Clinical trial NCT02988297: Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis
Official title: Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Revalesio Corporation
Sponsor class: INDUSTRY
First posted: 2016-12-09
Enrollment: 140
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility 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

Primary outcomes:
measure: ALS functional rating scale-revised (ALSFRS-R) score
timeFrame: 24 weeks
Secondary outcomes:
measure: Deaths or tracheostomies
timeFrame: 28 weeks
measure: Proportion of regulatory T cells (Treg)
timeFrame: 24 weeks
measure: Slow vital capacity (SVC)
timeFrame: 24 weeks
measure: ALS assessment questionnaire (ALSAQ-40) score
timeFrame: 24 weeks
measure: Adverse events (AEs)
timeFrame: 28 weeks
Other outcomes:
Locations:
Study officials:
References: