Spinal muscular atrophy: trials for referral

71 trials shown

• No phase23 sites

  RecruitingGermany, Spain, United Kingdom, Poland, United States

  NCT05866419

  Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

  Alcyone Therapeutics, Inc

  Registry updated 9 Nov 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 46 sites

  RecruitingUnited States

  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)

  Novartis Pharmaceuticals

  Registry updated 29 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Early Phase 11 site

  Not yet recruitingIran

  NCT07903376

  SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)

  Exir Gene Pharmed

  Registry updated 29 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• No phase1 site

  RecruitingBelgium

  NCT07894185

  SMAdult - Monitoring of Movement on Spinal Muscular Atrophy

  Centre Hospitalier Universitaire de Liege

  Registry updated 26 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• No phase1 site

  RecruitingChina

  NCT07822711

  Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA

  Peking University Third Hospital

  Registry updated 23 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 328 sites

  RecruitingAustralia, Brazil, Chile, China, Germany, Spain

  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)

  Biogen

  Pre-treatment window Registry sentence: “Any prior treatment with an approved SMA disease-modifying therapy (e.g., nusinersen, onasemnogene abeparvovec-xioi \[OA\], and/or risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA.”

  Registry updated 23 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 322 sites

  RecruitingBelgium, Germany, France, Croatia, Hungary, Ireland

  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

  Biogen

  Pre-treatment window Registry sentence: “Other than OA, any prior treatment with an approved SMA disease modifying therapy (e.g. nusinersen and/or risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA.”

  Registry updated 15 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 1/23 sites

  RecruitingChina

  NCT07554924

  A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

  Lanyue Biotech (Hangzhou) Co., Ltd.

  Registry updated 14 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 228 sites

  RecruitingAustralia, Canada, United Kingdom, United States

  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

  argenx

  Registry updated 8 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 223 sites

  RecruitingBelgium, Spain, France, United Kingdom, Italy, Netherlands

  NCT07047144

  A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

  Scholar Rock, Inc.

  Registry updated 2 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 33 sites

  RecruitingChina, United States

  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

  Biogen

  Pre-treatment window Registry sentence: “Participants with no prior treatment with myostatin inhibitors and a willingness to remain off concurrent myostatin inhibitor therapy for the duration of the study.”

  Registry updated 2 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 31 site

  Not yet recruitingCanada

  NCT07846696

  An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)

  London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's

  Registry updated 29 Sept 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• No phase1 site

  Not yet recruitingCanada

  NCT07838779

  An OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen

  London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's

  Registry updated 24 Sept 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• No phase1 site

  By invitation onlyChina

  NCT06772402

  GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy

  Genecombio Ltd.

  Registry updated 11 Sept 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 124 sites

  RecruitingGermany, Spain, France, United Kingdom, Italy, Poland

  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)

  Biogen

  Registry updated 3 Sept 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 1/21 site

  RecruitingBrazil

  NCT07070999

  Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

  Gemma Biotherapeutics

  Registry updated 29 Jun 2026

  Registry record: ClinicalTrials.gov
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• No phase8 sites

  RecruitingFrance

  NCT06321965

  Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

  Hospices Civils de Lyon

  Registry updated 30 Jan 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• No phase2 sites

  Not yet recruitingSpain, Mexico

  NCT07159360

  Safety, Usability, and Effectiveness of a Gait Exoskeleton for Children and Adolescents With Neurodevelopmental Disorders.

  MarsiBionics

  Registry updated 8 Sept 2025

  Registry record: ClinicalTrials.gov
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• No phase1 site

  By invitation onlyGermany

  NCT07072676

  The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

  LMU Klinikum

  Registry updated 18 Jul 2025

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 1/24 sites

  RecruitingChina

  NCT05824169

  Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

  GeneCradle Inc

  Registry updated 3 Jul 2025

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 1/21 site

  RecruitingChina

  NCT06421831

  Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

  GeneCradle Inc

  Registry updated 3 Jul 2025

  Registry record: ClinicalTrials.gov
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• No phase1 site

  RecruitingUkraine

  NCT06977269

  Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy

  Charitable Foundation Children with Spinal Muscular Atrophy

  Registry updated 18 Jun 2025

  Registry record: ClinicalTrials.gov
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• Phase 37 sites

  RecruitingChina

  NCT06971094

  Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III

  GeneCradle Inc

  Registry updated 5 Jun 2025

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Early Phase 12 sites

  By invitation onlyChina

  NCT06888661

  Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy

  Guangzhou Jiayin Biotech Ltd

  Registry updated 21 Mar 2025

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 1/26 sites

  RecruitingBelarus, Russia

  NCT05747261

  Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)

  Biocad

  Registry updated 22 Feb 2024

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 1/22 sites

  Not yet recruitingFrance

  2026-527323-10-00

  AAV9-ASAH1 intracisterna magna Gene Therapy (GNT0009) in Patients with Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA - PME)

  Genethon

  Registry updated 9 Nov 2026

  Registry record: EU CTIS
  Compare criteriaReferral packLetter to the site

• No phase1 site

  RecruitingFrance

  NCT07321977

  Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers

  Institut de Myologie, France

  Registry updated 3 Nov 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• No phase1 site

  RecruitingUnited Kingdom

  NCT07543003

  An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)

  University of Oxford

  Registry updated 2 Nov 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site

• Phase 211 sites

  RecruitingBelgium, Spain, France, Italy, Netherlands

  2024-520130-29-00

  A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

  Scholar Rock Inc.

  Registry updated 29 Oct 2026

  Registry record: EU CTIS
  Compare criteriaReferral packLetter to the site

• No phase1 site

  RecruitingGermany

  NCT07900672

  Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders

  Ambulanzpartner Soziotechnologie APST GmbH

  Registry updated 28 Oct 2026

  Registry record: ClinicalTrials.gov
  Compare criteriaReferral packLetter to the site