Adults With SMA Treated With Nusinersen

Study Identifier:
2018H0311
ClinicalTrials.gov Identifier:
NCT04591678
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Ohio State University
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: nusinersen
Date
Oct 2018 - May 2021
Patient Requirements
Sex: Female & Male
Age: 18 - 60 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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
Exclusion Criteria
  • 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

Protocol 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.

Trial Locations

Location
Status
Location
The Ohio State University
Columbus, Ohio, United States, 43210
Status
N/A

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