A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

NP001, Placebo



Phase 2

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

Robert G. Miller, MD, Forbes Norris ALS Treatment and Research, California Pacific Medical Center

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Forbes Norris ALS Treatment and Research, California Pacific Medical Center

2324 Sacramento Street, Suite 150
San Francisco, California, United States

Full Study Summary:

This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS conducted in multiple centers. Drug or placebo will be given intravenously. Approximately 105 subjects will be enrolled. Subjects will be allocated (1:1:1) to placebo and 2 dose levels of NP001.

Study Sponsor:

Neuraltus Pharmaceuticals, Inc.

Estimated Enrollment:


Estimated Study Start Date:

01 / 31 / 2011

Estimated Study Completion Date:

09 / 01 / 2012

Posting Last Modified Date:

11 / 22 / 2012

Date Study Added to neals.org:

01 / 24 / 2011

Minimum Age:

21 Years

Maximum Age:

80 Years

Can participants use Riluzole?


Subjects with sporadic or familial ALS classified as definite, probable, or laboratory-supported probable ALS according to the revised El Escorial criteria. A list of key criteria is listed below:

Inclusion Criteria:

- Onset of symptoms less than 3 years prior to study entry.

- Forced Vital Capacity (FVC) at least 70% of that predicted for age and height.

- Stable dose 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.

Exclusion Criteria:

- Unstable medical condition(s) other than ALS.

- Life expectancy of less than 6 months.

- Require life-sustaining interventions for the 6 months following randomization.

- Have a tracheotomy or be using ventilatory assistance [including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)].

- Active pulmonary disease.

- Immune modulator therapy within 12 weeks of study entry or participation in studies of other agents within the last 4 weeks prior to the randomization.

Mayo Clinic, Scottsdale

Scottsdale, Arizona 85259
United States

UC, Irvine

Irvine, California 92868-4281
United States


Los Angeles, California 90095
United States

California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center

San Francisco, California 94115
United States

Mayo Clinic, Jacksonville

Jacksonville, Florida 32224
United States

The Emory Clinic

Atlanta, Georgia 30322
United States

University of Kansas Medical Center, Landon Center on Aging

Kansas City, Kansas 66160
United States

University of Kentucky, Department of Neurology

Lexington, Kentucky 40536-0284
United States

Massachusetts General Hospital

Charlestown, Massachusetts 02129
United States

Columbia University

New York, New York 10032
United States

SUNY Upstate Medical University, Syracuse

Syracuse, New York 13210
United States

Carolinas Medical Center

Charlotte, North Carolina 28207
United States

Duke University, Dept of Neurology

Durham, North Carolina 27705
United States

Cleveland Clinic

Cleveland, Ohio 44195
United States

Providence ALS Center

Portland, Oregon 97213
United States

Methodist Hospital Research Institute, Methodist Neurologic Institute

Houston, Texas 77030
United States

Providence Saint Peter Hospital

Centralia, Washington 98531
United States