Status:
COMPLETED
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
Lead Sponsor:
Biogen
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
2-14 years
Phase:
PHASE1
Brief Summary
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscula...
Detailed Description
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc. In August 2016, sponsorship of the trial was transferred to Biogen.
Eligibility Criteria
Inclusion
- Key
- Documented Survival Motor Neuron1 (SMN1) homozygous gene deletion
- Clinical signs attributable to Spinal Muscular Atrophy (SMA)
- 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
- Key
Exclusion
- Respiratory insufficiency defined by the need for invasive or non-invasive ventilation during a 24 hour period
- Presence of a gastric feeding tube
- Previous scoliosis surgery or scoliosis surgery planned during the duration of the study that would interfere with the lumbar puncture (LP) injection procedure
- Hospitalization for surgery or pulmonary event within the last 2 months or planned during the study
- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy
- History of brain or spinal cord disease that would interfere with LP procedures or cerebrospinal fluid (CSF) circulation
- Presence of an implanted shunt for the draining of CSF or an implanted Central Nervous System (CNS) catheter
- History of bacterial meningitis
- Clinically significant abnormalities in hematology or clinical chemistry parameters
- Treatment with another investigational drug, biological agent, or device within 1-month of Screening or 5 half-lives of study agent whichever is longer. Any history of gene therapy or cell transplantation
- Ongoing medical condition that would interfere with the conduct and assessments of the study. Examples are medical disability (e.g. wasting or cachexia, severe anemia, etc.) that would interfere with the assessment of safety or would compromised the ability of the participant to undergo study procedures
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Key Trial Info
Start Date :
November 30 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 31 2013
Estimated Enrollment :
28 Patients enrolled
Trial Details
Trial ID
NCT01494701
Start Date
November 30 2011
End Date
January 31 2013
Last Update
February 18 2021
Active Locations (4)
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1
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
2
Columbia University Medical Center
New York, New York, United States, 10032
3
UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States, 75207
4
University of Utah School of Medicine
Salt Lake City, Utah, United States, 84132