Clinical trials for Spinal Muscular Atrophy
52 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- RECRUITINGN/ANCT07488338HABIT-ILE + FST in Children With SMA: Preliminary EffectivenessTeachers College, Columbia University
- RECRUITINGNCT07400198Gait and Bone Health in SMAJacqueline Montes
- RECRUITINGPHASE1, PHASE2NCT07070999Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1Gemma Biotherapeutics · PI: May Orfali, MD
- RECRUITINGN/ANCT07321977Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic VolunteersInstitut de Myologie, France · PI: Pauline SANTMARTY, MsC
- RECRUITINGPhase 2NCT07287982A 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 Atrophyargenx
- RECRUITINGN/ANCT07286565Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn ScreeningCentre Hospitalier Universitaire de Liege · PI: Tamara Dangouloff, PhD
- RECRUITINGNCT06532474Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed TherapiesSt. Jude Children's Research Hospital · PI: Richard Finkel, MD
- RECRUITINGPhase 3NCT07265232Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).Lantu Biopharma
- RECRUITINGN/ANCT07223489Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health SystemMedstar Health Research Institute · PI: Gabrielle Edwards, BS
- ACTIVE NOT RECRUITINGN/ANCT07223320HABIT-ILE + FST in Children With SMATeachers College, Columbia University
- RECRUITINGPhase 3NCT06971094Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase IIIGeneCradle Inc · PI: GeneCradle, Inc China
- RECRUITINGN/ANCT06977269Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular AtrophyCharitable Foundation Children with Spinal Muscular Atrophy · PI: Olena V Pisotska, MD, PhD
- RECRUITINGN/ANCT07332702Long Read Analysis in Spinal Muscular Atrophy - LOREASIUniversity Hospital, Rouen · PI: Pascale Saugier-Veber, PharmD PhD
- RECRUITINGN/ANCT06955897Characterizing Perceived Physical Fatigability in Nusinersen-treated SMAColumbia University · PI: Jacqueline Montes, Pt, EdD
- ENROLLING BY INVITATIONEarly Phase 1NCT06888661Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular AtrophyGuangzhou Jiayin Biotech Ltd
- ENROLLING BY INVITATIONNCT06588803A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMAGuangzhou Jiayin Biotech Ltd · PI: YI WANG, Ph D.
- RECRUITINGN/ANCT06648486Robot-assisted TrainingPeking University Third Hospital · PI: Fuzhen Yuan, Dr.
- RECRUITINGN/ANCT06396325A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular AtrophyMaryam Oskoui, MD, MSc, FRCPC, FAAN · PI: Maryam Oskoui
- RECRUITINGPHASE1, PHASE2NCT06288230An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular AtrophyLantu Biopharma
- RECRUITINGN/ANCT06321965Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring TherapyHospices Civils de Lyon · PI: Laure LE GOFF, PI
- RECRUITINGPHASE1, PHASE2NCT06421831Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 PatientsGeneCradle Inc
- ACTIVE NOT RECRUITINGNCT06599606Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular AtrophyIstanbul University - Cerrahpasa
- RECRUITINGN/ANCT06019637A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With ZolgensmaNovartis Pharmaceuticals · PI: Novartis Pharmaceuticals
- RECRUITINGNCT06310421Spinal Muscular Atrophy Neonatal Screening ProgramIRCCS Burlo Garofolo
- ACTIVE NOT RECRUITINGPhase 2NCT05794139Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular AtrophyNMD Pharma A/S
- ACTIVE NOT RECRUITINGN/ANCT06191354A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1Kun Sun · PI: Kun Sun, Doctor
- ACTIVE NOT RECRUITINGPhase 3NCT05626855Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of ApitegromabScholar Rock, Inc.
- ENROLLING BY INVITATIONPHASE1, PHASE2NCT05614531Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1Guangzhou Jiayin Biotech Ltd
- RECRUITINGPHASE1, PHASE2NCT05747261Study 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 · PI: Arina V Zinkina-Orikhan, PhD
- RECRUITINGPhase 3NCT05335876Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical TrialsNovartis Pharmaceuticals · PI: Novartis Pharmaceuticals
- RECRUITINGN/ANCT05768048Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying TreatmentsFondazione Policlinico Universitario Agostino Gemelli IRCCS · PI: Eugenio Mercuri, MD
- ACTIVE NOT RECRUITINGPhase 4NCT05522361Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With NusinersenClinic for Special Children
- RECRUITINGNCT06978985Adult SMA Research and Clinical HubNewcastle-upon-Tyne Hospitals NHS Trust · PI: Prof Chiara Marini-Bettolo, MD, PhD
- ACTIVE NOT RECRUITINGPhase 3NCT05337553A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular AtrophyBiohaven Pharmaceuticals, Inc. · PI: Lindsey Lair, MD
- ACTIVE NOT RECRUITINGPhase 2NCT05115110A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular AtrophyHoffmann-La Roche · PI: Clinical Trials
- RECRUITINGN/ANCT05341453Hippotherapy in Children With Spinal Muscular AtrophyCharles University, Czech Republic · PI: Kamila Rasova, as.prof.Dr.
- ACTIVE NOT RECRUITINGPhase 4NCT05232929Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)Genentech, Inc. · PI: Clinical Trials
- ACTIVE NOT RECRUITINGPhase 3NCT05067790A 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)Biogen · PI: Medical Director
- ACTIVE NOT RECRUITINGN/ANCT05117827Pediatric Powered Wheelchair Standing Devices: An Exploratory StudyGrand Valley State University
- ACTIVE NOT RECRUITINGN/ANCT04690998Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV PatientsAssistance Publique Hopitaux De Marseille
- ACTIVE NOT RECRUITINGN/ANCT04833348Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative TherapiesAssistance Publique - Hôpitaux de Paris · PI: Isabelle DESGUERRE, MD, PhD
- RECRUITINGNCT07231549CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in SpainFundació Sant Joan de Déu · PI: Andrés Nascimento
- ACTIVE NOT RECRUITINGN/ANCT04159987Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®Centre Hospitalier Universitaire de Nice
- ACTIVE NOT RECRUITINGPhase 3NCT04042025Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioiNovartis Gene Therapies · PI: Sitra Tauscher-Wisniewski, MD
- RECRUITINGNCT04010604A Registered Cohort Study on SMAWan-Jin Chen
- ENROLLING BY INVITATIONN/ANCT03655223Early Check: Expanded Screening in NewbornsRTI International · PI: Don Bailey, PhD
- RECRUITINGN/ANCT04174157Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)Novartis Pharmaceuticals · PI: Novartis Pharmaceuticals
- ACTIVE NOT RECRUITINGN/ANCT03709784Spinraza in Adult Spinal Muscular AtrophyWashington University School of Medicine · PI: Craig Zaidman, MD
- ACTIVE NOT RECRUITINGN/ANCT04888702Holter of Movement in Patients with SMA Undergoing Treatment.Centre Hospitalier Universitaire de Liege · PI: Margaux Poleur
- ACTIVE NOT RECRUITINGNCT03421977Long-Term Follow-up Study for Patients From AVXS-101-CL-101Novartis Gene Therapies
- RECRUITINGN/ANCT03217578Neonatal Spinal Muscular Atrophy (SMA) ScreeningKaohsiung Medical University Chung-Ho Memorial Hospital · PI: Yun-Hui Chou
- RECRUITINGN/ANCT04292574UK SMA Patient RegistryNewcastle University · PI: Chiara Marini-Bettolo, MD, PhD