A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

Study Identifier:
ISIS 396443-CS11
ClinicalTrials.gov Identifier:
NCT02594124
EudraCT Identifier:
2015-001870-16
EU CT Number:
N/A
Sponsor:
Biogen
Study Complete

Trial Results

Study Protocol
Available Languages: English
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Statistical Analysis Plan
Available Languages: English
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Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: nusinersen
Date
Nov 2015 - Aug 2023
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Signed informed consent of parent or guardian and signed informed assent of participant, if indicated per participant's age and institutional guidelines.
  • Completion of the index study in accordance with the study protocol or as a result of Sponsor decision (e.g., early termination of the index study) within the preceding 16 weeks
Exclusion Criteria
  • Have any condition or worsening condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study
  • Clinically significant abnormalities in hematology or clinical chemistry parameters or electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit that would render the participant unsuitable for participation in the study
  • Participant's parent or legal guardian is not willing or able to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
  • Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer
  • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Protocol Summary

The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.

Trial Locations

Location
Status
Location
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095-8344
Status
N/A
Location
Stanford University School of Medicine
Palo Alto, California, United States, 94305
Status
N/A
Location
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Status
N/A
Location
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
Status
N/A
Location
Nemours Children's Clinic
Orlando, Florida, United States, 32827
Status
N/A
Location
Ann & Robert H. Lurie Children's Hospital of Chicago
New York, Illinois, United States, 60611
Status
N/A
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