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

March 1, 2023 updated by: Nippon Shinyaku Co., Ltd.

A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy

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).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

6

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
      • Kagoshima, Japan
        • Kagoshima University Hospital
      • Tokyo, Japan
        • National Center of Neurology and Psychiatry

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NS-089/NCNP-02 40 mg/kg
Drug: NS-089/NCNP-02
The same dose administered in as Part 2 of Study NCNP/DMT02 (40 mg/kg) will be administered once weekly for 216 weeks (The total treatment period is 240 weeks including Part2 of Study NCNP/DMT02).
Drug: NS-089/NCNP-02
The same dose administered in as Part 2 of Study NCNP/DMT02 (80 mg/kg) will be administered once weekly for 216 weeks (The total treatment period is 240 weeks including Part2 of Study NCNP/DMT02).
Experimental: NS-089/NCNP-02 80 mg/kg
Drug: NS-089/NCNP-02
The same dose administered in as Part 2 of Study NCNP/DMT02 (40 mg/kg) will be administered once weekly for 216 weeks (The total treatment period is 240 weeks including Part2 of Study NCNP/DMT02).
Drug: NS-089/NCNP-02
The same dose administered in as Part 2 of Study NCNP/DMT02 (80 mg/kg) will be administered once weekly for 216 weeks (The total treatment period is 240 weeks including Part2 of Study NCNP/DMT02).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of adverse events
Time Frame: Up to Week 247
Up to Week 247

Secondary Outcome Measures

Outcome Measure
Time Frame
Expression of dystrophin protein (Western blot)
Time Frame: Week 99
Week 99
Percentage of exon 44-skipped mRNA of dystrophin
Time Frame: Week 99
Week 99
North Star Ambulatory Assessment (NSAA)
Time Frame: Up to Week 243
Up to Week 243
Time to stand test
Time Frame: Up to Week 243
Up to Week 243
Time to run/walk 10 meters test
Time Frame: Up to Week 243
Up to Week 243
Six-minute walk test/Two-minute walk test
Time Frame: Up to Week 243
Up to Week 243
Timed Up & Go test
Time Frame: Up to Week 243
Up to Week 243
Quantitative muscle strength assessment
Time Frame: Up to Week 243
Up to Week 243
Performance of Upper Limb test
Time Frame: Up to Week 243
Up to Week 243
Change in serum creatine kinase concentration from baseline
Time Frame: Up to Week 243
Up to Week 243

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nippon Shinyaku Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 23, 2021

Primary Completion (Anticipated)

January 31, 2026

Study Completion (Anticipated)

July 31, 2026

Study Registration Dates

First Submitted

October 5, 2021

First Submitted That Met QC Criteria

November 16, 2021

First Posted (Actual)

November 26, 2021

Study Record Updates

Last Update Posted (Estimate)

March 3, 2023

Last Update Submitted That Met QC Criteria

March 1, 2023

Last Verified

March 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NS089/NCNP02-P2OE
  • jRCT2031210162 (Registry Identifier: Japan Registry of Clinical Trials)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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