The purpose of this study is to determine the safety and tolerability of RNS60 in patients
with Amyotrophic lateral sclerosis (ALS). Investigators will also measure the impact of
RNS60 on several markers of neuro-inflammation, measured by blood biomarkers and positron
emission tomography (PET) imaging.
Amyotrophic lateral sclerosis (ALS) is a fatal, neurodegenerative disease for which there is
no cure. A substantial body of evidence implicates the neuroimmune system in ALS
pathophysiology. Of relevance to this study, microglia activation in the brain has been
found to correlate positively with faster rate of disease progression. In addition, studies
of blood cells in people with ALS have shown an increased activation of two of the major
inflammatory cell types in the body, monocytes and T cells. Among T cells, regulatory T
cells (Tregs) have been recently proposed to play a role in ALS progression.
RNS60 is an electrokinetically altered aqueous fluid. Chemically, RNS60 is composed of
saline and oxygen. The electrokinetic processing of RNS60 in Revalesio's patented Revalesio
Pump (RP) produces charge-stabilized nanostructures (CSNs) that exhibit electrical fields.
RNS60 is available for intravenous administration and inhalation. RNS60 has been extensively
tested in preclinical toxicological studies and has shown very little to no side effects. In
addition, RNS60 was well tolerated in three phase I human safety studies, one after
intravenous administration and two after inhalation.
Preclinical in vitro and in vivo studies in multiple disease models have demonstrated that
RNS60 has broad anti-inflammatory effects. These effects include reduction of microglia
activation, increase of the Tregs subpopulation of lymphocytes, and neuroprotection in
several disease models.
- Amyotrophic Lateral Sclerosis (ALS) volunteers must be diagnosed as having possible,
probable, probable-laboratory supported, or definite ALS, either sporadic or familial
according to modified El Escorial criteria.
- Age 18-80, able to provide informed consent, and comply with study procedures.
- Participants must not have taken riluzole for at least 30 days, or be on a stable
dose of riluzole for at least 30 days, prior to screening (riluzole-naïve
participants are permitted in the study).
- Women must not be able to become pregnant (e.g. post menopausal, surgically sterile,
or using adequate birth control) for the duration of the study and 3 months after
- Males should practice contraception for the duration of the study and 3 months after
- Ability to safely lie flat for 90 min for Positron Emission Tomography (PET)
procedures in the opinion of the study physician.
- High or mixed affinity to bind translocator (TSPO) protein (Ala/Ala or Ala/Thr)
- Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine
transaminase (ALT) > 3 times the upper limit of the normal.
- Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of
- The presence of unstable psychiatric disease, cognitive impairment, or dementia that
would impair ability of the participant to provide informed consent, according to PI
- Clinically significant unstable medical condition (other than ALS) that would pose a
risk to the participant if they were to participate in the study.
- History of HIV, clinically significant chronic hepatitis, or other active infection.
- Females must not be lactating or pregnant.
- Active participation in another ALS clinical trial within 30 days of the Screening
- Exposure to immunomodulatory medications within 30 days of the Screening Visit.
- Any contraindication to undergo MRI studies such as
- History of a cardiac pacemaker or pacemaker wires
- Metallic particles in the body
- Vascular clips in the head
- Prosthetic heart valves
- Radiation exposure that exceeds the site's current guidelines
- Current use of tobacco products including cigarettes, cigars, snuff and chewing
tobacco, or nicotine replacement products such as gum or patch