Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

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

Study Overview

• Status: Not yet recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: RNS60; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 140
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RNS60; Nebulized RNS60 will be administered by daily inhalation for 24 weeks.	Drug: RNS60; Nebulized RNS60 will be administered by daily inhalation for 24 weeks.
Placebo Comparator: Placebo; Nebulized Placebo will be administered by daily inhalation for 24 weeks.	Drug: Placebo; Nebulized Placebo will be administered by daily inhalation for 24 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ALS functional rating scale-revised (ALSFRS-R) score	The mean change of the ALSFRS-R total score	24 weeks

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Revalesio Corporation

Study record dates

Study Major Dates

• Study Start (Estimated): October 1, 2024
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026

Study Registration Dates

• First Submitted: December 7, 2016
• First Submitted That Met QC Criteria: December 7, 2016
• First Posted (Estimated): December 9, 2016

Study Record Updates

• Last Update Posted (Estimated): February 26, 2024
• Last Update Submitted That Met QC Criteria: February 23, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Physiological Effects of Drugs; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; Anti-Inflammatory Agents, Non-Steroidal; Analgesics, Non-Narcotic; Anti-Inflammatory Agents; Antirheumatic Agents; Pharmaceutical Solutions; RNS60
• Other Study ID Numbers: 06.2.1.H6

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No