Extension Study of NS-089/NCNP-02 in DMD
A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy
2 other identifiers
interventional
6
1 country
2
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2021
Longer than P75 for phase_2
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 23, 2021
CompletedFirst Submitted
Initial submission to the registry
October 5, 2021
CompletedFirst Posted
Study publicly available on registry
November 26, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 30, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 30, 2027
July 20, 2025
July 1, 2025
5.9 years
October 5, 2021
July 16, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Incidence of adverse events
Up to Week 319
Secondary Outcomes (10)
Expression of dystrophin protein (Western blot)
Week 99
Percentage of exon 44-skipped mRNA of dystrophin
Week 99
North Star Ambulatory Assessment (NSAA)
Up to Week 315
Time to stand test
Up to Week 315
Time to run/walk 10 meters test
Up to Week 315
- +5 more secondary outcomes
Study Arms (2)
NS-089/NCNP-02 40 mg/kg
EXPERIMENTALNS-089/NCNP-02 80 mg/kg
EXPERIMENTALInterventions
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).
Eligibility Criteria
You may qualify if:
- Patient completed Study NCNP/DMT02
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Kagoshima University Hospital
Kagoshima, Japan
National Center of Neurology and Psychiatry
Tokyo, Japan
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 5, 2021
First Posted
November 26, 2021
Study Start
June 23, 2021
Primary Completion (Estimated)
April 30, 2027
Study Completion (Estimated)
April 30, 2027
Last Updated
July 20, 2025
Record last verified: 2025-07