> Skip repeated content


Research Trials

We are committed to the cooperative implementation, timely completion, analysis, and reporting of clinical trials and other research studies aimed at improving patient care and understanding of the disease processes of amyotrophic lateral sclerosis (ALS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), myasthenia gravis (MG) and many other neurological conditions.

Upcoming Online Studies

Phase 2 Clinical Trial of RA101495 in Generalized Myasthenia Gravis

Will be evaluating the effectiveness of RA101495 as a treatment to possibly prevent the body’s attack on its neuromuscular junctions in patients with generalized myasthenia gravis (gMG).

Ongoing Online Survey Studies

Genetic (SOD-1 and C9orf) Testing/Repository and Biorepository

FLX-787-ODT for Muscle Cramps in Motor Neuron Disease
Evaluating how FLX-787_ODT works to prevent, relieve, and/or reduce muscle cramps in patients with ALS, PLS, or PMA.

Phase II Clinical Trial of CK-2127107
Evaluating the safety, effectiveness and tolerability of CK-2127107 as a potential new therapy for the improvement of muscle weakness and fatigue.

Non-invasive Brain Stimulation for the Treatment of Depression Symptoms in Neuromuscular Disorders: A Pilot Study
Assessing the role of repetitive transcranial magnetic stimulation (rTMS) as a treatment for symptoms of depression in patients with neuromuscular disorders.

Fluid Biomarkers with Deep Phenotyping in Patients with ALS (ALSA BIO3)
Identifying biomarkers associated with ALS (what changes are unique to ALS) and assessing therapeutic targets.

Enrolling Now

Triple Stimulation Technique (TST) in ALS Quantifying Upper Motor Neuron Function
Investigates the use of the Triple Stimulation Technique (TST) as an effective diagnostic tool for ALS and assesses TST identification of the presence of possible proximal conduction blocks in upper motor neuron function.

Gene Expression Analysis in ALS and Multifocal Motor Neuropathy
Aims to assess if the use of genetic profiling could be used as diagnostic and prognostic biomarkers in ALS and to differentiate ALS from MMN.

Non-Motor Manifestations of ALS
Investigates the occurrence of different symptoms in patients with ALS compared to healthy controls.

MRI quantitative susceptibility mapping (QSM) in ALS
Using MRI sequences to correlate MRI findings and disease severity in ALS.

Designed to see whether the use of FDA approved medication Ampyra (dalfampridine) is helpful in improving walking speed in patients with primary lateral sclerosis.

Awaiting Results

Enrollment is closed but data is being analyzed.

Safety of Caprylic Triglycerides in ALS: A Pilot Study
Will be determining if Axona (a medical food) is safe, tolerated and able to cause ketones to increase in the blood of patients with ALS.

The Role of A-Waves in Separating ALS/MND from Motor Neuropathy
The purpose of this study was to determine if abnormal A-waves will predict IVIG (intravenous immunoglobulin) responsiveness (improvement of strength) in patients with motor neuropathy without conduction block.

The Safety and Efficacy of SOD1 Inhibition by Pyrimethamine in Sporadic ALS
One of the first studies looking at a direct biomarker for ALS and our research team was the primary coordination center for multiple sites around the globe. Investigated the effects of SOD1 inhibition by Pyrimethamine in patients with familial and sporadic ALS.

New – Recently Published

Pyrimethamine Significantly Lowers Cerebrospinal Fluid Cu/Zn Superoxide Dismutase in Amyotrophic Lateral Sclerosis Patients with SOD1 Mutations

Research Team

To learn more about HSS Neurology Research, contact any member of the research team:

Dr. Dale Lange

Shara Holzberg