Study Purpose:
The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).Study Status:
Not yet recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
RNS60, Placebo
Placebo:
Yes
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
N/A
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Study Sponsor:
Revalesio Corporation
Estimated Enrollment:
140
Estimated Study Start Date:
04 / 01 / 2024
Estimated Study Completion Date:
05 / 01 / 2026
Posting Last Modified Date:
03 / 29 / 2023
Date Study Added to neals.org:
12 / 09 / 2016
Primary Outcome Measures:
The mean change of the ALS functional rating scale-revised (ALSFRS-R) total score [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
The cumulative proportion of deaths or tracheostomies [ Time Frame: 28 weeks ] [ Designated as safety issue: No ]
The mean change in the proportion of regulatory T cells (Treg) [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
The mean change of the slow vital capacity (SVC) score [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
The mean change of the ALS assessment questionnaire (ALSAQ-40) score [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
The mean number of adverse events (AEs) [ Time Frame: 28 weeks ] [ Designated as safety issue: Yes ]
Minimum Age:
18 Years
Maximum Age:
80 Years
Can participants use Riluzole?
Yes
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