nusinersen is provided as standard of care and not considered research in this study. Information will be collected regarding the general health, and function including muscle strength of, as well as any positive and/or adverse events experienced by the study participants.

Conditions

Spinal Muscular Atrophy

Keywords

nusinersen; Spinraza

Outcome Measures

Primary Outcomes (1)

• Muscle strength

  To evaluate the effect of nusinersen treatment on muscle strength in ambulatory SMA adults

  22 months

Secondary Outcomes (10)

• Change in Six Minute Walk Test

  22 months

• Change in Hammersmith Functional Motor Scale Expanded

  22 months

• Change in SMA Functional Rating Scale

  22 months

• Change in Forced Vital Capacity

  22 months

• Change in Negative Inspiratory Force

  22 months

Study Arms (1)

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.

Drug: nusinersen

Interventions

nusinersen DRUG

SPINRAZA (nusinersen) is FDA approved to treat spinal muscular atrophy (SMA) through intrathecal injection.

Also known as: SPINRAZA

SMA nusinersen adult cohort

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Adults with spinal muscular atrophy (SMA) as confirmed by clinical and genetic assessments.

You may qualify if:

• Age 18 to 60 years
• Genetic confirmation of 5q SMA documented by the homozygous deletion of both SMN1 genes on standard genetic tests for the disorder
• SMN2 copy number of 3 or greater
• Subjects must be able to walk thirty feet without assistance (i.e. no canes, walkers)
• Interest in participating and the ability to meet the study requirements
• Women of childbearing-age are required to be on birth control or abstain while participating in the research study

You may not qualify if:

• Subjects with history of spinal disease that will interfere with the lumbar puncture procedure
• Subjects with history of bacterial meningitis or encephalitis
• Subjects with history of use of investigational drug treatment for SMA in the last six months, or plan on enrolling in any other treatment trial during the duration of this trial
• History of treatment with gene therapy, stem cell or antisense oligonucleotide
• Patients with co-morbid conditions that preclude travel, testing or study medications
• Patients who are, in the investigator's opinion, mentally or legally incapacitated from providing informed consent for the study, or are otherwise unable to meet study requirements or cooperate reliably with study procedures, especially strength testing
• Women who are pregnant or who intend to become pregnant while participating in the research study or who are breastfeeding

Sponsors & Collaborators

• Ohio State University: lead
• Biogen: collaborator
• Cure SMA: collaborator

Study Sites (1)

The Ohio State University

Columbus, Ohio, 43210, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

blood/serum

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

nusinersen

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Bakri Elsheikh, MD

  Ohio State University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: October 23, 2019
• First Posted: October 19, 2020
• Study Start: October 1, 2018
• Primary Completion: May 14, 2021
• Study Completion: June 25, 2021
• Last Updated: September 22, 2021

Record last verified: 2021-09

Locations

US (1)