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Spinal Muscular Atrophy (SMA) clinical trials

View clinical trials related to Spinal Muscular Atrophy (SMA).

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NCT ID: NCT05747261 Recruiting - Clinical trials for Spinal Muscular Atrophy (SMA)

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)

BLUEBELL
Start date: February 2, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy. The study will have a standard 3+3 dose-escalation design.

NCT ID: NCT05354414 Recruiting - Clinical trials for Spinal Muscular Atrophy (SMA)

Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy

REALITY
Start date: May 9, 2022
Phase: N/A
Study type: Interventional

The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.

NCT ID: NCT05335876 Recruiting - Clinical trials for Spinal Muscular Atrophy (SMA)

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

SPECTRUM
Start date: December 19, 2022
Phase: Phase 3
Study type: Interventional

This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 15 years from the date of OAV101 administration in the previous clinical trial.

NCT ID: NCT05115110 Recruiting - Clinical trials for Spinal Muscular Atrophy (SMA)

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

MANATEE
Start date: June 2, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an investigational anti-myostatin antibody that is designed to target myostatin. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. RO7204239 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and clinical outcomes for people living with SMA. This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in patients with spinal muscular atrophy (SMA). The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either ambulant (aged 2-10 years) or non-ambulant (aged 5-10 years) within separate cohorts, and Part 2 is the pivotal part in SMA patients aged 2-25 years that are ambulant.

NCT ID: NCT04174157 Recruiting - Clinical trials for Spinal Muscular Atrophy (SMA)

Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Start date: September 25, 2018
Phase:
Study type: Observational [Patient Registry]

Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases. Until recently, the mainstay of treatment for these patients was supportive medical care. However, advances in medical treatment focusing on gene replacement, gene enhancement, motor neuron protection and muscle enhancement is likely to change the management and prognosis of these patients in the future. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options.