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A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

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. Identifier: NCT01281631
Recruitment Status : Completed
First Posted : January 24, 2011
Last Update Posted : November 22, 2012
Information provided by (Responsible Party):
Neuraltus Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: NP001 Drug: Placebo Phase 2

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 136 participants
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)
Study Start Date : February 2011
Actual Primary Completion Date : September 2012
Actual Study Completion Date : September 2012

Arm Intervention/treatment
Experimental: Low dose NP001
Low drug dose
Drug: NP001
Low dose of NP001

Experimental: High dose NP001
High drug dose
Drug: NP001
High dose of NP001

Placebo Comparator: Placebo
normal saline
Drug: Placebo
normal saline

Primary Outcome Measures :
  1. To evaluate the effects of NP001 on measures of clinical function [ Time Frame: over 9 months ]

Secondary Outcome Measures :
  1. Safety and tolerability in ALS [ Time Frame: duration of study ]
  2. Pulmonary function and biomarkers [ Time Frame: over 9 months ]

Information from the National Library of Medicine

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

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.

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 identifier (NCT number): NCT01281631

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United States, Arizona
Mayo Clinic, Scottsdale
Scottsdale, Arizona, United States, 85259
United States, California
UC, Irvine
Irvine, California, United States, 92868-4281
Los Angeles, California, United States, 90095
California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center
San Francisco, California, United States, 94115
United States, Florida
Mayo Clinic, Jacksonville
Jacksonville, Florida, United States, 32224
United States, Georgia
The Emory Clinic
Atlanta, Georgia, United States, 30322
United States, Kansas
University of Kansas Medical Center, Landon Center on Aging
Kansas City, Kansas, United States, 66160
United States, Kentucky
University of Kentucky, Department of Neurology
Lexington, Kentucky, United States, 40536-0284
United States, Massachusetts
Massachusetts General Hospital
Charlestown, Massachusetts, United States, 02129
United States, New York
Columbia University
New York, New York, United States, 10032
SUNY Upstate Medical University, Syracuse
Syracuse, New York, United States, 13210
United States, North Carolina
Carolinas Medical Center
Charlotte, North Carolina, United States, 28207
Duke University, Dept of Neurology
Durham, North Carolina, United States, 27705
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States, 44195
United States, Oregon
Providence ALS Center
Portland, Oregon, United States, 97213
United States, Texas
Methodist Hospital Research Institute, Methodist Neurologic Institute
Houston, Texas, United States, 77030
United States, Washington
Providence Saint Peter Hospital
Centralia, Washington, United States, 98531
Sponsors and Collaborators
Neuraltus Pharmaceuticals, Inc.
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Principal Investigator: Robert G. Miller, MD Forbes Norris ALS Treatment and Research, California Pacific Medical Center

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Responsible Party: Neuraltus Pharmaceuticals, Inc. Identifier: NCT01281631     History of Changes
Other Study ID Numbers: NP001-10-002
First Posted: January 24, 2011    Key Record Dates
Last Update Posted: November 22, 2012
Last Verified: November 2012
Keywords provided by Neuraltus Pharmaceuticals, Inc.:
Additional relevant MeSH terms:
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Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases