Spinal Muscular Atrophy Clinical Trials

221 clinical trials for Spinal Muscular Atrophy across international registries, including 43 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• 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 — RECRUITING · PHASE2
• Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies — RECRUITING
• VRehab-SMA Phase 1.2 — RECRUITING · NA
• An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered via the ThecaFlex DRx™ System (PIERRE-PK) — RECRUITING · PHASE1
• Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers — RECRUITING · NA
• Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA — RECRUITING
• 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) — RECRUITING
• A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy — RECRUITING · PHASE2
• 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 — RECRUITING · PHASE3
• Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy — RECRUITING · NA
• HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness — RECRUITING · NA
• Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease — RECRUITING · NA
• Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy — RECRUITING · NA
• Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases — RECRUITING
• Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System — RECRUITING
• Long Read Analysis in Spinal Muscular Atrophy - LOREASI — RECRUITING · NA
• Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening — RECRUITING · NA
• Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC). — RECRUITING · PHASE3
• The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder — RECRUITING · NA
• CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain — RECRUITING
• Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders — RECRUITING
• The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. — RECRUITING · NA
• A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular Atrophy — RECRUITING · NA
• Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients — RECRUITING · PHASE1
• Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy — RECRUITING · NA
• Adult SMA Research and Clinical Hub — RECRUITING
• French Register of Patients With Spinal Muscular Atrophy — RECRUITING
• Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy — RECRUITING · PHASE1
• A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA — RECRUITING
• Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) — RECRUITING
• Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases — RECRUITING
• 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) — RECRUITING · PHASE1
• Neonatal Spinal Muscular Atrophy (SMA) Screening — RECRUITING
• Multispectral optoacoustic tomography (MSOT) for translational molecular imaging in neuromuscular disease — RECRUITING
• A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN PATIENTS WITH SPINAL MUSCULAR ATROPHY — RECRUITING
• Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments — RECRUITING
• Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis — RECRUITING
• A LONG-TERM EXTENSION STUDY OF NUSINERSEN (BIIB058) ADMINISTERED AT HIGHER DOSES IN PARTICIPANTS WITH SPINAL MUSCULAR ATROPHY WHO PREVIOUSLY PARTICIPATED IN AN INVESTIGATIONAL STUDY WITH NUSINERSEN — RECRUITING
• A PHASE IIIB, OPEN-LABEL, SINGLE-ARM, SINGLE-DOSE, MULTICENTER STUDY TO EVALUATE THE SAFETY, TOLERABILITY AND EFFICACY OF GENE REPLACEMENT THERAPY WITH INTRAVENOUS OAV101 (AVXS-101) IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (SMA) — RECRUITING · PHASE3
• A Registered Cohort Study on SMA — RECRUITING
• A PHASE 2, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, MULTIPLE DOSE STUDY OF CK 2127107 IN TWO ASCENDING DOSE COHORTS OF PATIENTS WITH SPINAL MUSCULAR ATROPHY (SMA) — RECRUITING · PHASE2
• A, PILOT, OPEN-LABEL, DOSE-RESPONSE STUDY INVESTIGATING THE EFFECT OF LOW-DOSE CELECOXIB ON SMN2 IN PATIENTS WITH SPINAL MUSCULAR ATROPHY — RECRUITING
• A PHASE 3, RANDOMIZED, DOUBLE-BLIND, SHAM-PROCEDURE CONTROLLED STUDY TO ASSESS THE CLINICAL EFFICACY AND SAFETY OF ISIS 396443 ADMINISTERED INTRATHECALLY IN PATIENTS WITH INFANTILE-ONSET SPINAL MUSCULAR ATROPHY — RECRUITING · PHASE3
• An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of Salanersen (BIIB115) in Participants Aged 15-60 Years With Spinal Muscular Atrophy Who Are Either Treatment-Naïve or Have Previously Been Treated With Risdiplam — NOT_YET_RECRUITING · PHASE3
• A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia — NOT_YET_RECRUITING
• Equine-Assisted Physiotherapy for Spinal Muscular Atrophy — COMPLETED · NA
• Spinal Muscular Atrophy and School Transitions — NOT_YET_RECRUITING
• Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — ACTIVE_NOT_RECRUITING · PHASE4
• A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy — TERMINATED · PHASE3
• Effects of Developmental Support for Mothers of Infants With Spinal Muscular Atrophy — COMPLETED · NA

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