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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Neuraltus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT01281631
First received: January 17, 2011
Last updated: November 20, 2012
Last verified: November 2012
History of Changes
  Purpose

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


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

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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)

Resource links provided by NLM:

U.S. FDA Resources

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

Primary Outcome Measures:
  • To evaluate the effects of NP001 on measures of clinical function [ Time Frame: over 9 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety and tolerability in ALS [ Time Frame: duration of study ] [ Designated as safety issue: Yes ]
  • Pulmonary function and biomarkers [ Time Frame: over 9 months ] [ Designated as safety issue: No ]

Enrollment: 136
Study Start Date: February 2011
Study Completion Date: September 2012
Primary Completion Date: September 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   21 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01281631

Locations
United States, Arizona
Mayo Clinic, Scottsdale
Scottsdale, Arizona, United States, 85259
United States, California
UC, Irvine
Irvine, California, United States, 92868-4281
UCLA
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.
Investigators
Principal Investigator: Robert G. Miller, MD Forbes Norris ALS Treatment and Research, California Pacific Medical Center
  More Information

No publications provided

Responsible Party: Neuraltus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01281631     History of Changes
Other Study ID Numbers: NP001-10-002
Study First Received: January 17, 2011
Last Updated: November 20, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Neuraltus Pharmaceuticals, Inc.:
ALS

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

ClinicalTrials.gov processed this record on June 17, 2013