Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Phase 2

Not yet recruiting

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: RNS60; Drug: Placebo

• Registration Number: NCT02988297
• Lead Sponsor: Revalesio Corporation

Brief Summary

The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-12-07
• Study First Submitted QC: 2016-12-07
• Study First Posted: 2016-12-09
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-30
• Last Update Posted: 2025-05-04
• Study Start: 2026-04
• Primary Completion: 2028-05
• Study Completion: 2028-05

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 140

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
RNS60	RNS60	Nebulized RNS60 will be administered by daily inhalation for 24 weeks.
Placebo	Placebo	Nebulized Placebo will be administered by daily inhalation for 24 weeks.

Primary Outcome Measures

Name	Time	Method
ALS functional rating scale-revised (ALSFRS-R) score	24 weeks	The mean change of the ALSFRS-R total score

Secondary Outcome Measures

Name	Time	Method
ALS assessment questionnaire (ALSAQ-40) score	24 weeks	The mean change of the ALS assessment questionnaire (ALSAQ-40) score
Slow vital capacity (SVC)	24 weeks	The mean change of the SVC score
Proportion of regulatory T cells (Treg)	24 weeks	The mean change in the proportion of Tregs
Deaths or tracheostomies	28 weeks	The cumulative proportion of deaths or tracheostomies
Adverse events (AEs)	28 weeks	The mean number of AEs