Clinical trial NCT02794857: Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Inflammation
C-Reactive Protein
Lead sponsor: Neuraltus Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2016-06-09
Enrollment: 138
Sex: ALL
Minimum age (years): 21
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire
timeFrame: Baseline and 6 months
Secondary outcomes:
measure: Change in pulmonary function as measured by slow vital capacity readings
timeFrame: Baseline and 6 months
measure: Time to tracheotomy
timeFrame: Up to 6 months
measure: Change in levels of blood inflammatory biomarkers
timeFrame: Baseline, 3 and 6 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
state: Arizona
city: Scottsdale
country: United States
facility: Mayo Clinic Arizona
state: Arizona
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Orange
country: United States
facility: University of California, Irvine, Department of Neurology
state: California
city: San Francisco
country: United States
facility: Forbes Norris MDA/ALS Research Center, CPMC
state: California
city: Jacksonville
country: United States
facility: Mayo Clinic Florida
state: Florida
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Atlanta
country: United States
facility: Emory University, Department of Neurology
state: Georgia
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky, Albert B. Chandler Medical Center
state: Kentucky
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: Clinical & Translational Science Institute, University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Charlotte
country: United States
facility: Carolinas Medical Center, Neurosciences Instutite-Neurology
state: North Carolina
city: Durham
country: United States
facility: Duke Neurological Disorders Clinic at Morreene Road
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation-Cleveland Clinic Hospital
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Portland
country: United States
facility: Providence Brain & Spine Institute, ALS Center
state: Oregon
city: Houston
country: United States
facility: Houston Methodist Neurological Institute
state: Texas
city: San Antonio
country: United States
facility: University of Texas Health Sciences Center San Antonio
state: Texas
city: Montreal
country: Canada
facility: Montreal Neurological Institute
state: Quebec
Study officials:
affiliation: Neuraltus Pharmaceuticals, Inc.
name: Gil Block, MD, PhD
role: STUDY_DIRECTOR
affiliation: California Pacific Medical Center
name: Robert G. Miller, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: California Pacific Medical Center
name: Jonathan Katz, MD
role: PRINCIPAL_INVESTIGATOR
References: