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A pilot trial of RNS60 in amyotrophic lateral sclerosis
Author(s) -
Pagai Sabrina,
Alshikho Mohamad J.,
Luppino Sarah,
Chan James,
Pothier Lindsay,
Schoenfeld David,
Andres Patricia L.,
Babu Suma,
Zürcher Nicole R.,
Loggia Marco L.,
Barry Robert L.,
Luotti Silvia,
Nardo Giovanni,
Trolese Maria Chiara,
Pantalone Serena,
Bendotti Caterina,
Bonetto Valentina,
De Marchi Fabiola,
Rosen Bruce,
Hooker Jacob,
Cudkowicz Merit,
Atassi Nazem
Publication year - 2019
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.26385
Subject(s) - tolerability , medicine , amyotrophic lateral sclerosis , adverse effect , neuroprotection , multiple sclerosis , clinical trial , oncology , disease , immunology
ABSTRACT Introduction : RNS60 is a novel immune‐modulatory agent that has shown neuroprotective effects in amytrophic lateral sclerosis (ALS) preclinical models. RNS60 is administered by weekly intravenous infusion and daily nebulization. The objective of this pilot open‐label trial was to test the feasibility, safety, and tolerability of long‐term RNS60 administration in ALS patients. Methods : The planned treatment duration was 23 weeks and the primary outcomes were safety and tolerability. Secondary outcomes included PBR28 positron emission tomography (PET) imaging and plasma biomarkers of inflammation. Results : Sixteen participants with ALS received RNS60 and 13 (81%) completed 23 weeks of RNS60 treatment. There were no serious adverse events and no participants withdrew from the trial due to drug‐related adverse events. There were no significant changes in the biomarkers. Discussion : Long‐term RNS60 administration was safe and well‐tolerated. A large, multicenter, phase II trial of RNS60 is currently enrolling participants to test the effects of RNS60 on ALS biomarkers and disease progression. Muscle Nerve 59 :303–308, 2019