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27 studies found for:    Open Studies | "Muscular Atrophy, Spinal"
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1 Not yet recruiting A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (Firefish)
Condition: Muscular Atrophy, Spinal
Intervention: Drug: RO7034067
2 Recruiting A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish)
Condition: Muscular Atrophy, Spinal
Interventions: Drug: Placebo;   Drug: RO7034067
3 Recruiting An Open Label Study of LMI070 in Type 1 Spinal Muscular Atrophy (SMA)
Condition: Spinal Muscular Atrophy
Intervention: Drug: LMI070
4 Recruiting International SMA Patient Registry
Condition: Muscular Atrophy, Spinal
Intervention:
5 Recruiting Prospective Evaluation of Infants With Spinal Muscular Atrophy:
Condition: Spinal Muscular Atrophy
Intervention:
6 Recruiting Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproate, Levocarnitine;   Drug: Placebo
7 Recruiting A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: CK-2127107 150 mg;   Drug: Placebo;   Drug: CK-2127107 450 mg
8 Recruiting Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
Condition: Spinal Muscular Atrophy Type 3
Intervention: Drug: Pyridostigmine Bromide
9 Recruiting A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Nusinersen
10 Not yet recruiting Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy (SMA)
Intervention: Drug: celecoxib
11 Available Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
Condition: Infantile-onset Spinal Muscular Atrophy (SMA)
Intervention: Drug: nusinersen
12 Recruiting Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)
Condition: Infantile Spinal Muscular Atrophy of Type 2 or 3
Intervention: Other: Physical exercise in a swimming pool during 6 months
13 Recruiting A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Olesoxime
14 Recruiting Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene
Condition: Spinal Muscular Atrophy
Intervention: Procedure: blood sampling
15 Not yet recruiting Oxidative Capacity and Exercise Tolerance in Ambulatory SMA
Conditions: Spinal Muscular Atrophy Type 3;   Mitochondrial Myopathy
Intervention:
16 Recruiting SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4
Conditions: Spinal Muscular Atrophy;   SMA;   Kugelberg-Welander Disease
Interventions: Drug: Pyridostigmine;   Drug: Placebo
17 Recruiting Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients
Condition: Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
Intervention: Biological: Adipose derived mesenchymal stem cell
18 Recruiting Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy
Conditions: Spinal Muscular Dystrophy;   Neuromuscular Disability
Intervention: Other: Assisted Standing Treatment Program
19 Recruiting Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
Conditions: Spinal Muscular Atrophy;   Charcot-Marie-Tooth Disease;   Muscular Dystrophy;   Spinal Muscular Atrophy With Respiratory Distress 1;   Amyotrophic Lateral Sclerosis;   Motor Neuron Disease;   Neuromuscular Disease;   Peroneal Muscular Atrophy;   Fragile X Syndrome
Intervention: Other: sample collection
20 Not yet recruiting A Patient Centric Motor Neuron Disease Activities of Daily Living Scale
Conditions: Amyotrophic Lateral Sclerosis;   Progressive Muscular Atrophy;   Primary Lateral Sclerosis;   Hereditary Spastic Paraplegia
Intervention: Other: Web-based Survey

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Study has passed its completion date and status has not been verified in more than two years.