The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).
Inclusion Criteria:
- Probable, probable-laboratory supported, or definite ALS, either sporadic or
familial according to modified El Escorial criteria
- Disease duration < 3 years
- Age 18 to 80
- Able to provide informed consent and to comply with study procedures
- Subjects 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 lactating or able to become pregnant (e.g. post menopausal,
surgically sterile, or using adequate birth control) for the duration of the study,
and for 3 months after study completion
- Men should practice contraception for the duration of the study and for 3 months
after completion
Exclusion Criteria:
- Diagnosis of a motor neuron disease other than ALS (e.g., primary lateral sclerosis,
progressive muscular atrophy, progressive bulbar palsy)
- Clinically significant unstable medical condition (other than ALS) that would pose a
risk to the participant if they were to participate in the study or that would
impact survival or functional disability in the next 12 months
- Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine
aminotransferase (ALT) > 3 times the upper limit of the normal
- Renal insufficiency (Glomerular Filtration Rate < 60)
- Active pulmonary disease
- Prior poor compliance with an inhalation device
- The presence of unstable psychiatric disease, cognitive impairment, or dementia that
would impair ability of the participant to provide informed consent, according to
judgment by the principal investigator.
- History of human immunodeficiency virus (HIV) infection, clinically significant
chronic hepatitis, or other active infection.
- Active participation in another ALS clinical trial within 30 days of the Screening
Visit
- Other experimental treatments or anti-inflammatory/immunomodulatory medications used
in the preceding 3 months