Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02988297
• Other study ID #: 06.2.1.H6
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: October 2024
• Est. completion date: November 2026

Study information

• Verified date: February 2024
• Source: Revalesio Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 140
• Est. completion date: November 2026
• Est. primary completion date: November 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• RNS60
Nebulized RNS60 will be administered by daily inhalation for 24 weeks.
• Placebo
Nebulized Placebo will be administered by daily inhalation for 24 weeks.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Revalesio Corporation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ALS functional rating scale-revised (ALSFRS-R) score	The mean change of the ALSFRS-R total score	24 weeks
Secondary	Deaths or tracheostomies	The cumulative proportion of deaths or tracheostomies	28 weeks
Secondary	Proportion of regulatory T cells (Treg)	The mean change in the proportion of Tregs	24 weeks
Secondary	Slow vital capacity (SVC)	The mean change of the SVC score	24 weeks
Secondary	ALS assessment questionnaire (ALSAQ-40) score	The mean change of the ALS assessment questionnaire (ALSAQ-40) score	24 weeks
Secondary	Adverse events (AEs)	The mean number of AEs	28 weeks