Extension Study of NS-089/NCNP-02 in DMD

Nippon Shinyaku

Status and phase: Active, not recruiting; Phase 2

Conditions: Duchenne Muscular Dystrophy (DMD)

Treatments: Drug: NS-089/NCNP-02

Study type: Interventional

Funder types: Industry

Identifiers

NCT05135663

jRCT2031210162 (Registry Identifier)

NS089/NCNP02-P2OE

Details and patient eligibility

About

This is the extension study of NS-089/NCNP-02 (Study NCNP/DMT02), which is designed to assess the safety, tolerability and efficacy of NS-089/NCNP-02 in patients with Duchenne muscular dystrophy (DMD).

Enrollment: 6 patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patient completed Study NCNP/DMT02

Exclusion criteria

1. Patient had any serious adverse events in Study NCNP/DMT02 that, in the opinion of the Investigator and/or Sponsor, was probably or definitely related to NS-089/NCNP-02 use and precludes safe use of NS-089/NCNP-02 for the patient in this study.
2. Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NCNP/DMT02.
3. Patient took any other investigational drugs after completion of Study NCNP/DMT02.
4. Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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6 participants in 2 patient groups

NS-089/NCNP-02 40 mg/kg

Experimental group

Treatment:

Drug: NS-089/NCNP-02

Drug: NS-089/NCNP-02

NS-089/NCNP-02 80 mg/kg

Experimental group

Treatment:

Drug: NS-089/NCNP-02

Drug: NS-089/NCNP-02