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Trial record 3 of 5 for:    NP001

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2017 by Neuraltus Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Neuraltus Pharmaceuticals, Inc. Identifier:
First received: May 24, 2016
Last updated: March 20, 2017
Last verified: March 2017
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: 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 ]

Estimated Enrollment: 120
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.

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

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
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02794857

Contact: Frances Faurot 415-912-1981

United States, Arizona
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics Recruiting
Phoenix, Arizona, United States, 85013
Contact: Ashley Reece    602-406-4775   
Principal Investigator: Shafeeq Ladha, MD         
Mayo Clinic Arizona Recruiting
Scottsdale, Arizona, United States, 85259
Contact: Alisa Powell    480-301-8788   
Principal Investigator: Mark Ross, MD         
United States, California
Cedars-Sinai Medical Center Recruiting
Los Angeles, California, United States, 90048
Contact: Vy Nguyen    424-315-2361   
Principal Investigator: Richard Lewis, MD         
University of California, Irvine, Department of Neurology Recruiting
Orange, California, United States, 92862
Contact: Veena Mathew    714-456-2864   
Principal Investigator: Namita Goyal, MD         
Forbes Norris MDA/ALS Research Center, CPMC Recruiting
San Francisco, California, United States, 94115
Contact: Dallas A. Forshew, RN, BSN    415-309-5178   
Principal Investigator: Jonathan S. Katz, MD         
United States, Florida
Mayo Clinic Florida Recruiting
Jacksonville, Florida, United States, 32224
Contact: Carla Palmucci, B.A.    904-953-3182   
Principal Investigator: Bjorn E. Oskarsson, MD         
University of Miami Miller School of Medicine Recruiting
Miami, Florida, United States, 33136
Contact: Julie Steele, RN    305-243-7336   
Principal Investigator: Michael Benatar, MD         
United States, Georgia
Emory University, Department of Neurology Recruiting
Atlanta, Georgia, United States, 30322
Contact: Meraida Polak, RN    404-778-3807   
Principal Investigator: Christina Fournier, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Christy Nichols    913-945-9945   
Principal Investigator: Richard J. Barohn, MD         
United States, Kentucky
University of Kentucky, Albert B. Chandler Medical Center Recruiting
Lexington, Kentucky, United States, 40536-0293
Contact: Meghann Bruno, RN, BSN    859-218-5064   
Principal Investigator: Edward J. Kasarskis, MD, PhD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Leah Miller    617-724-7398   
Contact: Taylor Mezoian    617-643-0312   
Principal Investigator: James Berry, MD, MPH         
United States, Minnesota
Clinical & Translational Science Institute, University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55414
Contact: Susan Rolandelli, RN    612-624-7745   
Principal Investigator: Georgios Manousakis, MD         
United States, New York
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Contact: Jess Singleton   
Principal Investigator: Jinsy Andrews, MD         
United States, North Carolina
Carolinas Medical Center, Neurosciences Instutite-Neurology Recruiting
Charlotte, North Carolina, United States, 28207
Contact: Cynthia Lary, RN    704-446-6063   
Principal Investigator: Benjamin Brooks, MD         
Duke Neurological Disorders Clinic at Morreene Road Recruiting
Durham, North Carolina, United States, 27705
Contact: Karen Grace, RN    919-668-2844   
Principal Investigator: Richard Bedlack, MD, PhD, MS         
United States, Ohio
Cleveland Clinic Foundation-Cleveland Clinic Hospital Recruiting
Cleveland, Ohio, United States, 44195
Contact: Irys B. Caristo    216-444-0173   
Principal Investigator: Erik P. Pioro, MD, PhD         
The Ohio State University Wexner Medical Center Recruiting
Columbus, Ohio, United States, 43210
Contact: Sarah Heintzman    614-293-4973   
Principal Investigator: Stephen Kolb, MD, PhD         
United States, Oregon
Providence Brain & Spine Institute, ALS Center Recruiting
Portland, Oregon, United States, 97213
Contact: Arlena Georgeson    503-962-1171   
Principal Investigator: Kimberly Goslin, MD, PhD         
United States, Texas
Houston Methodist Neurological Institute Recruiting
Houston, Texas, United States, 77030
Contact: Luis F. Lay, Jr., CCRC    713-441-3057   
Principal Investigator: Ericka Simpson, MD         
Canada, Quebec
Montreal Neurological Institute Recruiting
Montreal, Quebec, Canada, H3A 2B4
Contact: Kristiana Salmon    514-398-1779   
Principal Investigator: Angela Genge, MD         
Sponsors and Collaborators
Neuraltus Pharmaceuticals, Inc.
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. Identifier: NCT02794857     History of Changes
Other Study ID Numbers: NP001-10-003
Study First Received: May 24, 2016
Last Updated: March 20, 2017

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

Additional relevant MeSH terms:
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 processed this record on March 24, 2017