Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Overview

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

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Triple (Participant, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: June 2024

Interventions

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

Arms, Groups and Cohorts

  • Experimental: 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.

Clinical Trial Outcome Measures

Primary Measures

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

Secondary Measures

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

Participating in This Clinical Trial

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-na├»ve 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

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 80 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Revalesio Corporation
  • Provider of Information About this Clinical Study
    • Sponsor

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