17 completed, 3 recruiting, 2 active not recruiting, 2 terminated. 6 Phase 3, 5 Phase 1, 5 Phase 2, 1 Early Phase 1, 1 Not applicable.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix .
Trial Phase Status Sponsor Enrollment Start Updated
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy Apitegromab, Nusinersen, Risdiplam - Spinal Muscular Atrophy, SMA Phase 2 Recruiting Scholar Rock, Inc. 52 2025-09-15 2026-10-02 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) Nusinersen, ThecaFlex DRx System - Muscular Atrophy, Spinal Phase 1 Recruiting Biogen 78 2025-01-16 2026-09-03 Adult SMA Research and Clinical Hub Risdiplam, Nusinersen Injectable Product - Spinal Muscular Atrophy (SMA) Recruiting Newcastle-upon-Tyne Hospitals NHS Trust 600 2022-07-29 2025-05-18 Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) Monthly assessments of functional motor abilities by a trained therapist, Nusinersen - Spinal Muscular Atrophy Not applicable Completed CHU de Reims 4 2022-01-24 2024-12-04 A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) Nusinersen - Spinal Muscular Atrophy Phase 3 Active not recruiting Biogen 45 2022-01-21 2026-05-22 Antisense Oligonucleotide for Spinal Muscular Atrophy Nusinersen - Spinal Muscular Atrophy Unknown First Affiliated Hospital of Fujian Medical University 1000 2022-01-01 2022-01-26 A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD) Nusinersen - Muscular Atrophy, Spinal Phase 3 Completed Biogen 115 2021-04-19 2026-08-11 A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND) Nusinersen - Muscular Atrophy, Spinal Phase 4 Completed Biogen 46 2021-01-04 2025-10-14 Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection Nusinersen Sodium Injection - Muscular Atrophy, Spinal Completed Biogen 50 2020-11-18 2023-12-07 Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study Nusinersen Injectable Product - Spinal Muscular Atrophy Completed Hospital Israelita Albert Einstein 155 2020-05-27 2021-04-27 Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Nusinersen - Muscular Atrophy, Spinal Phase 3 Completed Biogen 145 2020-03-26 2025-06-05 Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments Nusinersen Treatments - Spinal Muscular Atrophy Early Phase 1 Withdrawn NYU Langone Health 2019-07-17 2021-06-29 Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) Nusinersen Sodium Injection - Muscular Atrophy, Spinal Active not recruiting Biogen 74 2019-07-02 2026-07-27 Effect of Nusinersen on Adults With Spinal Muscular Atrophy nusinersen - Adult Spinal Muscular Atrophy Completed Northwell Health 12 2019-04-01 2023-03-20 Adults With SMA Treated With Nusinersen nusinersen - Spinal Muscular Atrophy Completed Ohio State University 15 2018-10-01 2021-09-22 Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen Nusinersen - Tremor, Involuntary Movements Unknown University Medical Centre Ljubljana 110 2017-10-01 2021-04-01 Metabolomics of Children With SMA Nusinersen - Spinal Muscular Atrophy Completed University Medical Centre Ljubljana 35 2017-01-01 2022-11-10 A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies nusinersen - Spinal Muscular Atrophy Phase 3 Completed Biogen 292 2015-11-04 2024-10-22 A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). Nusinersen, Sham Procedure - Spinal Muscular Atrophy Phase 2 Terminated Biogen 21 2015-08-19 2021-02-17 A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy Nusinersen - Spinal Muscular Atrophy Phase 2 Completed Biogen 25 2015-05-18 2025-10-20
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Conditions studied Muscular Atrophy, Spinal (27) · Spinal Muscular Atrophies of Childhood (2) · Amyotrophic Lateral Sclerosis (1) · Charcot-Marie-Tooth disease, Type 1F (1) · Dyskinesias (1) · Muscular Atrophy (1) · Neuromuscular Manifestations (1) · Tremor (1)
Sponsors Biogen (18) · University Medical Centre Ljubljana (2) · CHU de Reims (1) · First Affiliated Hospital of Fujian Medical University (1) · Hospital Israelita Albert Einstein (1) · NYU Langone Health (1) · Newcastle-upon-Tyne Hospitals NHS Trust (1) · Northwell Health (1) · Ohio State University (1) · Scholar Rock, Inc. (1)
Countries United States (19) · Germany (10) · Italy (10) · Spain (8) · Canada (6) · France (6) · Japan (6) · United Kingdom (5) · China (4) · South Korea (4) · Turkey (Türkiye) (4) · Australia (3) · Belgium (3) · Brazil (3) · Colombia (3)
Top investigators Anthony Geraci, MD (1) · Bakri Elsheikh, MD (1) · Edmar Zanoteli, PhD (1) · Elice Batista, PhD (1) · Melodi Pirzada, MD (1) · Prof Chiara Marini-Bettolo, MD, PhD (1) · Vanessa Teich, PhD (1) · Wan-Jin Chen (1)
Results publications PMID 31420846 , PMID 27939059 , PMID 26823478 , PMID 29091570 , PMID 29443664 , PMID 38705943 , PMID 30879249 , PMID 31019106 , PMID 33501671 , PMID 34089650 , PMID 35567345 , PMID 37409780 , PMID 38722537 , PMID 40956616 , PMID 41512690 , PMID 41634391 , PMID 41830075 , PMID 42104555 , PMID 42413222
NIH funding 2014 (3), 2023 (3), 2017 (2), 2015 (1), 2019 (1)