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: -

Clinical Trials

A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)

Phase 1

Not yet recruiting

Conditions: Muscular Atrophy, Spinal

Interventions: Drug: Nusinersen
Device: ThecaFlex DRx System

First Posted Date: 2024-08-15

Last Posted Date: 2024-08-15

Lead Sponsor: Biogen

Target Recruit Count: 55

Registration Number: NCT06555419

Antisense Oligonucleotide for Spinal Muscular Atrophy

Conditions: Spinal Muscular Atrophy

Interventions: Drug: Nusinersen

First Posted Date: 2022-01-11

Last Posted Date: 2022-01-26

Lead Sponsor: First Affiliated Hospital of Fujian Medical University

Target Recruit Count: 1000

Registration Number: NCT05187260

Locations: 🇨🇳
Department of Neurology, Peking Union Medical College Hospital, Beijing, Beijing, China
🇨🇳
Department of Pediatrics, Fujian Medical University Union Hospital, Fuzhou, Fujian, China
🇨🇳
Department of Neurology, First Affiliated Hospital Fujian Medical University, Fuzhou, Fujian, China

A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam

Phase 3

Recruiting

Conditions: Spinal Muscular Atrophy

Interventions: Drug: Nusinersen

First Posted Date: 2021-10-05

Last Posted Date: 2024-02-29

Lead Sponsor: Biogen

Target Recruit Count: 45

Registration Number: NCT05067790

Locations: 🇺🇸
Memorial Healthcare, Owosso, Michigan, United States
🇺🇸
Columbia University, New York, New York, United States
🇺🇸
Neurology Rare Disease Center, Denton, Texas, United States

and more 26 locations

Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen

Conditions: Involuntary Movements
MND (Motor Neurone DIsease)
Tremor
SMA

Interventions: Drug: Nusinersen

First Posted Date: 2021-04-01

Last Posted Date: 2021-04-01

Lead Sponsor: University Medical Centre Ljubljana

Target Recruit Count: 110

Registration Number: NCT04825119

Locations: 🇸🇮
University Medical Centre Ljubljana, Ljubljana, Slovenia

Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen

Phase 3

Conditions: Muscular Atrophy, Spinal

Interventions: Drug: Nusinersen

First Posted Date: 2021-01-29

Last Posted Date: 2024-08-21

Lead Sponsor: Biogen

Target Recruit Count: 145

Registration Number: NCT04729907

Locations: 🇨🇴
Hospital Universitario San Ignacio, Bogota, Colombia
🇲🇽
Hospital Infantil de Mexico Federico Gomez, Mexico, Distrito Federal, Mexico
🇷🇺
Russian Children Neuromuscular Center of Veltischev, Moskva, Russian Federation

and more 35 locations

Adults With SMA Treated With Nusinersen

Completed

Conditions: Spinal Muscular Atrophy

Interventions: Drug: nusinersen

First Posted Date: 2020-10-19

Last Posted Date: 2021-09-22

Lead Sponsor: Ohio State University

Target Recruit Count: 15

Registration Number: NCT04591678

Locations: 🇺🇸
The Ohio State University, Columbus, Ohio, United States

Metabolomics of Children With SMA

Completed

Conditions: Spinal Muscular Atrophy

Interventions: Drug: Nusinersen

First Posted Date: 2020-10-14

Last Posted Date: 2022-11-10

Lead Sponsor: University Medical Centre Ljubljana

Target Recruit Count: 35

Registration Number: NCT04587492

Locations: 🇸🇮
University Medical Centre Ljubljana, Ljubljana, Slovenia
🇸🇮
Biotechnical faculty, Ljubljana, Slovenia

Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms)

Not Applicable

Completed

Conditions: Spinal Muscular Atrophy

Interventions: Other: Monthly assessments of functional motor abilities by a trained therapist
Drug: Nusinersen

First Posted Date: 2020-10-06

Last Posted Date: 2024-12-04

Lead Sponsor: CHU de Reims

Target Recruit Count: 4

Registration Number: NCT04576494

Locations: 🇫🇷
Damien JOLLY, Reims, France

A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

Phase 4

Active, not recruiting

Conditions: Muscular Atrophy, Spinal

Interventions: Drug: Nusinersen

First Posted Date: 2020-07-27

Last Posted Date: 2023-12-20

Lead Sponsor: Biogen

Target Recruit Count: 46

Registration Number: NCT04488133

Locations: 🇪🇸
Hospital Sant Joan de Déu, Esplugues Del Llobregat, Barcelona, Spain
🇺🇸
University of Utah, Salt Lake City, Utah, United States
🇮🇹
Fondazione IRCCS Istituto Neurologico Carlo Besta, Milano, Milan, Italy

and more 12 locations

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Phase 3

Completed

Conditions: Muscular Atrophy, Spinal

Interventions: Drug: Nusinersen

First Posted Date: 2019-09-13

Last Posted Date: 2024-07-23

Lead Sponsor: Biogen

Target Recruit Count: 145

Registration Number: NCT04089566

Locations: 🇯🇵
Tokyo Women's Medical University Hospital, Shinjuku-ku, Tokyo-To, Japan
🇵🇱
Uniwersyteckie Centrum Kliniczne, Gdansk, Poland
🇷🇺
Regional Pediatric Clinical Hospital #1, Ekaterinburg, Russian Federation

and more 63 locations

Drug Development Updates

Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.

Home Terms & Conditions Privacy Policy