NS-065/NCNP-01

DRUG3 trials

Sponsors

National Center of Neurology and Psychiatry, Japan, NS Pharma, Inc.

Conditions

Duchenne Muscular Dystrophy

Phase 1

Exploratory Study of NS-065/NCNP-01 in DMD

CompletedNCT02081625

National Center of Neurology and Psychiatry, JapanDuchenne Muscular Dystrophy

Start: 2013-06-30End: 2015-08-31Updated: 2020-02-26

Phase 2

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

CompletedNCT02740972

NS Pharma, Inc.Duchenne Muscular Dystrophy

Start: 2016-12-31End: 2018-04-30Updated: 2021-12-07

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

CompletedNCT03167255

NS Pharma, Inc.Duchenne Muscular Dystrophy

Start: 2017-07-06End: 2021-11-15Updated: 2022-12-28

Related Papers

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study

Journal of Neuromuscular Diseases2023-03-3133 citations

Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Journal of Neuromuscular Diseases2022-05-2455 citations

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping

JAMA Neurology2020-05-26250 citations

136. First-in-Human Study of NS-065/NCNP-01; the Morpholino Based Antisense Oligonucleotide for Exon 53 Skipping in Duchenne Muscular Dystrophy

Molecular Therapy2015-05-01