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Spinal Muscular Atrophy Clinical Trials

A listing of Spinal Muscular Atrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (64) clinical trials

Muscular Biomarkers in Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS), the most common MND, is a fatal adult-onset neuromuscular disease. Due to clinical heterogeneity and absence of biological tools to diagnose ALS, the delay between the first symptoms and diagnosis averages 9-13 months. A group of pathophysiological processes, including oxidative stress and glutamate-mediated excitotoxicity contribute to ...

Phase N/A

Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND

Multicenter, open-label, phase 1 study of Cu(II)ATSM administered orally to patients wit amyotrophic lateral sclerosis/motor neuron disease. The study will be conducted in three phases. In the first two phases, dose cohorts of six patients each will participate in a single dose pharmacokinetic study followed by a 28-day repeated daily ...

Phase

Open Label Extension Study of AMX0035 in Patients With ALS

The Centaur Open Label Extension Study (CENTAUR-OLE) is designed to provide longer term access to AMX0035 for patients with ALS who participated in the CENTAUR study. The study will assess longer term safety and therapeutic potential of AMX0035.

Phase

Study of Intrathecal Administration of AVXS-101 for Spinal Muscular Atrophy

This is a Phase I, single-dose administration study of infants and children with a genetic diagnosis consistent with SMA, bi-allelic deletion of SMN1 and 3 copies of SMN2 without the genetic modifier who are able to sit but cannot stand or walk at the time of study entry. Patients will ...

Phase

Non-invasive Brain Stimulation for the Treatment of Depression Symptoms in ALS: A Pilot Study

The objective of this study is to evaluate the role of repetitive transcranial magnetic stimulation (rTMS) for symptom reduction of depression and cognitive loss among patients with Amyotrophic Lateral Sclerosis (ALS) and other Neurodegenerative Diseases. Behavioral symptoms in ALS have been under scrutiny since its earliest descriptions, nearly 30 years ...

Phase N/A

SSRIs vs. TCAs for Depression in ALS Patients

This study is a 12-week, open-label, non-randomized, pilot clinical intervention trial. This is investigator initiated study.This trial will be done at St Louis University ALS clinic. ALS patients will be screened for depression using Beck depression inventory (BDI-II) scale. A mental healthcare provider will evaluate the patients scoring 19 or ...

Phase N/A

The Pre-SPG4 Study

Study goals Prospective longitudinal data on progression in the natural course of SPG4 in presymptomatic mutation carriers prior to clinical disease onset and in early stages of disease Biomarkers providing objective measures of disease activity

Phase N/A

Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II

This is a prospective, interventional, sequential, open-label research involving ASI II children with acute respiratory decompensation requiring ventilatory support by NIV. Airway pressure, transcutaneous CO2 monitoring and hemodynamic parameters will be recorded continuously during the study, after inclusion of the patient.

Phase N/A

The Use of Airway Clearance Devices in ALS

This 180 day (25.7 weeks) pilot study is designed to evaluate the effectiveness of airway clearance devices in adults with ALS. Subjects will be randomized in a 1:1 ratio to one of two treatment groups: treatment with a mechanical HCFF device alone or treatment with both a mechanical HFCC device ...

Phase N/A

BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

The goal of the BrainGate2 research and development project is to identify the core methods and features for a medical device that could allow people with paralysis to recover a host of abilities that normally rely on the hands.

Phase N/A