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Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT02988297
Recruitment Status : Not yet recruiting
First Posted : December 9, 2016
Last Update Posted : April 12, 2017
Sponsor:
Information provided by (Responsible Party):

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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: RNS60 Drug: Placebo Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 140 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis
Anticipated Study Start Date : January 2019
Estimated Primary Completion Date : January 2022
Estimated Study Completion Date : January 2022


Arms and Interventions

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.


Outcome Measures

Primary Outcome Measures :
  1. ALS functional rating scale-revised (ALSFRS-R) score [ Time Frame: 24 weeks ]
    The mean change of the ALSFRS-R total score


Secondary Outcome Measures :
  1. Deaths or tracheostomies [ Time Frame: 28 weeks ]
    The cumulative proportion of deaths or tracheostomies

  2. Proportion of regulatory T cells (Treg) [ Time Frame: 24 weeks ]
    The mean change in the proportion of Tregs

  3. Slow vital capacity (SVC) [ Time Frame: 24 weeks ]
    The mean change of the SVC score

  4. ALS assessment questionnaire (ALSAQ-40) score [ Time Frame: 24 weeks ]
    The mean change of the ALS assessment questionnaire (ALSAQ-40) score

  5. Adverse events (AEs) [ Time Frame: 28 weeks ]
    The mean number of AEs


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
More Information

Responsible Party: Revalesio Corporation
ClinicalTrials.gov Identifier: NCT02988297     History of Changes
Other Study ID Numbers: 06.2.1.H6
First Posted: December 9, 2016    Key Record Dates
Last Update Posted: April 12, 2017
Last Verified: April 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases