A pilot trial of RNS60 in amyotrophic lateral sclerosis.
Administration, Inhalation
Adult
Aged
Amyotrophic Lateral Sclerosis
/ diagnostic imaging
Anti-Inflammatory Agents, Non-Steroidal
/ administration & dosage
Biomarkers
/ analysis
Brain
/ diagnostic imaging
Female
Healthy Volunteers
Humans
Infusions, Intravenous
Male
Middle Aged
Muscle Strength
Neuroimaging
Pilot Projects
Positron-Emission Tomography
Sodium Chloride
/ adverse effects
Treatment Outcome
Young Adult
ALS
PBR28
clinical trial
motor neuron disease (MND)
neuroinflammation
Journal
Muscle & nerve
ISSN: 1097-4598
Titre abrégé: Muscle Nerve
Pays: United States
ID NLM: 7803146
Informations de publication
Date de publication:
03 2019
03 2019
Historique:
received:
03
08
2018
revised:
11
11
2018
accepted:
13
11
2018
pubmed:
21
11
2018
medline:
6
7
2019
entrez:
21
11
2018
Statut:
ppublish
Résumé
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. 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. 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. 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.
Identifiants
pubmed: 30458059
doi: 10.1002/mus.26385
pmc: PMC6379136
mid: NIHMS998614
doi:
Substances chimiques
Anti-Inflammatory Agents, Non-Steroidal
0
Biomarkers
0
RNS60
0
Sodium Chloride
451W47IQ8X
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
303-308Subventions
Organisme : NINDS NIH HHS
ID : K23 NS083715
Pays : United States
Organisme : NIH HHS
ID : S10 OD023517
Pays : United States
Organisme : NCRR NIH HHS
ID : S10 RR026666
Pays : United States
Organisme : MGH ALS Therapy Fund
Pays : International
Informations de copyright
© 2018 Wiley Periodicals, Inc.
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