Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation
- Registration Number
- NCT02794857
- Lead Sponsor
- Neuraltus Pharmaceuticals, Inc.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 138
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description NP001 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 Placebo Placebo Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
- Primary Outcome Measures
Name Time Method Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire Baseline and 6 months
- Secondary Outcome Measures
Name Time Method Change in pulmonary function as measured by slow vital capacity readings Baseline and 6 months Change in levels of blood inflammatory biomarkers Baseline, 3 and 6 months Time to tracheotomy Up to 6 months
Trial Locations
- Locations (22)
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
🇺🇸Phoenix, Arizona, United States
Mayo Clinic Arizona
🇺🇸Scottsdale, Arizona, United States
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
University of California, Irvine, Department of Neurology
🇺🇸Orange, California, United States
Forbes Norris MDA/ALS Research Center, CPMC
🇺🇸San Francisco, California, United States
Mayo Clinic Florida
🇺🇸Jacksonville, Florida, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Emory University, Department of Neurology
🇺🇸Atlanta, Georgia, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
University of Kentucky, Albert B. Chandler Medical Center
🇺🇸Lexington, Kentucky, United States
Scroll for more (12 remaining)St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics🇺🇸Phoenix, Arizona, United States