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Active, Not Recruiting

NCT Number: NCT05135663

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

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).

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Duchenne Muscular Dystrophy (DMD) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Kagoshima University Hospital, Kagoshima, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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).

Other names: Brogidirsen

Primary outcomes

  1. Incidence of adverse events

    Time frame: Up to Week 319

Secondary outcomes

  1. Expression of dystrophin protein (Western blot)

    Time frame: Week 99

  2. Percentage of exon 44-skipped mRNA of dystrophin

    Time frame: Week 99

  3. North Star Ambulatory Assessment (NSAA)

    Time frame: Up to Week 315

  4. Time to stand test

    Time frame: Up to Week 315

  5. Time to run/walk 10 meters test

    Time frame: Up to Week 315

  6. Six-minute walk test/Two-minute walk test

    Time frame: Up to Week 315

  7. Timed Up & Go test

    Time frame: Up to Week 315

  8. Quantitative muscle strength assessment

    Time frame: Up to Week 315

  9. Performance of Upper Limb test

    Time frame: Up to Week 315

  10. Change in serum creatine kinase concentration from baseline

    Time frame: Up to Week 315

Sponsors and collaborators

Lead sponsor

Nippon Shinyaku Co., Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2027
Study completion
2027
First posted
Nov 26, 2021
Registry last updated
Jul 20, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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