Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation

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) and Evidence of Elevated Systemic Inflammation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02794857
• Other study ID #: NP001-10-003
• Status: Completed
• Phase: Phase 2
• Start date: August 29, 2016
• Est. completion date: December 12, 2017

Study information

• Verified date: May 2018
• Source: Neuraltus Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo.

Description:

This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS and evidence of elevated systemic inflammation. Subjects will be allocated (1:1) to NP001 and placebo. Drug or placebo will be given intravenously.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 138
• Est. completion date: December 12, 2017
• Est. primary completion date: December 12, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 21 Years to 80 Years

Eligibility

Key Inclusion Criteria:

• Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)

• Forced vital capacity greater than or equal to 65% of that predicted for age and height

• Onset of ALS-related weakness less than 3 years prior to first dose of study drug

• Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening

• Stable dose (greater than 30 days) 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

Key Exclusion Criteria:

• Life expectancy of less than 6 months

• Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)

• Active pulmonary disease

• Gastrostomy

• Stem cell therapy

• Immune modulator therapy or participation in studies of other agents within 12 weeks of pre-screening/screening

• Unstable medical condition other than ALS

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Inflammation
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• NP001

• Placebo

Locations

Country	Name	City	State
Canada	Montreal Neurological Institute	Montreal	Quebec
United States	Emory University, Department of Neurology	Atlanta	Georgia
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Carolinas Medical Center, Neurosciences Instutite-Neurology	Charlotte	North Carolina
United States	Cleveland Clinic Foundation-Cleveland Clinic Hospital	Cleveland	Ohio
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio
United States	Duke Neurological Disorders Clinic at Morreene Road	Durham	North Carolina
United States	Houston Methodist Neurological Institute	Houston	Texas
United States	Mayo Clinic Florida	Jacksonville	Florida
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of Kentucky, Albert B. Chandler Medical Center	Lexington	Kentucky
United States	Cedars-Sinai Medical Center	Los Angeles	California
United States	University of Miami Miller School of Medicine	Miami	Florida
United States	Clinical & Translational Science Institute, University of Minnesota	Minneapolis	Minnesota
United States	Columbia University Medical Center	New York	New York
United States	University of California, Irvine, Department of Neurology	Orange	California
United States	St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics	Phoenix	Arizona
United States	Providence Brain & Spine Institute, ALS Center	Portland	Oregon
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Texas Health Sciences Center San Antonio	San Antonio	Texas
United States	Forbes Norris MDA/ALS Research Center, CPMC	San Francisco	California
United States	Mayo Clinic Arizona	Scottsdale	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Neuraltus Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire		Baseline and 6 months
Secondary	Change in pulmonary function as measured by slow vital capacity readings		Baseline and 6 months
Secondary	Time to tracheotomy		Up to 6 months
Secondary	Change in levels of blood inflammatory biomarkers		Baseline, 3 and 6 months