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.

Ongoing Online Survey Studies

Genetic (SOD-1 and C9orf) Testing/Repository

Intimacy and ALS (Online Survey)
Assesses patients’ and significant others’ perceptions of the effects of diagnosis and symptoms on intimacy and their comfort level in intimacy-related discussions with clinic team members .

Misconceptions in Palliative Care in ALS (Online Survey)
Survey measuring common patterns in patients’ and caregivers’ misconceptions of palliative care.

Enrolling Now

Will be evaluating the efficacy of the drug Tirasemtiv versus placebo in improving muscle strength in patients with ALS.

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.

Will be assessing the effects of Ezogabine (a potassium channel opener) on neuronal excitability in patients with ALS.

Will be evaluating the efficacy and safety of oral fingolimod versus placebo in patients with CIPD.

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.

NeuroRx Diaphragmatic Stimulator in ALS
Designed to see whether electrical stimulation of the diaphragm is of benefit to people with ALS.

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.

Subcutaneous IVIG in CIDP
Investigates the efficacy of two different IVIG (intravenous immunoglobulin) doses in the maintenance treatment of CIDP.

Designed to find out if Rituximab is effective and safe in patients with myasthenia gravis (MG) who are also taking prednisone.

Investigates safety and efficacy of Ecluzimab in subjects with Refractory Myasthenia Gravis (gMG).

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

Open Chart Review Studies

Edema in ALS
Chart review of prevalence of swelling and management strategies in patients with ALS.

Pain in ALS
Chart review of frequency and etiology of pain in ALS.

Awaiting Results

Enrollment is closed but data is being analyzed.

The Safety and Efficacy of SOD1 Inhibition by Pyrimethamine in Sporadic and Familial 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.

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.


Research Team

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

Dr. Dale Lange

Lindsay Kaplan

Shara Holzberg