A Pilot Study of RNS60 in Amyotrophic Lateral Sclerosis (ALS)
Condition: ALS
Intervention: RNS60
Intervention description: RNS60 will be administered in two ways: by intravenous (IV) infusion one day a week (infusion dose: 375ml, infused over a 40-min period) and by inhalation (the remaining 6 days a week, 4 ml/day) for 23 weeks.
In addition, subjects will be given the option to continue to receive drug for approximately an additional 24 weeks, for a total of approximately 48 weeks on study drug. Study drug will be given by intravenous (IV) infusion one day a week (infusion dose: 375ml, infused over a 40-min period) and by inhalation (the remaining 6 days a week, 4 ml/day) during the extension phase.
Intervention MeSH terms: RNS60
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 24.0
Sex: All
Minimum age: 18 Years
Maximum age: 80 Years
Healthy volunteers: No

Summary:

      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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  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
             study completion.

          -  Males should practice contraception for the duration of the study and 3 months after
             completion.

          -  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)

        Exclusion Criteria:

          -  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
             normal.

          -  The presence of unstable psychiatric disease, cognitive impairment, or dementia that
             would impair ability of the participant to provide informed consent, according to PI
             judgment.

          -  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
             Visit

          -  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

               -  Claustrophobia

          -  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