A pilot trial of RNS60 in amyotrophic lateral sclerosis

Sabrina Paganoni; Mohamad J. Alshikho; Sarah Luppino; James Chan; Lindsay Pothier; David Schoenfeld; Patricia L. Andres; Suma Babu; Nicole R. Zürcher; Marco L. Loggia; Robert L. Barry; Silvia Luotti; Giovanni Nardo; Maria Chiara Trolese; Serena Pantalone; Caterina Bendotti; Valentina Bonetto; Fabiola De Marchi; Bruce Rosen; Jacob Hooker; Merit Cudkowicz; Nazem Atassi

doi:10.1002/mus.26385

A pilot trial of RNS60 in amyotrophic lateral sclerosis

Sabrina Paganoni, Mohamad J. Alshikho, Sarah Luppino, James Chan, Lindsay Pothier, David Schoenfeld, Patricia L. Andres, Suma Babu, Nicole R. Zürcher, Marco L. Loggia, Robert L. Barry, Silvia Luotti, Giovanni Nardo, Maria Chiara Trolese, Serena Pantalone, Caterina Bendotti, Valentina Bonetto, Fabiola De Marchi, Bruce Rosen, Jacob HookerMerit Cudkowicz, Nazem Atassi

Risultato della ricerca: Contributo su rivista › Articolo in rivista › peer review

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.

Lingua originale	Inglese
pagine (da-a)	303-308
Numero di pagine	6
Rivista	Muscle and Nerve
Volume	59
Numero di pubblicazione	3
DOI	https://doi.org/10.1002/mus.26385
Stato di pubblicazione	Pubblicato - mar 2019
Pubblicato esternamente	Sì

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10.1002/mus.26385

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Paganoni, S., Alshikho, M. J., Luppino, S., Chan, J., Pothier, L., Schoenfeld, D., Andres, P. L., Babu, S., Zürcher, N. R., Loggia, M. L., Barry, R. L., Luotti, S., Nardo, G., Trolese, M. C., Pantalone, S., Bendotti, C., Bonetto, V., De Marchi, F., Rosen, B., ... Atassi, N. (2019). A pilot trial of RNS60 in amyotrophic lateral sclerosis. Muscle and Nerve, 59(3), 303-308. https://doi.org/10.1002/mus.26385

@article{d09ecb5485b4428c9cca40bbb684514b,

title = "A pilot trial of RNS60 in amyotrophic lateral sclerosis",

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.",

keywords = "ALS, PBR28, clinical trial, motor neuron disease (MND), neuroinflammation",

author = "Sabrina Paganoni and Alshikho, {Mohamad J.} and Sarah Luppino and James Chan and Lindsay Pothier and David Schoenfeld and Andres, {Patricia L.} and Suma Babu and Z{\"u}rcher, {Nicole R.} and Loggia, {Marco L.} and Barry, {Robert L.} and Silvia Luotti and Giovanni Nardo and Trolese, {Maria Chiara} and Serena Pantalone and Caterina Bendotti and Valentina Bonetto and {De Marchi}, Fabiola and Bruce Rosen and Jacob Hooker and Merit Cudkowicz and Nazem Atassi",

note = "Publisher Copyright: {\textcopyright} 2018 Wiley Periodicals, Inc.",

year = "2019",

month = mar,

doi = "10.1002/mus.26385",

language = "English",

volume = "59",

pages = "303--308",

journal = "Muscle and Nerve",

issn = "0148-639X",

publisher = "John Wiley & Sons Inc.",

number = "3",

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Paganoni, S, Alshikho, MJ, Luppino, S, Chan, J, Pothier, L, Schoenfeld, D, Andres, PL, Babu, S, Zürcher, NR, Loggia, ML, Barry, RL, Luotti, S, Nardo, G, Trolese, MC, Pantalone, S, Bendotti, C, Bonetto, V, De Marchi, F, Rosen, B, Hooker, J, Cudkowicz, M & Atassi, N 2019, 'A pilot trial of RNS60 in amyotrophic lateral sclerosis', Muscle and Nerve, vol. 59, n. 3, pagg. 303-308. https://doi.org/10.1002/mus.26385

TY - JOUR

T1 - A pilot trial of RNS60 in amyotrophic lateral sclerosis

AU - Paganoni, Sabrina

AU - Alshikho, Mohamad J.

AU - Luppino, Sarah

AU - Chan, James

AU - Pothier, Lindsay

AU - Schoenfeld, David

AU - Andres, Patricia L.

AU - Babu, Suma

AU - Zürcher, Nicole R.

AU - Loggia, Marco L.

AU - Barry, Robert L.

AU - Luotti, Silvia

AU - Nardo, Giovanni

AU - Trolese, Maria Chiara

AU - Pantalone, Serena

AU - Bendotti, Caterina

AU - Bonetto, Valentina

AU - De Marchi, Fabiola

AU - Rosen, Bruce

AU - Hooker, Jacob

AU - Cudkowicz, Merit

AU - Atassi, Nazem

PY - 2019/3

Y1 - 2019/3

N2 - 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.

AB - 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.

KW - ALS

KW - PBR28

KW - clinical trial

KW - motor neuron disease (MND)

KW - neuroinflammation

UR - http://www.scopus.com/inward/record.url?scp=85059009389&partnerID=8YFLogxK

U2 - 10.1002/mus.26385

DO - 10.1002/mus.26385

M3 - Article

SN - 0148-639X

VL - 59

SP - 303

EP - 308

JO - Muscle and Nerve

JF - Muscle and Nerve

IS - 3

ER -

A pilot trial of RNS60 in amyotrophic lateral sclerosis

Abstract

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