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    ALS / Neuromuscular Disorders
    Clinical Trials

    The Forbes Norris MDA/ALS Research Center

    Adult Clinical Trials Main Page

    Further protocol information can be found on the links below at ClinicalTrials.gov


    Amyotrophic Lateral Sclerosis (ALS)  |  Neuromuscular Studies


    Active Trials

    Amyotrophic Lateral Sclerosis (ALS)

    Title: Phenotype, Genotype & Biomarkers in ALS and Related Disorders
    Description: 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.
    Investigator: Jonathan Katz, MD
    Eligibility: 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
    Contact: Dallas Forshew, RN, 415-309-5178, Email: ForsheD@cpmcri.org about Study Phenotype, Genotype and Biomarker

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    Neuromuscular Studies

    Title: 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
    Investigator: Jonathan Katz, MD
    Eligibility: 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

    Contact: Marguerite Engel, 415-600-3758, Email: EngelM@cpmcri.org about Study Refractory MG

    Title: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate Efficacy and Safety of Repeated Administrations of NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) in Patients with Amyotrophic Lateral Sclerosis (ALS)
    Description: The purpose of this study is to determine efficacy and safety of repeat administrations of intrathecal injections (into spinal fluid) of NurOwn® (MSC-NTF cells), autologous Mesenchymal Stem Cells [MSC] secreting neurotrophic factors as compared to placebo given three times, each two-months apart
    Investigator: Robert Miller, MD
    Eligibility: Subjects with amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig's disease who are aged 60 years or less, have good breathing (FVC greater than 65%) and who have had weakness for less than 24 months
    Status: Active and open to enrollment
    ClinicalTrials.gov ID number: NCT 03280056
    Contact: Dallas Forshew, RN, ForsheD@cpmcri.org or 415-309-5178

    Title: A Phase 2, Multi-Center, Double-Blind, Randomized, Dose-Ranging, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of CK-2127107 in Patients with Amyotrophic Lateral Sclerosis
    Description: The purpose of this study is to evaluate the safety and effect of CK-2127107 on respiratory function (SVC) and muscle function in ALS
    Investigator: Jonathan Katz, MD
    Eligibility: Subjects with amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig's disease who are aged 80 years or less, have good breathing (FVC greater than 65%) and who have been diagnosed for less than 24 months
    Status: Active and open to enrollment
    ClinicalTrials.gov ID number: NCT 03160898
    Contact: Marguerite Engel, EngelM@cpmcri.org or 415-600-3758

    Title: A Randomized, Double-Blind, Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of FLX-787-ODT for Treatment of Muscle Cramps in Adult Subjects with Motor Neuron Disease
    Description: The purpose of this study is to evaluate the safety, tolerability and effectiveness of FLX-787-ODTto treat muscle cramps in motor neuron disease
    Investigator: Liberty Jenkins, MD
    Eligibility: Subjects with motor neuron disease including amyotrophic lateral sclerosis (ALS), primary lateral sclerosis(PLS), or progressive muscular atrophy (PMA), who have at least 3 "Charlie horse" type muscle cramps per week.
    Status: Active and open to enrollment
    ClinicalTrials.gov ID number: NCT 03196375
    Contact: Simon Carty, CartyS@cpmcri.org or 415-600-1368

    Title: A Multicenter, Double Blind, Placebo Controlled Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis
    Description: The purpose of this study is to evaluate the safety, tolerability and effectiveness of Acthar to slow new muscle weakness in ALS
    Investigator: Jonathan Katz, MD
    Eligibility: Subjects with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, who are aged 75 years or less, have good breathing (VFC greater than 60%) and who have had weakness for less than 24 months
    Status: Active and open to enrollment
    ClinicalTrials.gov ID number: NCT 03068754
    Contact: Simon Carty, CartyS@cpmcri.org or 415-600-1368

    Title: Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for Treatment of ALS
    Description: The purpose of this study is to evaluate the safety and efficacy of AMX0035 in subjects with ALS
    Investigator: Jonathan Katz, MD
    Eligibility: Subjects with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, who are aged 80 years or less, have good breathing (FVC greater than 60%) and who have had weakness for less than 18 months
    Status: Active and open to enrollment
    ClinicalTrials.gov ID number: NCT 03127514
    Contact: Marguerite Engel, EngelM@cpmcri.org or 415-600-3758

    Last Updated 4/2018
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