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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02794857
Recruitment Status : Completed
First Posted : June 9, 2016
Last Update Posted : May 7, 2018
Sponsor:
Information provided by (Responsible Party):
Neuraltus Pharmaceuticals, Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: NP001 Drug: Placebo Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 138 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : August 29, 2016
Actual Primary Completion Date : December 12, 2017
Actual Study Completion Date : December 12, 2017

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 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
 Drug: NP001
Placebo Comparator: Placebo
Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
 Drug: Placebo


Outcome Measures
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Primary Outcome Measures :
  1. Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire [ Time Frame: Baseline and 6 months ]

Secondary Outcome Measures :
  1. Change in pulmonary function as measured by slow vital capacity readings [ Time Frame: Baseline and 6 months ]
  2. Time to tracheotomy [ Time Frame: Up to 6 months ]
  3. Change in levels of blood inflammatory biomarkers [ Time Frame: Baseline, 3 and 6 months ]

Eligibility Criteria
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Ages Eligible for Study:   21 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02794857


Locations
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Sponsors and Collaborators
Neuraltus Pharmaceuticals, Inc.
Investigators
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Study Director: Gil Block, MD, PhD Neuraltus Pharmaceuticals, Inc.
Principal Investigator: Robert G. Miller, MD California Pacific Medical Center
Principal Investigator: Jonathan Katz, MD California Pacific Medical Center
More Information
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Responsible Party: Neuraltus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02794857    
Other Study ID Numbers: NP001-10-003
First Posted: June 9, 2016    Key Record Dates
Last Update Posted: May 7, 2018
Last Verified: May 2018
Keywords provided by Neuraltus Pharmaceuticals, Inc.:
ALS
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Central Nervous System Diseases
Nervous System Diseases
 Neurodegenerative Diseases
Neuromuscular Diseases
Inflammation
C-Reactive Protein
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Inflammation
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases