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:

Active, currently recruiting

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:

120

Estimated Study Start Date:

08/01/2016

Estimated Study Completion Date:

09/30/2017

Posting Last Modified Date:

02/14/2017

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

    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

    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

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

    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

    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

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

    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

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

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

    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

    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

    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

    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

    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

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