Spinal muscular atrophy: open trials in United States

17 open trials have a site in United States. Registry data; only the trial team decides who can take part.

NCT07448610
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
Trial Treatment · Phase 4 · Novartis Pharmaceuticals
Boston Childrens Hospital, Boston · Ohio State University Medical Center, Columbus · Arkansas Children's Hospital, Little Rock · Columbia University Medical Center, New York · University of Pennsylvania, Philadelphia · Stanford Hospital, Stanford
Registry updated 2026-10-29
NCT07221669
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
Trial Treatment · Phase 3 · Biogen
Ann and Robert H Lurie Childrens Hospital of Chicago, Chicago · Neurology Rare Disease Center, Flower Mound · Childrens Hospital of the Kings Daughter Norfolk, Norfolk
Registry updated 2026-10-23
NCT07488338
HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
Trial Treatment · Teachers College, Columbia University
Center for Cerebral Palsy Research, Teachers College, Columbia University, New York
Registry updated 2026-10-22
NCT07805278
A Registry of Cases of Spinal Muscular Atrophy
Research · St. Jude Children's Research Hospital
St. Jude Children's Research Hospital, Memphis
Registry updated 2026-10-22
NCT07444450
A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
Trial Treatment · Phase 3 · Biogen
Neurology Rare Disease Center, Flower Mound
Registry updated 2026-10-15
NCT06955897
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Research · Columbia University
Columbia University Irving Medical Center, New York
Registry updated 2026-10-14
NCT07287982
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Trial Treatment · Phase 2 · argenx
The Johns Hopkins Hospital, Baltimore · Boston Children's Hospital, Boston · Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago · The Curators of the University of Missouri on behalf of University of Missouri Health Care, Columbia · Neurology Rare Disease Center, Flower Mound · Connecticut Children's Medical Center, Hartford · Rare Disease Research NC, LLC, Hillsborough · Texas Children's Hospital, Houston · University of Iowa Stead Family Children's Hospital, Iowa City · Rare Disease Research FL LLC, Kissimmee · Arkansas Children's Hospital, Little Rock · Columbia University / Herbert Irving Comprehensive Cancer Center, New York · Childrens Hospital of The Kings Daughters, Norfolk · Children's Hospital Philadelphia - Neurology, Philadelphia · Rady Childrens Hospital, San Diego · Stanford University Medical Center, Stanford
Registry updated 2026-10-08
NCT07047144
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Trial Treatment · Phase 2 · Scholar Rock, Inc.
Children's Healthcare of Atlanta, Atlanta · Children's Hospital Colorado, Aurora · University of Texas Southwestern Medical Center, Dallas · Neurology Rare Disease Center, Flower Mound · Cook Children's Medical Center, Fort Worth · Helen DeVos Children's Hospital at Spectrum Health, Grand Rapids · Texas Children's Hospital (TCH)-Clinical Care Center (CCC), Houston · University of Iowa, Iowa City · Children's Hospital of Orange County (CHOC), Orange · Stanford Neuroscience Health Center (SNHC), Palo Alto · The Children's Hospital Of Philadelphia, Philadelphia · Phoenix Children s Hospital, Phoenix
Registry updated 2026-10-02
NCT07444476
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
Trial Treatment · Phase 3 · Biogen
Childrens Hospital of the Kings Daughter Norfolk, Norfolk · Stanford University Medical Center, Palo Alto
Registry updated 2026-10-02
NCT06555419
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)
Trial Treatment · Phase 1 · Biogen
Boston Children's Hospital, Boston · Stacey Hall Developmental Pediatrics, Charlottesville · Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago · Helen DeVos Children's Hospita, Grand Rapids · Milton S. Hershey Medical Center | Pennsylvania State University_Hershey, Hershey · Texas Childrens Hospital Houston, Houston · Children's Hospital of the King's Daughters_Norfolk, Norfolk · Children's Hospital of Orange County, Orange · Nemours Children's Hospital - Orlando, Orlando · Stanford University Medical Center | Department of Neurology_Palo Alto, Palo Alto · Children's Hospital of Philadelphia, Philadelphia · St. Joseph's Hospital and Medical Center/Barrow Neurological Institute, Phoenix
Registry updated 2026-09-03
NCT05866419
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Trial Treatment · Alcyone Therapeutics, Inc
Boston Children's Hospital, Boston · University of Virginia, Charlottesville · Lurie Childrens Hospital, Chicago · UH Rainbow Babies and Children's Hospital, Cleveland · Helen DeVos Children's Hospital, Grand Rapids · Penn State Milton S. Hershey Medical Center, Hershey · Texas Children's Hospital, Houston · Columbia University Irving Medical Center / NewYork-Presbyterian Hospital, New York · Childrens Hospital of the King's Daughters, Norfolk · Children's Hospital Orange County, Orange · Nemours Children's Hospital, Florida, Orlando · Stanford Medical Center, Palo Alto · Children's Hospital Philadelphia, Philadelphia · Barrow Neurological Institute, Phoenix · Rady Children's Hospital, San Diego
Registry updated 2026-08-19
NCT06532474
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
Research · St. Jude Children's Research Hospital
St. Jude Children's Research Hospital, Memphis
Registry updated 2026-07-09
NCT06839469
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
Research · Columbia University
Boston Children's Hospital, Boston · Columbia University Irving Medical Center, New York · Stanford University, Palo Alto
Registry updated 2026-04-09
NCT07400198
Gait and Bone Health in SMA
Research · Jacqueline Montes
Columbia University Irving Medical Center, New York
Registry updated 2026-03-18
NCT05789758
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
Research · Biogen
Boston Children's Hospital, Boston · Cambridge · Columbia University Irving Medical Center, New York · Stanford Neuroscience Health Center, Palo Alto
Registry updated 2026-03-09
NCT07223489
Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
Research · Medstar Health Research Institute
MedStar Health Research Institute, Washinton
Registry updated 2026-01-16
NCT04174157
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Research · Novartis Pharmaceuticals
Akron Children's Hospital, Akron · Children's Hospital Colorado, Aurora · Child Neurology Consultants of Austin, Austin · University of Virginia Health System, Charlottesville · Cincinnati Children's Hospital Medical Center, Cincinnati · University Hospitals, Cleveland · University of Missouri Health System, Columbia · Nationwide Children's Hospital, Columbus · Children's Health, Dallas · Duke Health, Durham · Connecticut Children's Medical Center, Farmington · Golisano Children's Hospital of Southwest Florida, Fort Myers · Cook Children's, Fort Worth · Prisma Health, Greenville · Penn State Hershey, Hershey · Texas Children's Hospital, Houston · Indiana University Health University Hospital (IUHUH), Indianapolis · University of Iowa, Iowa City · University of Kansas Medical Center, Kansas City · Arkansas Children's Hospital, Little Rock · Loma Linda University Health, Loma Linda · University of California Los Angeles Health, Los Angeles · Children's Hospital of Los Angeles, Los Angeles · University of Louisville, Louisville · Children's Hospital of Orange County, Madera · Valley Children's Healthcare, Madera · University of Wisconsin, Madison · Methodist Le Bonheur Healthcare, Memphis · Nicklaus Children's Hospital, Miami · Children's Hospital of Wisconsin, Milwaukee · University of Minnesota, Minneapolis · Atlantic Health System, Morristown · Yale-New Haven Health System, New Haven · Children's Hospital of The King's Daughters, Norfolk · Oklahoma University Medical Center, Oklahoma City · Adventist Health System - Florida, Orlando · Jackson South Medical Center, Palmetto Bay · Phoenix Children s Hospital, Phoenix · University of Pittsburgh Medical Center, Pittsburgh · Oregon Health and Science University, Portland · Virginia Commonwealth University Health System, Richmond · University of Rochester Medical Center, Rochester · University of California Davis Health System, Sacramento · University of Utah, Salt Lake City · CHRISTUS Health, San Antonio · Rady Children's Hospital - San Diego, San Diego · Seattle Children's, Seattle · Washington University School of Medicine in St. Louis, St Louis · The State University of New York, Stony Brook · MultiCare Health System, Tacoma
Registry updated 2025-01-31

All Spinal muscular atrophy trials and the map

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