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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02794857
First Posted: June 9, 2016
Last Update Posted: July 13, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Neuraltus Pharmaceuticals, Inc.
  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.

Condition Intervention Phase
Amyotrophic Lateral Sclerosis Drug: NP001 Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Neuraltus Pharmaceuticals, Inc.:

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 ]

Enrollment: 138
Study Start Date: August 2016
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   21 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02794857


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

Responsible Party: Neuraltus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02794857     History of Changes
Other Study ID Numbers: NP001-10-003
First Submitted: May 24, 2016
First Posted: June 9, 2016
Last Update Posted: July 13, 2017
Last Verified: July 2017

Keywords provided by Neuraltus Pharmaceuticals, Inc.:
ALS
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Inflammation
C-Reactive Protein

Additional relevant MeSH terms:
Sclerosis
Inflammation
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases