Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation

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

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.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: Placebo; Drug: NP001

Detailed Description

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 Type: Interventional
• Enrollment (Actual): 138
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Montreal, Quebec, Canada, H3A 2B4
      • Montreal Neurological Institute
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
    • Scottsdale, Arizona, United States, 85259
      • Mayo Clinic Arizona
  • California
    • Los Angeles, California, United States, 90048
      • Cedars-Sinai Medical Center
    • Orange, California, United States, 92862
      • University of California, Irvine, Department of Neurology
    • San Francisco, California, United States, 94115
      • Forbes Norris MDA/ALS Research Center, CPMC
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic Florida
    • Miami, Florida, United States, 33136
      • University of Miami Miller School of Medicine
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University, Department of Neurology
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
  • Kentucky
    • Lexington, Kentucky, United States, 40536-0293
      • University of Kentucky, Albert B. Chandler Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Minnesota
    • Minneapolis, Minnesota, United States, 55414
      • Clinical & Translational Science Institute, University of Minnesota
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • New York
    • New York, New York, United States, 10032
      • Columbia University Medical Center
  • North Carolina
    • Charlotte, North Carolina, United States, 28207
      • Carolinas Medical Center, Neurosciences Instutite-Neurology
    • Durham, North Carolina, United States, 27705
      • Duke Neurological Disorders Clinic at Morreene Road
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic Foundation-Cleveland Clinic Hospital
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexner Medical Center
  • Oregon
    • Portland, Oregon, United States, 97213
      • Providence Brain & Spine Institute, ALS Center
  • Texas
    • Houston, Texas, United States, 77030
      • Houston Methodist Neurological Institute
    • San Antonio, Texas, United States, 78229
      • University of Texas Health Sciences Center San Antonio

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NP001; NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6	Drug: NP001
Placebo Comparator: Placebo; Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6	Drug: Placebo

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Neuraltus Pharmaceuticals, Inc.
• Investigators: 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

Study record dates

Study Major Dates

• Study Start (Actual): August 29, 2016
• Primary Completion (Actual): December 12, 2017
• Study Completion (Actual): December 12, 2017

Study Registration Dates

• First Submitted: May 24, 2016
• First Submitted That Met QC Criteria: June 6, 2016
• First Posted (Estimate): June 9, 2016

Study Record Updates

• Last Update Posted (Actual): May 7, 2018
• Last Update Submitted That Met QC Criteria: May 3, 2018
• Last Verified: May 1, 2018

More Information

Terms related to this study

• Keywords: Inflammation; Amyotrophic Lateral Sclerosis; ALS; Motor Neuron Disease; C-Reactive Protein; Nervous System Diseases; Central Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Diseases
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Inflammation; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: NP001-10-003