Nusinersen
- Generic Name
- Nusinersen
- Brand Names
- Spinraza
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1258984-36-9
- Unique Ingredient Identifier
- 5Z9SP3X666
- Background
An antisense oligonucleotide that induces survival motor neuron (SMN) protein expression, it was approved by the U.S. FDA in December, 2016 as Spinraza for the treatment of children and adults with spinal muscular atrophy (SMA). It is adminstrated as direct intrathecal injection.
- Indication
Indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
- Associated Conditions
- Spinal Muscular Atrophy (SMA)
- Associated Therapies
- -
Effect of Nusinersen on Adults With Spinal Muscular Atrophy
- First Posted Date
- 2019-03-18
- Last Posted Date
- 2023-03-20
- Lead Sponsor
- Northwell Health
- Target Recruit Count
- 12
- Registration Number
- NCT03878030
- Locations
- 🇺🇸
Northwell Health Neuroscience, Great Neck, New York, United States
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
- Conditions
- First Posted Date
- 2016-08-12
- Last Posted Date
- 2021-04-05
- Lead Sponsor
- Biogen
- Registration Number
- NCT02865109
- Locations
- 🇳🇿
Auckland District Health Board ADHB, Grafton, Auckland, New Zealand
🇹🇷Erciyes University Hospital, Kayseri, Anatolia, Turkey
🇳🇿Auckland City Hospital, Grafton, Auckland, New Zealand
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
- First Posted Date
- 2015-11-01
- Last Posted Date
- 2024-10-22
- Lead Sponsor
- Biogen
- Target Recruit Count
- 292
- Registration Number
- NCT02594124
- Locations
- 🇺🇸
The Johns Hopkins Hospital, Baltimore, Maryland, United States
🇧🇪Universitair Kinderziekenhuis Koningin Fabiola, Brussels, Belgium
🇮🇹Pediatric Neurology Unit, Catholic University, Essen, Italy
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
- Conditions
- Interventions
- First Posted Date
- 2015-06-04
- Last Posted Date
- 2021-02-17
- Lead Sponsor
- Biogen
- Target Recruit Count
- 21
- Registration Number
- NCT02462759
- Locations
- 🇺🇸
The Johns Hopkins Hospital, Baltimore, Maryland, United States
🇺🇸Connecticut Childrens Medical, Hartford, Connecticut, United States
🇺🇸The University of Texas Southwestern Medical Center, Dallas, Texas, United States
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
- First Posted Date
- 2015-03-12
- Last Posted Date
- 2024-05-16
- Lead Sponsor
- Biogen
- Target Recruit Count
- 25
- Registration Number
- NCT02386553
- Locations
- 🇺🇸
The Johns Hopkins Hospital, Baltimore, Maryland, United States
🇺🇸Massachusetts General Hospital, Boston, Massachusetts, United States
🇺🇸Nemours Children's Hospital, Orlando, Orlando, Florida, United States
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
- Conditions
- Interventions
- First Posted Date
- 2014-11-17
- Last Posted Date
- 2021-02-17
- Lead Sponsor
- Biogen
- Target Recruit Count
- 126
- Registration Number
- NCT02292537
- Locations
- 🇺🇸
UCLA Clinical and Translational Research Center, Los Angeles, California, United States
🇺🇸Lucile Packard Children's Hospital at Stanford, Palo Alto, California, United States
🇺🇸Children's Hospital Colorado, Aurora, Colorado, United States
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
- Conditions
- Interventions
- First Posted Date
- 2014-07-17
- Last Posted Date
- 2021-02-17
- Lead Sponsor
- Biogen
- Target Recruit Count
- 122
- Registration Number
- NCT02193074
- Locations
- 🇺🇸
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸Children's Hospital of Philadelphia - Neurology, Philadelphia, Pennsylvania, United States
🇺🇸UT Southwestern Medical Center/Children's Medical Center Dallas, Dallas, Texas, United States
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
- First Posted Date
- 2014-02-03
- Last Posted Date
- 2021-02-16
- Lead Sponsor
- Biogen
- Target Recruit Count
- 47
- Registration Number
- NCT02052791
- Locations
- 🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸UT Southwestern Medical Center - Children's Medical Center Dallas, Dallas, Texas, United States
🇺🇸University of Utah School of Medicine, Salt Lake City, Utah, United States
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
- First Posted Date
- 2013-04-25
- Last Posted Date
- 2021-02-17
- Lead Sponsor
- Biogen
- Target Recruit Count
- 21
- Registration Number
- NCT01839656
- Locations
- 🇺🇸
Nemours Children's Hospital, Orlando, Florida, United States
🇨🇦The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada
🇺🇸Stanford University Medical Center, Stanford, California, United States
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)
- First Posted Date
- 2013-01-31
- Last Posted Date
- 2021-02-16
- Lead Sponsor
- Biogen
- Target Recruit Count
- 18
- Registration Number
- NCT01780246
- Locations
- 🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸University of Utah School of Medicine, Salt Lake City, Utah, United States
🇺🇸Columbia University Medical Center, New York, New York, United States
Drug Development Updates
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