ALS / Neuromuscular Disorders Clinical Trials

California Pacific CURRENTS: The online journal of CPMC Research Institute

Below is a listing of ALS/neuromuscular disorders clinical trials approved by CPMC's Institutional Review Board:

Amyotrophic Lateral Sclerosis (ALS)

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

The purpose of this study is to identify the links that exist between the disease phenotype (phenotype refers to observable signs and symptoms) and the disease genotype (genotype refers to your genetic information). We also want to identify biomarkers of ALS and related diseases.

Investigators

Jonathan Katz, MD

Eligibility Requirements

Subjects with amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig's disease and other related neurodegenerative diseases, including frontotemporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA) and multisystem proteinopathy (MSP).

Status

Active and open to enrollment

Contacts

Dallas Forshew, RN, 415-309-5178

Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis

Description: The purpose of this study is to look at new methods of measuring ALS symptoms over time to see if these new approaches are better at detecting changes than the currently used methods

Investigators

Jonathan Katz, MD

Eligibility Requirements

Subjects with amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig's disease who have had weakness for less than 2 years and whose breathing vital capacity is greater than 65%

Status

Active and open to enrollment

Contacts

Dallas Forshew, RN, 415-309-5178

A Phase 2A Safety and Biomarker Study of EPI-589 in Subjects With Amyotrophic Lateral Sclerosis

Description: The purpose of this Phase II research study is to determine safety of EPI-589 and its effects on glutathione in ALS

Investigators

Jonathan Katz, MD

Eligibility Requirements

Patients with amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig's disease who have had symptoms for less than 3 years and FVC above 70%

Status

Active and open to enrollment

Contacts

Dallas Forshew, RN, 415-309-5178

 

A Phase 3, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group, Study to Evaluate the Safety, Tolerability and Efficacy of Tirasemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)

Description: The purpose of this Phase III research study is to see if the drug Tirasemtiv will improve breathing capacity and muscle strength in People with ALS

Investigators

Jonathan Katz, MD

Eligibility Requirements

Patients with amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig's disease who have been diagnosed for no more than 2 years

Status

Closed to enrollment

Contacts

Marguerite Engel, 415-600-3758

 

(MDA Registry 1212M25862): Muscular Dystrophy Association U.S. Neuromuscular Disease Registry

Description: The purpose of this study is to gain insight into disease trends of progression and treatment so as to improve treatment and patient survival. As these neuromuscular diseases become better understood, the quality of care should improve

Investigators

Jonathan Katz, MD

Eligibility Requirements

Subjects with amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig's disease

Status

Active and open to enrollment

Contacts

Dallas Forshew, RN, 415-309-5178

 

HDE Post-Approval Study (PAS) of NeuRx DPS TM (Diaphragm Pacing System) for ALS

Description: The purpose of this research study is to collect more information about the possible risks and benefits of the NeuRx DPS TM device (Diaphragm Pacing System)

Investigators

Jonathan Katz, MD

Eligibility Requirements

Subjects with amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig's disease who have moderate diaphragm weakness

Status

Active but closed to enrollment

Contacts

Dallas Forshew, RN, 415-309-5178

 

Neuromuscular Studies

(IGOS): A Prospective INC Study on Clinical and Biological Predictors of Disease Course and Outcome in GBS

Description: The purpose of this study is to help researchers learn more about the clinical and biological factors that influence the course of GBS, the outcome of patients with GBS, and to find factors that could help diagnose GBS sooner

Investigators

Jonathan Katz, MD

Eligibility Requirements

Patients with Guillain-Barré Syndrome (GBS) who have not yet received treatment for GBS

Status

Active and open to enrollment

Contacts

Dallas Forshew, RN, 415-309-5178

 

A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)

Description: The purpose of this study is to determine if eculizumab is safe and effective for the treatment of patients with refractory gMG

Investigators

Jonathan Katz, MD

Eligibility Requirements

Subjects with generalized myasthenia gravis (gMG) who have needed at least two immunosuppressive drugs, or one immunosuppressive drug and regular plasma exchange or IVIG to control symptoms

Status

Active and closed to enrollment

Contacts

Marguerite Engel, 415-600-3758

 

Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations (PAIN-CONTRoLS)

Description: The purpose of this study is to test four different drugs to see which might work best for the symptoms of cryptogenic sensory polyneuropathy

Investigators

Jonathan Katz, MD

Eligibility Requirements

Patients with painful neuropathy for which the cause is unknown. This is called cryptogenic sensory polyneuropathy (CSPN)

Status

Active and open to enrollment

Contacts

Marguerite Engel, 415-600-3758