The Effect of RNS60 on ALS Biomarkers

• Register: ClinicalTrials.gov
• Last refreshed on: 1 May 2023
• Main ID: NCT03456882
• Date of registration: 02/02/2018
• Prospective Registration: No
• Primary sponsor: Mario Negri Institute for Pharmacological Research
• Public title: The Effect of RNS60 on ALS Biomarkers RNS60
• Scientific title: The Effect of RNS60 on ALS Biomarkers
• Date of first enrolment: May 30, 2017
• Target sample size: 147
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03456882
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Other. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Italy, United States

Contacts

• Name: Ettore Beghi, MD
• Affiliation: IRCCS Istituto di ricerche farmacologiche Mario Negri di Milano

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age 18 through 80 years inclusive;

2. Geographically accessible to the site and able to come to the site once a week for 24
weeks;

3. Definite, probable, probable laboratory supported ALS diagnosis according to the
revised El Escorial criteria; 4) Disease duration 6 to 24 months from symptom onset;

5) Self sufficiency: Satisfactory bulbar and spinal function (score 3+ on the ALSFRS-R for
swallowing, cutting food and handling utensils, and walking); 6) Satisfactory respiratory
function (FVC =80% of predicted); 7) Documented progression of symptoms in the last three
months, as measured by the ALSFRS-R scale; 8) Ability to understand and comply with the
study requirements and to give written informed consent personally or via a legally
authorized representative; 9) Treatment with riluzole 50 mg twice/day for at least 1 month
prior to screening visit.

Self sufficiency: this term reflect independence in daily living activities. It is an
intuitive parameter to indicate preservation of key functional activities, and - not least
- it has shown to be a valid and reliable measure

Exclusion Criteria:

1. History of HIV, clinically significant chronic hepatitis, antecedent polio infection,
or other active infection;

2. Motor neuron disease (MND) other than ALS;

3. Involvement of systems other than motor possibly determining a functional impairment
(as measured by the end-points) for the entire duration of the study;

4. Other severe clinical conditions (e.g., cardiovascular disorders, neoplasms) with
impact on survival or functional disability in the next 12 months;

5. Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of
normal;

6. Poor compliance with previous treatments;

7. Other experimental treatments in the preceding 3 months;

8. Women who are lactating or able to become pregnant (e.g. who are not post menopausal,
surgically sterile, or using inadequate birth control) and men unable to practice
contraception for the duration of the treatment and 3 months after its completion;

9. Unwillingness or inability to take riluzole; 10) Poor capability to use an inhalation
device;

10. Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of the
normal.



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: RNS60

Primary Outcome(s)

Pharmacodynamic Biomarkers: Nfl On-treatment Period Variation [Time Frame: 24 weeks (week 0 - week 24)]

Pharmacodynamic Biomarkers: Cyp-A On-treatment Period Variation [Time Frame: 24 weeks (week 0 - week 24)]

Pharmacodynamic Biomarkers: Actin-NT On-treatment Period Variation [Time Frame: 24 weeks (week 0 - week 24)]

Pharmacodynamic Biomarkers: MCP-1 On-treatment Period Variation [Time Frame: 24 weeks (week 0 - week 24)]

Pharmacodynamic Biomarkers: CD25 mRNA On-treatment Period Variation [Time Frame: 24 weeks (week 0 - week 24)]

Pharmacodynamic Biomarkers: IL-17 On-treatment Period Variation [Time Frame: 24 weeks (week 0 - week 24)]

Pharmacodynamic Biomarkers: 3-NT On-treatment Period Variation [Time Frame: 24 weeks (week 0 - week 24)]

Pharmacodynamic Biomarkers: FOXP3 mRNA On-treatment Period Variation [Time Frame: 24 weeks (week 0 - week 24)]

Secondary Outcome(s)

Pharmacodynamic Biomarkers: 3-NT Off-treatment Period Variation [Time Frame: 24 weeks (week 24 - week 48)]

Pharmacodynamic Biomarkers: MCP-1 Off-treatment Period Variation [Time Frame: 24 weeks (week 24 - week 48)]

Survival [Time Frame: 24 weeks on-treatment period (week 0 - week 24) + 24 weeks off-treatment follow-up period (week 24 - week 48)]

ALSFRS-R On-treatment and Off-treatment Variation [Time Frame: 24 weeks on-treatment period (week 0 - week 24) + 24 weeks off-treatment follow-up period (week 24 - week 48)]

ALSAQ-40 Scale [Time Frame: 24 weeks on-treatment period (week 0 - week 24) + 24 weeks off-treatment follow-up period (week 24 - week 48)]

FVC% On-treatment and Off-treatment Variation [Time Frame: 24 weeks on-treatment period (week 0 - week 24) + 24 weeks off-treatment follow-up period (week 24 - week 48)]

Pharmacodynamic Biomarkers: Actin-NT Off-treatment Period Variation [Time Frame: 24 weeks (week 24 - week 48)]

Mean Number of AE [Time Frame: 24 weeks on-treatment period (week 0 - week 24) + 24 weeks off-treatment follow-up period (week 24 - week 48)]

Pharmacodynamic Biomarkers: Cyp-A Off-treatment Period Variation [Time Frame: 24 weeks (week 24 - week 48)]

Pharmacodynamic Biomarkers: FOXP3 mRNA Off-treatment Period Variation [Time Frame: 24 weeks (week 24 - week 48)]

AE Leading to Treatment Discontinuation [Time Frame: 24 weeks on-treatment period]

Pharmacodynamic Biomarkers: CD25 mRNA Off-treatment Period Variation [Time Frame: 24 weeks (week 24 - week 48)]

Pharmacodynamic Biomarkers: IL-17 Off-treatment Period Variation [Time Frame: 24 weeks (week 24 - week 48)]

Secondary ID(s)

RNS60

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Get out ONLUS

ALS Association

Results

• Results available: Yes
• Date Posted: 21/04/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03456882