Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
Completed · Phase 3
Conditions studied: Muscular Atrophy, Spinal
In brief
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C). The secondary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to examine the effect of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen administered intrathecally at higher doses compared to the currently approved dose in participants with SMA (Part B).
Key facts
- Study ID
- NCT04089566
- Run by
- Biogen
- People needed
- 145
- Starts
- 2020-03-26
- Expected to finish
- 2024-05-30
- Last updated by the study team
- 2025-06-05
Who can join
Age: 0 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Stanford Hospital and Clinics — Palo Alto, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- The Johns Hopkins Hospital — Baltimore, Maryland, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States
- The University of Texas Southwestern Medical Center — Dallas, Texas, United States
- HC-UFMG - Hospital das Clinicas da Universidade Federal de Minas Gerais — Belo Horizonte, Minas Gerais, Brazil
- Hospital de Clínicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil
- Hospital das Clínicas da Faculdade de Medicina da USP — São Paulo, São Paulo, Brazil
- London Health Sciences Centre (LHSC) - Children's Hospital — London, Ontario, Canada
- Hospital Luis Calvo Mackenna — Santiago, Chile
- Clinica Las Condes — Santiago, Chile
- Clinica MEDS La Dehesa — Santiago, Chile
- Peking University First Hospital — Beijing, Beijing Municipality, China
- The Children's Hospital of Zhejiang University school of Med_Hangzhou — Hangzhou, Binjiang, China
- Guangzhou Woman and Children's Medical Center — Guangzhou, Guangzhou, China
- Xiangya Hospital, Central South University — Changsha, Hu'nan, China
- Hospital Universitario San Ignacio — Bogotá, Colombia
- Fundacion Hospitalaria San Vicente de Paul — Medellín, Colombia
- Tallinn Children's Hospital — Tallinn, Estonia
- Universitaetsklinikum Freiburg — Freiburg im Breisgau, Baden-Wurttemberg, Germany
- Universitaetsklinikum Giessen und Marburg GmbH — Giessen, Hesse, Germany
- Semmelweis Egyetem — Budapest, Hungary
- Magyarorszagi Reformatus Egyhaz Bethesda Gyermekkorhaz — Budapest, Hungary
Full record on ClinicalTrials.gov
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