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Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified April 2017 by Revalesio Corporation
Sponsor:
Information provided by (Responsible Party):
Revalesio Corporation
ClinicalTrials.gov Identifier:
NCT02988297
First received: December 7, 2016
Last updated: April 10, 2017
Last verified: April 2017
  Purpose
The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).

Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Drug: RNS60
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Investigator, Outcomes Assessor
Primary Purpose: Treatment
Official Title: Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Resource links provided by NLM:


Further study details as provided by Revalesio Corporation:

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


Secondary Outcome 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


Estimated Enrollment: 140
Anticipated Study Start Date: January 2019
Estimated Study Completion Date: January 2022
Estimated Primary Completion Date: January 2022 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

No Contacts or Locations Provided
  More Information

Responsible Party: Revalesio Corporation
ClinicalTrials.gov Identifier: NCT02988297     History of Changes
Other Study ID Numbers: 06.2.1.H6
Study First Received: December 7, 2016
Last Updated: April 10, 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

ClinicalTrials.gov processed this record on May 25, 2017