ALS Clinical Trials

What is a Clinical Trial?

A clinical trial is a method for medical researchers to study, test and discover effective treatments. Clinical trial research studies can occur in a lab or involve human beings.

Study Title: NA_00017913 A Multicenter Study for the Validation of ALS Biomarkers

Summary: This research is being done to collect and compare blood and cerebrospinal fluid (CSF) samples from people with and without amyotrophic lateral sclerosis (ALS).  We hope to learn more about the underlying cause of ALS, as well as find unique biological markers (biomarkers), which could be used to develop new therapies.

Human Study

Recruiting Status: Enrollment is closed

Study Title: NA_00033726   Generation and characterization of cell lines for Amyotrophic Lateral Sclerosis

Summary: This research is being done to gain a better understanding of Amyotrophic Lateral Sclerosis (ALS) by looking at skin cells. Skin cells will be obtained from individuals with genetically confirmed familial ALS and some healthy individuals. Once the skins cells have been grown up in the laboratory, they will be used to generate induced pluripotent stem cells, which in turn, will be made into nerve cells. Scientists will then be able to study these cells in a variety of ways to learn more about ALS and potential treatments.

Lab Study

Recruiting Status: Enrollment is closed

Study Title: NA_00021979 Skin Biopsies to generate cells lines for study of Amyotrophic Lateral Sclerosis

Summary: This research is being done to gain a better understanding of Amyotrophic Lateral Sclerosis (ALS) by looking at skin cells. Skin cells will be obtained from individuals with sporadic ALS, similar neuromuscular diseases and some healthy individuals. Once the skins cells have been grown up in the laboratory, they will be used to generate induced pluripotent stem cells, which in turn, will be made into nerve cells. Scientists will then be able to study these cells in a variety of ways to learn more about ALS and potential treatments.

Lab Study

Recruiting Status: Actively enrolling

Study Title: NA_00022650: Trial of resistance and endurance exercise in Amyotrophic Lateral Sclerosis

Summary: A study to see whether one type of exercise is tolerated better and is safer than another type for people with ALS. Study will also collect information on how the body responds to exercise in people with ALS.

Each participant will be randomly assigned to one of three types of exercise – weightlifting (resistance exercise), stationary bicycling (endurance exercise), and range of motion stretching exercise (which is the current standard of care for ALS patients).

Human Study

Recruiting Status: Actively enrolling

Study Title: NA_00044354: Long-Term follow-up of subjects with Amyotrophic Lateral Sclerosis treated with SB-509 Plasmid Gene Therapy (SB-509-1001)

Summary: A study to evaluate any potential long-term consequence in patients who have been treated with SB-509. This is a study to collect information only, and is not a treatment study.

Eligible participants include those with ALS who have been treated with SB-509.

Human Study

Recruiting Status: Actively enrolling

Study Title: NA_00044953: Use of the Penn State Scree of Frontal and Temporal Dysfunction (PSSFTS) in Amyotrophic Lateral Sclerosis: A multi-center study

Summary: A study to clarify the relationship between ALS and the development of cognitive change. Cognition refers to thinking processes involved in language, memory, spatial skills and problem solving. Caregivers of participants will also be participating to some extent.

Eligible participants must have ALS and be between ages of 20 and 85. This study will help improve our current understanding of changes in cognition that occur in the presence of ALS.

Human Study

Recruiting Status: Actively enrolling

Study Title: NA_00047257: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center study of the Safety and Efficacy of Dexpramipexole in subjects with Amyotrophic Lateral Sclerosis

Summary: Dexpramipexole in treatment of patients with ALS. Dexpramipexole is not approved by the FDA, however it is being allowed by the FDA for the purpose of this study.

Mitochondria are the energy source for cells in the body. In ALS, mitochondria undergo oxidative stress which causes cell damage. Dexpramipexole is thought to improve function and efficiency of mitochondria within motor neurons.

Human Study

Recruiting Status: Closed

Study Title: NA_00071274: An Open-Label, Multicenter, Extension study to evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in subjects with Amyotrophic Lateral Sclerosis

Summary: Dexpramipexole in treatment of patients with ALS. Dexpramipexole is not approved by the FDA, however it is being allowed by the FDA for the purpose of this study.

Mitochondria are the energy source for cells in the body. In ALS, mitochondria undergo oxidative stress which causes cell damage. Dexpramipexole is thought to improve function and efficiency of mitochondria within motor neurons.

Human Study

Recruiting Status: Currently enrolling only patients in NA_00047257