Amyotrophic Lateral Sclerosis
Conditions
Keywords
ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases, Inflammation, C-Reactive Protein
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.
Interventions
DRUGNP001
DRUGPlacebo
Sponsors
Neuraltus Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
21 Years to 80 Years
Healthy volunteers
No
Inclusion 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire | Baseline and 6 months |
Secondary
| Measure | 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 |
Countries
Canada, United States
Outcome results
None listed