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

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: NP001; Drug: Placebo

• Registration Number: NCT01281631
• Lead Sponsor: Neuraltus Pharmaceuticals, Inc.

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2011-01-17
• Study First Submitted QC: 2011-01-21
• Study First Posted: 2011-01-24
• Study Start: 2011-02
• Primary Completion: 2012-09
• Study Completion: 2012-09
• Status Verified: 2012-11
• Disposition First Submitted: 2012-11-20
• Disposition First Submitted QC: 2012-11-20
• Last Update Submitted: 2012-11-20
• Disposition First Posted: 2012-11-22
• Last Update Posted: 2012-11-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 136

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Low dose NP001	NP001	Low drug dose
High dose NP001	NP001	High drug dose
Placebo	Placebo	normal saline

Primary Outcome Measures

Name	Time	Method
To evaluate the effects of NP001 on measures of clinical function	over 9 months

Secondary Outcome Measures

Name	Time	Method
Safety and tolerability in ALS	duration of study
Pulmonary function and biomarkers	over 9 months

Trial Locations

Locations (17)

University of Kansas Medical Center, Landon Center on Aging; 🇺🇸 Kansas City, Kansas, United States

Massachusetts General Hospital; 🇺🇸 Charlestown, Massachusetts, United States

Columbia University; 🇺🇸 New York, New York, United States

UC, Irvine; 🇺🇸 Irvine, California, United States

The Emory Clinic; 🇺🇸 Atlanta, Georgia, United States

Providence ALS Center; 🇺🇸 Portland, Oregon, United States

UCLA; 🇺🇸 Los Angeles, California, United States

California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center; 🇺🇸 San Francisco, California, United States

SUNY Upstate Medical University, Syracuse; 🇺🇸 Syracuse, New York, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

Methodist Hospital Research Institute, Methodist Neurologic Institute; 🇺🇸 Houston, Texas, United States

Mayo Clinic, Scottsdale; 🇺🇸 Scottsdale, Arizona, United States

Mayo Clinic, Jacksonville; 🇺🇸 Jacksonville, Florida, United States

Carolinas Medical Center; 🇺🇸 Charlotte, North Carolina, United States

University of Kentucky, Department of Neurology; 🇺🇸 Lexington, Kentucky, United States

Duke University, Dept of Neurology; 🇺🇸 Durham, North Carolina, United States

Providence Saint Peter Hospital; 🇺🇸 Centralia, Washington, United States