Phase 1/2 study of NS-065/NCNP-01 in patients with Duchenne muscular dystrophy

Status

Unknown

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080223236

Enrollment

Registered

2016-06-08

Start date

2016-06-08

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne muscular dystrophy

Interventions

investigational material(s) Generic name etc : NS-065/NCNP-01 INN of investigational material : Therapeutic category code : 49- Other agents affecting cellular function Dosage and Administration for

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1) Ambulant or non-ambulant patient 2) Able to treat with skipping of exon 53

Exclusion criteria

Exclusion criteria: Has participated in other clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.

Design outcomes

Primary

Measure	Time frame
Efficacy	—

Secondary

Measure	Time frame
Safety, Pharmacokinetic profile	—

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026