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

NS Pharma

Status and phase: Completed; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: NS-065/NCNP-01

Study type: Interventional

Funder types: NETWORK; Industry; Other

Identifiers

NCT03167255

NS-065/NCNP-01-202

Details and patient eligibility

About

This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

Full description

This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose levels of up to 80 mg/kg/week administered by weekly IV infusion over an additional treatment period of 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.

Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.

Enrollment: 16 patients

Sex: Male

Ages: 4 to 10 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Completed Study NS-065/NCNP-01-201 through Week 25.
2. Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures.
3. Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.

Exclusion criteria

1. Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of NS-065/NCNP-01.
2. Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completion of Study NS-065/NCNP-01-201.
3. Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-201.
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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16 participants in 2 patient groups

NS-065/NCNP-01 40mg/kg

Experimental group

Description:

Patients receiving 40mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.

Treatment:

Drug: NS-065/NCNP-01

NS-065/NCNP-01 80mg/kg

Experimental group

Description:

Patients receiving 80mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.

Treatment:

Drug: NS-065/NCNP-01