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28 recruiting studies matching your filters

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with…

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

Hoffmann-La RocheNCT05861999

A Registry of Cases of Spinal Muscular Atrophy Diagnosed in the Prenatal Period That Received Disease-Modifying Therapy

Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the sa…

Spinal Muscular Atrophy

Estimated

~$10 – $750

St. Jude Children's Research HospitalNCT07805278

Phase 1

An Open Label Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)

In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusiners…

Muscular Atrophy, Spinal

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pe…

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

Hoffmann-La RocheNCT05861986

Observational

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

The goal of this clinical trial is to evaluate the safety and preliminary efficacy of an implantable device that records and stimulates different areas of the …

Tetraplegia/TetraparesisAmyotrophic Lateral Sclerosis (ALS)Muscular Disorders, Atrophic+6 more

Estimated

~$10 – $6,250

Johns Hopkins UniversityNCT07521930

Biofluid Research on Age-Related or Inherited Neurodegeneration (BRAIN)

The aim of this study is to contribute to and continue to sustain a repository of blood samples, nucleic acid (DNA and/or RNA) samples, family history informat…

Frontotemporal Degeneration (FTD)ALS (Amyotrophic Lateral Sclerosis)Primary Progressive Aphasia (PPA)+12 more

Estimated

~$10 – $750

University of PennsylvaniaNCT07798700

Phase 2

A Phase 2 Double-Blinded, Randomized, Placebo-Controlled 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

This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it m…

Spinal Muscular Atrophy (SMA)

Safety and Performance of the ThecaFlex DRx™ System Port and Catheter for Chronic Intrathecal Access, Cerebrospinal Fluid (CSF) Aspiration, and DElivery of Nusinersen in Spinal Muscular Atrophy (SMA) Patients Resistant to Lumbar PunctuRE Trial (PIERRE)

The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects…

Spinal Muscular AtrophySpine DeformityScoliosis

Estimated

~$10 – $8,750

Alcyone Therapeutics, IncNCT05866419

Phase 3

An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy

In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about …

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

BiogenNCT07221669

An Observational Study to Assess Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

Background: SBMA is an inherited chronic disease. It affects males in mid to late adulthood. It causes slowly progressive weakness of muscles and hand tremors…

Spinal and Bulbar Muscular AtrophyKennedys DiseaseMotor Neuron Disease

Estimated

~$10 – $675

National Institute of Neurological Disorders and Stroke (NINDS)NCT04944940

Phase 3

A Phase 3, Randomized, Double-Blinded Study to Evaluate the Safety and Efficacy of Salanersen (BIIB115) After Onasemnogene Abeparvovec Treatment in Infants With Genetically Diagnosed Spinal Muscular Atrophy

In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about h…

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

BiogenNCT07444450

Pilot Study Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

In this observational study, researchers are looking at the effects of spinal muscular atrophy (SMA) drugs on the muscles and nerve cells in patients with SMA.…

Spinal Muscular Atrophy

Estimated

~$10 – $750

St. Jude Children's Research HospitalNCT06532474