Amyotrophic Lateral Sclerosis
Conditions
Brief summary
The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).
Interventions
DRUGRNS60
Nebulized RNS60 will be administered by daily inhalation for 24 weeks.
DRUGPlacebo
Nebulized Placebo will be administered by daily inhalation for 24 weeks.
Sponsors
Revalesio Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ALS functional rating scale-revised (ALSFRS-R) score | 24 weeks | The mean change of the ALSFRS-R total score |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Deaths or tracheostomies | 28 weeks | The cumulative proportion of deaths or tracheostomies |
| Proportion of regulatory T cells (Treg) | 24 weeks | The mean change in the proportion of Tregs |
| Slow vital capacity (SVC) | 24 weeks | The mean change of the SVC score |
| ALS assessment questionnaire (ALSAQ-40) score | 24 weeks | The mean change of the ALS assessment questionnaire (ALSAQ-40) score |
| Adverse events (AEs) | 28 weeks | The mean number of AEs |
Outcome results
None listed