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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 (10) clinical trials

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

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

CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS

Primary objective: to assess the safety and tolerability of multiple doses of orally administered CC100 in subjects with amyotrophic lateral sclerosis (ALS). Secondary objectives: to determine pharmacokinetics and pharmacodynamics of CC100 in plasma after single and after multiple doses; and to determine short-term effects of CC100 on potential blood-cell ALS ...

Phase

Safety/Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in ALS

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease characterized by motor neuron loss. Despite of many trials for disease-modifying, no treatment has so far changed natural course of disease. The investigators had performed the pre-clinical and clinical studies using autologous bone marrow-derived stem cells in ALS. In the investigators' results ...

Phase

Intraspinal Transplantation of Autologous ADRC in ALS Patients

Amyotrophic Lateral Sclerosis (ALS) is an incurable disease of unknown etiology that in a short time leads to significant impairment of motor functions and death. The frequency of ALS is 4-8/100 000. Mostly it affects people between 40 and 70 years old, but it can occur at a younger age. ...

Phase

Molecular Imaging Modality by Positron Emission Tomography Using 18F-X : Study of Microglial Activation in Amyotrophic Lateral Sclerosis

18F-X PET will be carried out requiring arterial sampling in 2 patients suffering from ALS and 2 normal subjects in order to evaluate the 18F-X quantification. Then simplified PET using 18F-X will be carried out in 13 patients and 13 normal subjects. Binding potential maps showing specific binding of 18f-X ...

Phase

A Study of GDC-0134 to Determine Initial Safety Tolerability and Pharmacokinetic Parameters in Participants With Amyotrophic Lateral Sclerosis

This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134. It will include three components: a Single-Ascending-Dose (SAD) stage, a Multiple-Ascending-Dose (MAD) stage, and an Open-Label Safety Expansion (OSE) stage.

Phase

A Study to Assess the Safety Tolerability and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis

The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-ALS. The secondary objective of this study is to evaluate the pharmacokinetic profile of BIIB078.

Phase

ECoG BMI for Motor and Speech Control

ECoG is a type of electrophysiological monitoring that uses electrodes placed directly on the exposed surface of the brain to record electrical activity. With this ECoG-based neural interface, study patients will undergo training and assessment of their ability to control a wearable hand robotic exoskeleton and determine if ECoG brain ...

Phase

Study to Evaluate DNL747 in Subjects With Amyotrophic Lateral Sclerosis

This is a Phase 1b randomized, placebo-controlled, double-blind, crossover study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL747 in subjects with Amyotrophic Lateral Sclerosis (ALS)

Phase