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The Effect of RNS60 on ALS Biomarkers

NCT03456882 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 147

Last updated 2023-04-21

Study results available
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Summary

Amyotrophic Lateral Sclerosis (ALS) is a rare lethal neurodegenerative disease involving inflammation. Riluzole, the only drug for ALS, improves median survival by 3 months. This prompts new treatments of ALS. RNS60 is an experimental drug with favorable effects in preclinical studies of neuroinflammation and neurodegeneration. Based on significant efficacy demonstrated in preclinical studies and its excellent clinical safety profile, RNS60 is a promising candidate for a drug to treat ALS. Developing a pharmacodynamic marker will be a first and important step for dose finding and exploration of the mechanism of action in human, and pave the way to trials measuring drug efficacy.

The Investigator propose a multicenter, randomized, double-blind, placebo-controlled, parallel group, Phase II trial. The study centers will be located in Italy and at Massachusetts General Hospital (MGH) in Boston. A total of 142 ALS patients will be randomly assigned to RNS60 or placebo (administered by intravenous infusion once/week and inhaled via nebulization every morning for 24 weeks). All participants will also take riluzole (50-mg tablet twice/day). Blood samples for biomarker analysis (protein, RNA) will be collected in the screening period, on day 1, week 4,12 and 24. Both safety and potential therapeutic effects of RNS60 will be also assessed.

Conditions

Interventions

DRUG

RNS60

normal saline plus oxigen in nanobubble

Sponsors & Collaborators

  • ALS Association

    collaborator OTHER

  • Get Out Onlus

    collaborator OTHER

  • Mario Negri Institute for Pharmacological Research

    lead OTHER

Principal Investigators

  • Ettore Beghi, MD · IRCCS Istituto di ricerche farmacologiche Mario Negri di Milano

Study Design

Allocation
RANDOMIZED
Purpose
OTHER
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-05-30
Primary Completion
2020-11-23
Completion
2021-05-30
FDA Drug
Yes

Countries

  • United States
  • Italy

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03456882 on ClinicalTrials.gov