A Pilot Study of RNS60 in Amyotrophic Lateral Sclerosis (ALS)
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|ClinicalTrials.gov Identifier: NCT02525471|
Recruitment Status : Completed
First Posted : August 17, 2015
Last Update Posted : February 22, 2018
|Condition or disease||Intervention/treatment||Phase|
|ALS||Drug: RNS60||Phase 1|
Amyotrophic lateral sclerosis (ALS) is a fatal, neurodegenerative disease for which there is no cure. A substantial body of evidence implicates the neuroimmune system in ALS pathophysiology. Of relevance to this study, microglia activation in the brain has been found to correlate positively with faster rate of disease progression. In addition, studies of blood cells in people with ALS have shown an increased activation of two of the major inflammatory cell types in the body, monocytes and T cells. Among T cells, regulatory T cells (Tregs) have been recently proposed to play a role in ALS progression.
RNS60 is an electrokinetically altered aqueous fluid. Chemically, RNS60 is composed of saline and oxygen. The electrokinetic processing of RNS60 in Revalesio's patented Revalesio Pump (RP) produces charge-stabilized nanostructures (CSNs) that exhibit electrical fields. RNS60 is available for intravenous administration and inhalation. RNS60 has been extensively tested in preclinical toxicological studies and has shown very little to no side effects. In addition, RNS60 was well tolerated in three phase I human safety studies, one after intravenous administration and two after inhalation.
Preclinical in vitro and in vivo studies in multiple disease models have demonstrated that RNS60 has broad anti-inflammatory effects. These effects include reduction of microglia activation, increase of the Tregs subpopulation of lymphocytes, and neuroprotection in several disease models.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Study Start Date :||October 2015|
|Actual Primary Completion Date :||June 21, 2017|
|Actual Study Completion Date :||October 18, 2017|
Following screening visit to determine eligibility, enrolled subjects will undergo the baseline visit within 6 weeks where the first intravenous (IV) infusion of study medication, RNS60, will be administered. Study medication for inhalation use will be dispensed at this time, and again at weeks 7 and 15. Subjects will continue once a week follow ups to receive RNS60 by IV infusion, continuing inhalation use the remaining 6 days per week, for 24 weeks total. Additionally, eligible subjects will undergo PET imaging at baseline and again between weeks 18 and 23.
In addition, upon nearing completion of the core study, subjects will be given the option to continue to receive drug for approximately an additional 24 weeks, for a total of approximately 48 weeks on study drug, following the optional extension phase schedule of activities.
RNS60 will be administered in two ways: by intravenous (IV) infusion one day a week (infusion dose: 375ml, infused over a 40-min period) and by inhalation (the remaining 6 days a week, 4 ml/day) for 24 weeks.
In addition, subjects will be given the option to continue to receive drug for approximately an additional 24 weeks, for a total of approximately 48 weeks on study drug. Study drug will be given by intravenous (IV) infusion one day a week (infusion dose: 375ml, infused over a 40-min period) and by inhalation (the remaining 6 days a week, 4 ml/day) during the extension phase.
- Safety as measured by the Number of Participants Experiencing Adverse Events [ Time Frame: 24 weeks ]Safety will be assessed by the occurrence of adverse events.
- Tolerability to Complete the Entire 24 Week Study on Study Drug [ Time Frame: 24 weeks ]Tolerability will be defined as the ability of subjects to complete the entire 24-week study on study drug.
- Blood biomarkers of inflammation. [ Time Frame: 24 weeks ]
- Neuroimaging biomarkers ([11C]-PBR28-Positron Emission Tomography (PET)) including both regions of interest and voxel-based analyses. [ Time Frame: 24 weeks ]
- Clinical outcome: Pulmonary Function, Measured by Slow Vital Capacity (SVC) [ Time Frame: 24 weeks ]
- Clinical outcome: Strength, Measured by Accurate Test of Limb Isometric Strength (ATLIS) [ Time Frame: 24 weeks ]
- Clinical outcome: Functional Status, Measured by Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: 24 weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02525471
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|Principal Investigator:||Sabrina Paganoni, MD||Massachusetts General Hospital|
|Principal Investigator:||Nazem Atassi, MD||Massachusetts General Hospital|