Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Revalesio

Status and phase: Begins enrollment in 7 months; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo; Drug: RNS60

Study type: Interventional

Funder types: Industry

Identifiers

NCT02988297

06.2.1.H6

Details and patient eligibility

About

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

Enrollment: 140 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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140 participants in 2 patient groups, including a placebo group

RNS60

Experimental group

Description:

Nebulized RNS60 will be administered by daily inhalation for 24 weeks.

Treatment:

Drug: RNS60

Placebo

Placebo Comparator group

Description:

Nebulized Placebo will be administered by daily inhalation for 24 weeks.

Treatment:

Drug: Placebo