An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)

Study Identifier:
ISIS 396443-CS10
ClinicalTrials.gov Identifier:
NCT01780246
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Biogen
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: nusinersen
Date
Jan 2013 - Feb 2014
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 2 - 15 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Clinical signs attributable to Spinal Muscular Atrophy
  • Satisfactory completion of dosing and all study visits in ISIS 396443-CS1 (NCT01494701) with an acceptable safety profile, per Investigator judgement.
  • Able to complete all study procedures, measurements and visits and parent/participant has adequately supportive psychosocial circumstances, in the opinion of the investigator
  • Estimated life expectancy \> 2 years from Screening
  • Meets age-appropriate institutional criteria for use of anesthesia/sedation, if use is planned for study procedure
Exclusion Criteria
  • Have any new or worsening of existing 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.
  • Dosing in ISIS 396443-CS1 (NCT01494701) within 270 days (9 months) of screening, or longer ago than 450 days (15 months)
  • Dosing in ISIS 396443-CS2 (NCT01703988)
  • Hospitalization for surgery (i.e. scoliosis surgery) or pulmonary event within 2 months of screening or planned during the duration of the study
  • Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy any time during the screening period
  • Clinically significant abnormalities in hematology or clinical chemistry parameters
  • Treatment with investigational drug, biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Treatment with valproate or hydroxyurea within 1 months of screening. Any history of gene therapy or cell transplantation
  • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Protocol Summary

The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS 396443-CS1 (NCT02865109). The secondary objective was to examine the plasma pharmacokinetics of a single dose of ISIS 396443 administered intrathecally to participants with SMA who previously participated in ISIS 396443-CS1.

Trial Locations

Location
Status
Location
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Status
N/A
Location
Columbia University Medical Center
New York, New York, United States, 10032
Status
N/A
Location
UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States, 75207
Status
N/A
Location
University of Utah School of Medicine
Salt Lake City, Utah, United States, 84132
Status
N/A

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