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

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.

• 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

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

  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

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

  Columbia University Medical Center
  New York, New York 10032
  NY