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Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Not yet recruiting

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

RNS60

Placebo

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

RNS60

Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

ALS functional rating scale-revised (ALSFRS-R) score

The mean change of the ALSFRS-R total score

Secondary Outcome Measures

Deaths or tracheostomies

The cumulative proportion of deaths or tracheostomies

Proportion of regulatory T cells (Treg)

The mean change in the proportion of Tregs

Slow vital capacity (SVC)

The mean change of the SVC score

ALS assessment questionnaire (ALSAQ-40) score

The mean change of the ALS assessment questionnaire (ALSAQ-40) score

Adverse events (AEs)

The mean number of AEs

Full Information

NCT ID

NCT02988297

First Posted

December 7, 2016

Last Updated

September 14, 2023

Sponsor

Revalesio Corporation

1. Study Identification

Unique Protocol Identification Number

NCT02988297

Brief Title

Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Official Title

Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Not yet recruiting

Study Start Date

May 2024 (Anticipated)

Primary Completion Date

June 2026 (Anticipated)

Study Completion Date

June 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Revalesio Corporation

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. 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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

140 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

RNS60

Arm Type

Experimental

Arm Description

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

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

RNS60

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

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

Primary Outcome Measure Information:

Title

ALS functional rating scale-revised (ALSFRS-R) score

Description

The mean change of the ALSFRS-R total score

Time Frame

24 weeks

Secondary Outcome Measure Information:

Title

Deaths or tracheostomies

Description

The cumulative proportion of deaths or tracheostomies

Time Frame

28 weeks

Title

Proportion of regulatory T cells (Treg)

Description

The mean change in the proportion of Tregs

Time Frame

24 weeks

Title

Slow vital capacity (SVC)

Description

The mean change of the SVC score

Time Frame

24 weeks

Title

ALS assessment questionnaire (ALSAQ-40) score

Description

The mean change of the ALS assessment questionnaire (ALSAQ-40) score

Time Frame

24 weeks

Title

Adverse events (AEs)

Description

The mean number of AEs

Time Frame

28 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

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

12. IPD Sharing Statement

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Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

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