A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Overview
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Brief Summary
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This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS.

Detailed Description
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This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS conducted in multiple centers. Drug or placebo will be given intravenously. Approximately 105 subjects will be enrolled. Subjects will be allocated (1:1:1) to placebo and 2 dose levels of NP001.

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)

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: NP001
* Drug: NP001
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Subjects with sporadic or familial ALS classified as definite, probable, or laboratory-supported probable ALS according to the revised El Escorial criteria. A list of key criteria is listed below: Inclusion Criteria: Onset of symptoms less than 3 years prior to study entry. Forced Vital Capacity (FVC) at least 70% of that predicted for age and height. Stable dose 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. Exclusion Criteria: Unstable medical condition(s) other than ALS. Life expectancy of less than 6 months. Require life-sustaining interventions for the 6 months following randomization. Have a tracheotomy or be using ventilatory assistance [including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)]. Active pulmonary disease. Immune modulator therapy within 12 weeks of study entry or participation in studies of other agents within the last 4 weeks prior to the randomization.

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: Low dose NP001<br>Low drug dose | Drug: NP001<br>* Low dose of NP001<br>|
| Experimental: High dose NP001<br>High drug dose | Drug: NP001<br>* High dose of NP001<br>|
| Placebo Comparator: Placebo<br>normal saline | Drug: Placebo<br>* normal saline<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the effects of NP001 on measures of clinical function |  | over 9 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability in ALS |  | duration of study | 
| Pulmonary function and biomarkers |  | over 9 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALS