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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
NP001
Placebo
Sponsored by
Neuraltus Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases, Inflammation, C-Reactive Protein

Eligibility Criteria

21 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
  • Mayo Clinic Arizona
  • Cedars-Sinai Medical Center
  • University of California, Irvine, Department of Neurology
  • Forbes Norris MDA/ALS Research Center, CPMC
  • Mayo Clinic Florida
  • University of Miami Miller School of Medicine
  • Emory University, Department of Neurology
  • University of Kansas Medical Center
  • University of Kentucky, Albert B. Chandler Medical Center
  • Massachusetts General Hospital
  • Clinical & Translational Science Institute, University of Minnesota
  • Washington University School of Medicine
  • Columbia University Medical Center
  • Carolinas Medical Center, Neurosciences Instutite-Neurology
  • Duke Neurological Disorders Clinic at Morreene Road
  • Cleveland Clinic Foundation-Cleveland Clinic Hospital
  • The Ohio State University Wexner Medical Center
  • Providence Brain & Spine Institute, ALS Center
  • Houston Methodist Neurological Institute
  • University of Texas Health Sciences Center San Antonio
  • Montreal Neurological Institute

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

NP001

Placebo

Arm Description

NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6

Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6

Outcomes

Primary Outcome Measures

Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire

Secondary Outcome Measures

Change in pulmonary function as measured by slow vital capacity readings
Time to tracheotomy
Change in levels of blood inflammatory biomarkers

Full Information

First Posted
May 24, 2016
Last Updated
May 3, 2018
Sponsor
Neuraltus Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02794857
Brief Title
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

2. Study Status

Record Verification Date
May 2018
Overall Recruitment Status
Completed
Study Start Date
August 29, 2016 (Actual)
Primary Completion Date
December 12, 2017 (Actual)
Study Completion Date
December 12, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neuraltus Pharmaceuticals, Inc.

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
138 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NP001
Arm Type
Experimental
Arm Description
NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
Intervention Type
Drug
Intervention Name(s)
NP001
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire
Time Frame
Baseline and 6 months
Secondary Outcome Measure Information:
Title
Change in pulmonary function as measured by slow vital capacity readings
Time Frame
Baseline and 6 months
Title
Time to tracheotomy
Time Frame
Up to 6 months
Title
Change in levels of blood inflammatory biomarkers
Time Frame
Baseline, 3 and 6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gil Block, MD, PhD
Organizational Affiliation
Neuraltus Pharmaceuticals, Inc.
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Robert G. Miller, MD
Organizational Affiliation
California Pacific Medical Center
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jonathan Katz, MD
Organizational Affiliation
California Pacific Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Facility Name
Mayo Clinic Arizona
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
Cedars-Sinai Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
University of California, Irvine, Department of Neurology
City
Orange
State/Province
California
ZIP/Postal Code
92862
Country
United States
Facility Name
Forbes Norris MDA/ALS Research Center, CPMC
City
San Francisco
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
Mayo Clinic Florida
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Emory University, Department of Neurology
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Kentucky, Albert B. Chandler Medical Center
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536-0293
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Clinical & Translational Science Institute, University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55414
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Carolinas Medical Center, Neurosciences Instutite-Neurology
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28207
Country
United States
Facility Name
Duke Neurological Disorders Clinic at Morreene Road
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Facility Name
Cleveland Clinic Foundation-Cleveland Clinic Hospital
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
The Ohio State University Wexner Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Providence Brain & Spine Institute, ALS Center
City
Portland
State/Province
Oregon
ZIP/Postal Code
97213
Country
United States
Facility Name
Houston Methodist Neurological Institute
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Texas Health Sciences Center San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Montreal Neurological Institute
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada

12. IPD Sharing Statement

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

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