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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

NP001, Placebo



Phase 2

Study Chair(s)/Principal Investigator(s):

Gil Block, MD, PhD, Neuraltus Pharmaceuticals, Inc., Robert G. Miller, MD, California Pacific Medical Center, Jonathan Katz, MD, California Pacific Medical Center

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information


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.

Estimated Enrollment:


Estimated Study Start Date:

08 / 29 / 2016

Estimated Study Completion Date:

12 / 12 / 2017

Posting Last Modified Date:

05 / 07 / 2018

Date Study Added to neals.org:

06 / 09 / 2016

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 ]

Minimum Age:

21 Years

Maximum Age:

80 Years

Can participants use Riluzole?


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

St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics

Phoenix, Arizona 85013
United States

Mayo Clinic Arizona

Scottsdale, Arizona 85259
United States

Cedars-Sinai Medical Center

Los Angeles, California 90048
United States

University of California, Irvine, Department of Neurology

Orange, California 92862
United States

Forbes Norris MDA/ALS Research Center, CPMC

San Francisco, California 94115
United States

Mayo Clinic Florida

Jacksonville, Florida 32224
United States

University of Miami Miller School of Medicine

Miami, Florida 33136
United States

Emory University, Department of Neurology

Atlanta, Georgia 30322
United States

University of Kansas Medical Center

Kansas City, Kansas 66160
United States

University of Kentucky, Albert B. Chandler Medical Center

Lexington, Kentucky 40536-0293
United States

Massachusetts General Hospital

Boston, Massachusetts 02114
United States

Clinical & Translational Science Institute, University of Minnesota

Minneapolis, Minnesota 55414
United States

Washington University School of Medicine

Saint Louis, Missouri 63110
United States

Columbia University Medical Center

New York, New York 10032
United States

Carolinas Medical Center, Neurosciences Instutite-Neurology

Charlotte, North Carolina 28207
United States

Duke Neurological Disorders Clinic at Morreene Road

Durham, North Carolina 27705
United States

Cleveland Clinic Foundation-Cleveland Clinic Hospital

Cleveland, Ohio 44195
United States

The Ohio State University Wexner Medical Center

Columbus, Ohio 43210
United States

Providence Brain & Spine Institute, ALS Center

Portland, Oregon 97213
United States

Houston Methodist Neurological Institute

Houston, Texas 77030
United States

University of Texas Health Sciences Center San Antonio

San Antonio, Texas 78229
United States

Montreal Neurological Institute

Montreal, Quebec H3A 2B4