Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

NCT02988297 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: 06.2.1.H6
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• ALS functional rating scale-revised (ALSFRS-R) score

  The mean change of the ALSFRS-R total score

  24 weeks

Secondary Outcomes (5)

• Deaths or tracheostomies

  28 weeks

• Proportion of regulatory T cells (Treg)

  24 weeks

• Slow vital capacity (SVC)

  24 weeks

• ALS assessment questionnaire (ALSAQ-40) score

  24 weeks

• Adverse events (AEs)

  28 weeks

Study Arms (2)

RNS60

EXPERIMENTAL

Nebulized RNS60 will be administered by daily inhalation for 24 weeks.

Drug: RNS60

Placebo

PLACEBO COMPARATOR

Nebulized Placebo will be administered by daily inhalation for 24 weeks.

Drug: Placebo

Interventions

RNS60 DRUG

Nebulized RNS60 will be administered by daily inhalation for 24 weeks.

RNS60

Placebo DRUG

Nebulized Placebo will be administered by daily inhalation for 24 weeks.

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Revalesio Corporation: lead

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

RNS60

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 7, 2016
• First Posted: December 9, 2016
• Study Start (Estimated): October 1, 2026
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): November 1, 2028
• Last Updated: April 16, 2026

Record last verified: 2026-04