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



Status:Active, not recruiting
Conditions:Neurology, Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:Any
Updated:2/1/2019
Start Date:November 4, 2015
End Date:August 29, 2023

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An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443

The primary objective is to evaluate the 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.

This study was initiated and the protocol was registered by Ionis Pharmaceuticals, Inc.

In August 2016, Biogen assumed responsibility for this study.

Key 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

Key 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.
We found this trial at
16
sites
282 Washington St
Hartford, Connecticut 06106
(860) 545-9000
Connecticut Children's Medical Center Connecticut Children’s Medical Center is a nationally recognized, 187-bed not-for-profit children’s...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Philadelphia, PA
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Aurora, CO
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Boston, MA
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225 East Chicago Avenue
Chicago, Illinois 60611
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Chicago, IL
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Dallas, TX
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Durham, North Carolina 27710
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Durham, NC
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757 Westwood Plaza
Los Angeles, California 90024
(310) 825-9111
UCLA Medical Center Founded in 1955, UCLA Medical Center became Ronald Reagan UCLA Medical Center...
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Los Angeles, CA
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Minneapolis, MN
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630 W 168th St
New York, New York
212-305-2862
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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New York, NY
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13535 Nemours Parkway
Orlando, Florida 32827
(407) 567-4000
Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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Orlando, FL
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Palo Alto, California 94304
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Portland, Oregon 97239
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660 S Euclid Ave
Saint Louis, Missouri 63110
(314) 362-5000
Washington University School of Medicine Washington University Physicians is the clinical practice of the School...
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Salt Lake City, UT
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Sydney, New South Wales
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Sydney,
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