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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: NP001
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 21 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NP001<br>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<br> <br> |
| Placebo Comparator: Placebo<br>Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6 | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire |  | Baseline and 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in pulmonary function as measured by slow vital capacity readings |  | Baseline and 6 months | 
| Time to tracheotomy |  | Up to 6 months | 
| Change in levels of blood inflammatory biomarkers |  | Baseline, 3 and 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases, Inflammation, C-Reactive Protein