A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS) and Evidence of Elevated Systemic Inflammation

Study Purpose:

This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Study Director: Gil Block, MD, PhD, Neuraltus Pharmaceuticals, Inc.
Principal Investigator: Robert G. Miller, MD, California Pacific Medical Center
Principal Investigator: Jonathan Katz, MD, California Pacific Medical Center

Clinicaltrials.gov ID (11 digit #):

NCT02794857

Neals Affiliated?

Yes

Coordinating Center Contact Information

Neuraltus
Frances Faurot / .(JavaScript must be enabled to view this email address) / 415-912-1981
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS and evidence of elevated systemic inflammation. Subjects will be allocated (1:1) to NP001 and placebo. Drug or placebo will be given intravenously.

Study Sponsor:

Neuraltus Pharmaceuticals, Inc.

Participant Duration:

Estimated Enrollment:

138

Estimated Study Start Date:

08/01/2016

Estimated Study Completion Date:

09/30/2017

Posting Last Modified Date:

08/25/2017

Date Study Added to alsconsortium.org:

08/16/2016
  • More Information

    Primary Outcome Measures:
    Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire [ Time Frame: Baseline and 6 months ]

    Secondary Outcome Measures:
    Change in pulmonary function as measured by slow vital capacity readings [ Time Frame: Baseline and 6 months ]
    Time to tracheotomy [ Time Frame: Up to 6 months ]
    Change in levels of blood inflammatory biomarkers [ Time Frame: Baseline, 3 and 6 months ]

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    21

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Key Inclusion Criteria:
    -Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)
    -Forced vital capacity greater than or equal to 65% of that predicted for age and height
    -Onset of ALS-related weakness less than 3 years prior to first dose of study drug
    -Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening
    -Stable dose (greater than 30 days) of riluzole if undergoing treatment with this agent
    -For females: Not be of childbearing potential or agree to use adequate birth control during the study

    Key Exclusion Criteria:
    -Life expectancy of less than 6 months
    -Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)
    -Active pulmonary disease
    -Gastrostomy
    -Stem cell therapy
    -Immune modulator therapy or participation in studies of other agents within 12 weeks of pre-screening/screening
    -Unstable medical condition other than ALS

  • Site Contact Information

    St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
    Ashley Reece / .(JavaScript must be enabled to view this email address) / 602-406-4775
    Phoenix, Arizona 85013
    United States

    Mayo Clinic Arizona
    Alisa Powell / .(JavaScript must be enabled to view this email address) / 480-301-8788
    Scottsdale, Arizona 85259
    United States

    University of California, Irvine, Department of Neurology
    Veena Mathew / .(JavaScript must be enabled to view this email address) / 714-456-2864
    Orange, California , 92862
    United States

    Forbes Norris MDA/ALS Research Center, CPMC
    Dallas A. Forshew, RN, BSN / .(JavaScript must be enabled to view this email address) / 415-309-5178

    San Francisco, California 94115
    United States

    Cedars-Sinai Medical Center
    Vy Nguyen / .(JavaScript must be enabled to view this email address) / 424-315-2361
    Los Angeles, California 90048
    United States

    Mayo Clinic Florida
    Carla Palmucci, B.A. / .(JavaScript must be enabled to view this email address) / 904-953-3182
    Jacksonville, Florida 32224
    United States

    University of Miami Miller School of Medicine
    Julie Steele, RN / .(JavaScript must be enabled to view this email address) / 305-243-7336
    Miami, Florida 33136
    United States

    Emory University
    Meraida Polak, RN / .(JavaScript must be enabled to view this email address) / 404-778-3807
    Atlanta, Georgia 30322
    United States

    University of Kansas Medical Center
    Christy Nichols / .(JavaScript must be enabled to view this email address) / 913-945-9945
    Kansas City, Kansas 66160
    United States

    University of Kentucky, Albert B. Chandler Medical Center
    Meghann Bruno, RN, BSN / .(JavaScript must be enabled to view this email address) / 859-218-5064
    Lexington, Kentucky 40536-0293
    United States

    Massachusetts General Hospital
    Leah Miller / .(JavaScript must be enabled to view this email address) / 617-724-7398
    Boston, Massachusetts 02114
    United States

    Clinical & Translational Science Institute, University of Minnesota
    Susan Rolandelli, RN / .(JavaScript must be enabled to view this email address) / 612-624-7745
    Minneapolis, Minnesota 55414
    United States

    Washington University School of Medicine
    Saint Louis, Missouri 63110
    United States

    Carolinas Medical Center, Neurosciences Instutite-Neurology
    Cynthia Lary, RN / .(JavaScript must be enabled to view this email address) / 704-446-6063
    Charlotte, North Carolina 28207
    United States

    Duke Neurological Disorders Clinic at Morreene Road
    Karen Grace, RN / .(JavaScript must be enabled to view this email address) / 919-668-2844
    Durham, North Carolina 27705
    United States

    Cleveland Clinic Foundation-Cleveland Clinic Hospital
    Irys B. Caristo / .(JavaScript must be enabled to view this email address) / 216-444-0173
    Cleveland, Ohio 44195
    United States

    The Ohio State University Wexner Medical Center
    Columbus, Ohio 43210
    United States

    Providence Brain & Spine Institute
    Arlena Georgeson / .(JavaScript must be enabled to view this email address) / 503-962-1171
    ALS Center
    Portland, Oregon 97213
    United States

    Houston Methodist Neurological Institute
    Luis F. Lay, Jr., CCRC / .(JavaScript must be enabled to view this email address) / 713-441-3057
    Houston, Texas 77030
    United States

    University of Texas Health Sciences Center San Antonio
    San Antonio, Texas 78229
    United States

    Columbia University Medical Center
    New York, New York 10032
    NY

    Montreal Neurological Institute
    Kristiana Salmon / .(JavaScript must be enabled to view this email address) / 514-398-1779
    Montreal, Quebec
    Canada