Spinal Muscular Atrophies of Childhood
Spinal Muscular Atrophies of Childhood
Explore linked clinical trials. Standard reference: MESH:M002291919 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type III | Recruiting | Phase 2 | 2026-10-02 | NCT07047144 | |
| Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Juvenile Spinal Muscular Atrophy, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Phase 3 | 2026-09-24 | NCT04042025 | |
| Long-Term Follow-up Study for Patients From AVXS-101-CL-101 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Not stated | 2026-09-22 | NCT03421977 | |
| GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Enrolling by invitation | Not applicable | 2026-09-11 | NCT06772402 | |
| Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab | Atrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, muscular atrophy, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type III | Active, not recruiting | Phase 3 | 2026-09-01 | NCT05626855 | |
| A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Recruiting | Phase 1 / Phase 2 | 2026-08-26 | NCT07554924 | |
| UK SMA Patient Registry | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, Spinal Muscular Atrophies of Childhood, X-linked disease, autosomal recessive distal spinal muscular atrophy 1, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy | Recruiting | Not stated | 2026-08-20 | NCT04292574 | |
| Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Recruiting | Phase 1 / Phase 2 | 2026-06-29 | NCT07070999 | |
| Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deglutition Disorders, Digestive System Diseases, Pharyngeal Diseases, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, digestive system disorder, esophageal disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, otorhinolaryngologic disease, spinal cord disorder, spinal muscular atrophy | Not yet recruiting | Not stated | 2026-06-02 | NCT07596277 | |
| Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fatigue, Juvenile Spinal Muscular Atrophy, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Recruiting | Not applicable | 2026-05-06 | NCT06562283 | |
| Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, Spinal Muscular Atrophies of Childhood, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type III | Recruiting | Not stated | 2026-04-09 | NCT06839469 | |
| Gait and Bone Health in SMA | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, bone fracture, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, injury, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type III | Recruiting | Not stated | 2026-03-18 | NCT07400198 | |
| A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Not applicable | 2025-11-21 | NCT06191354 | |
| Test of Aquatic Mobility of SMA Infants | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscle Hypotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Recruiting | Not applicable | 2025-11-21 | NCT06152302 | |
| Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Not yet recruiting | Not stated | 2025-10-06 | NCT07208903 | |
| Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, Spinal muscular atrophy 4, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type III | Active, not recruiting | Not applicable | 2025-08-26 | NCT06300996 | |
| Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type III | Recruiting | Phase 1 / Phase 2 | 2025-07-03 | NCT06421831 | |
| Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Phase 1 / Phase 2 | 2025-07-03 | NCT05901987 | |
| Spinraza in Adult Spinal Muscular Atrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type III | Active, not recruiting | Not stated | 2024-11-20 | NCT03709784 |