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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01281631
• Other study ID #: NP001-10-002
• Status: Completed
• Phase: Phase 2
• First received: January 17, 2011
• Last updated: November 20, 2012
• Start date: February 2011
• Est. completion date: September 2012

Study information

• Verified date: November 2012
• Source: Neuraltus Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 136
• Est. completion date: September 2012
• Est. primary completion date: September 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 21 Years to 80 Years

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• NP001
Low dose of NP001
• NP001
High dose of NP001
• Placebo
normal saline

Locations

Country	Name	City	State
United States	The Emory Clinic	Atlanta	Georgia
United States	Providence Saint Peter Hospital	Centralia	Washington
United States	Massachusetts General Hospital	Charlestown	Massachusetts
United States	Carolinas Medical Center	Charlotte	North Carolina
United States	Cleveland Clinic	Cleveland	Ohio
United States	Duke University, Dept of Neurology	Durham	North Carolina
United States	Methodist Hospital Research Institute, Methodist Neurologic Institute	Houston	Texas
United States	UC, Irvine	Irvine	California
United States	Mayo Clinic, Jacksonville	Jacksonville	Florida
United States	University of Kansas Medical Center, Landon Center on Aging	Kansas City	Kansas
United States	University of Kentucky, Department of Neurology	Lexington	Kentucky
United States	UCLA	Los Angeles	California
United States	Columbia University	New York	New York
United States	Providence ALS Center	Portland	Oregon
United States	California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center	San Francisco	California
United States	Mayo Clinic, Scottsdale	Scottsdale	Arizona
United States	SUNY Upstate Medical University, Syracuse	Syracuse	New York

Sponsors (1)

Lead Sponsor	Collaborator
Neuraltus Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the effects of NP001 on measures of clinical function		over 9 months	No
Secondary	Safety and tolerability in ALS		duration of study	Yes
Secondary	Pulmonary function and biomarkers		over 9 months	No