Extension Study of NS-089/NCNP-02 in DMD | NCT05135663

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

NCT05135663

Sponsor

Nippon Shinyaku Co., Ltd.

Status

Active, not recruiting

Last Update Posted

Jul 20, 2025Actual

Enrollment

6Actual

Phase

Phase 2

Conditions

Duchenne Muscular Dystrophy (DMD)

Interventions

NS-089/NCNP-02
NS-089/NCNP-02

Countries

Japan

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
jRCT2031210162	Registry Identifier	Japan Registry of Clinical Trials

Status Module

Record Verification Date

Jul 2025

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 23, 2021Actual

Primary Completion Date

Apr 30, 2027Estimated

Completion Date

Apr 30, 2027Estimated

First Submitted Date

Oct 5, 2021

First Submission Date that Met QC Criteria

Nov 16, 2021

First Posted Date

Nov 26, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 16, 2025

Last Update Posted Date

Jul 20, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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).

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

6Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
NS-089/NCNP-02 40 mg/kg	Experimental		Drug: NS-089/NCNP-02
NS-089/NCNP-02 80 mg/kg	Experimental		Drug: NS-089/NCNP-02

Interventions

Name	Type	Description	Arm Group Labels	Other Names
NS-089/NCNP-02	Drug	The same dose administered in as Part 2 of Study NCNP/DMT02 (40 mg/kg) will be administered once weekly for 288 weeks (The total treatment period is 312 weeks including Part2 of Study NCNP/DMT02).

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Incidence of adverse events		Up to Week 319

Secondary Outcomes

Measure	Description	Time Frame
Expression of dystrophin protein (Western blot)		Week 99
Percentage of exon 44-skipped mRNA of dystrophin		Week 99
North Star Ambulatory Assessment (NSAA)

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Patient completed Study NCNP/DMT02

Exclusion Criteria:

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.
Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NCNP/DMT02.
Patient took any other investigational drugs after completion of Study NCNP/DMT02.
Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Kagoshima University Hospital		Kagoshima			Japan
National Center of Neurology and Psychiatry

Condition Browse Module

MeSH Terms

ID	Term
D020388	Muscular Dystrophy, Duchenne

Ancestor Terms

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases

Browse Leaves

Not provided

Browse Branches

Not provided