Neuraltus NP001
/in Study /by Joanne WuuOfficial Title: Phase II Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS) and Evidence of Elevated Systemic Inflammation.
Principal Investigators: Robert G. Miller, MD (California Pacific Medical Center), Jonathan Katz, MD (California Pacific Medical Center)
Site PI: Michael Benatar, MD, PhD (University of Miami)
This is a 6 month 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 administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6.
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
Research Coordinator
- Julie Steele, RN
Contact Information
- Email: j.steele@med.miami.edu
Back to Research Studies
This is a longitudinal study designed to assess whether electrical impedance myography (EIM) is a useful measure of disease progression in ALS. EIM is a painless and non-invasive technique that can be used to quantify the biophysical properties of muscle. The study will involve several visits to a study center for serial evaluations. If successful, this study will demonstrate that the use of EIM as an outcome measure would make it possible to complete clinical trials in a shorter period of time and require participation of a small number of people.
The Cochrane Collaboration is an organization, based in the UK that is dedicated to producing systematic reviews and meta-analyses of the best available evidence in the medical literature that is relevant to a specific topic. We have authored reviews of topics such as the treatment of familial ALS, and are currently working on one about the use of enteral feeding in patients with ALS.
This is an ancillary study to Pre-fALS and Arimocomol (University of Miami site only).
