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

Phase 2

Completed

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

• Registration Number: NCT02794857
• Lead Sponsor: Neuraltus Pharmaceuticals, Inc.

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.

Study Timeline

• Study First Submitted: 2016-05-24
• Study First Submitted QC: 2016-06-06
• Study First Posted: 2016-06-09
• Study Start: 2016-08-29
• Primary Completion: 2017-12-12
• Study Completion: 2017-12-12
• Status Verified: 2018-05
• Last Update Submitted: 2018-05-03
• Last Update Posted: 2018-05-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 138

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NP001	NP001	NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
Placebo	Placebo	Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6

Primary Outcome Measures

Name	Time	Method
Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire	Baseline and 6 months

Secondary Outcome Measures

Name	Time	Method
Change in pulmonary function as measured by slow vital capacity readings	Baseline and 6 months
Change in levels of blood inflammatory biomarkers	Baseline, 3 and 6 months
Time to tracheotomy	Up to 6 months

Trial Locations

Locations (22)

University of California, Irvine, Department of Neurology; 🇺🇸 Orange, California, United States

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

University of Miami Miller School of Medicine; 🇺🇸 Miami, Florida, United States

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

Forbes Norris MDA/ALS Research Center, CPMC; 🇺🇸 San Francisco, California, United States

Emory University, Department of Neurology; 🇺🇸 Atlanta, Georgia, United States

University of Kentucky, Albert B. Chandler Medical Center; 🇺🇸 Lexington, Kentucky, United States

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

Clinical & Translational Science Institute, University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

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

Duke Neurological Disorders Clinic at Morreene Road; 🇺🇸 Durham, North Carolina, United States

Cleveland Clinic Foundation-Cleveland Clinic Hospital; 🇺🇸 Cleveland, Ohio, United States

Carolinas Medical Center, Neurosciences Instutite-Neurology; 🇺🇸 Charlotte, North Carolina, United States

The Ohio State University Wexner Medical Center; 🇺🇸 Columbus, Ohio, United States

Providence Brain & Spine Institute, ALS Center; 🇺🇸 Portland, Oregon, United States

Houston Methodist Neurological Institute; 🇺🇸 Houston, Texas, United States

University of Texas Health Sciences Center San Antonio; 🇺🇸 San Antonio, Texas, United States

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

Washington University School of Medicine; 🇺🇸 Saint Louis, Missouri, United States

Montreal Neurological Institute; 🇨🇦 Montreal, Quebec, Canada

St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics; 🇺🇸 Phoenix, Arizona, United States

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