Duchenne Muscular Dystrophy (DMD)Phase 2NCT05135663
Extension Study of NS-089/NCNP-02 in DMD
A Phase 2 study testing NS-089/NCNP-02 in people with Duchenne Muscular Dystrophy (DMD), run by Nippon Shinyaku Co., Ltd. at 2 sites in Japan.
Trial 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). Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- NS-089/NCNP-02
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 2 sitesJapan
- Study dates
- 23 Jun 2021to 30 Apr 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient completed Study NCNP/DMT02
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
Japan2 sites
Kagoshima University Hospital
Kagoshima
National Center of Neurology and Psychiatry
Tokyo
About this study
Official title: 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).
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- NS-089/NCNP-02
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NS-089/NCNP-02Drug
Also called Brogidirsen
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).
NS-089/NCNP-02Drug
Also called Brogidirsen
The same dose administered in as Part 2 of Study NCNP/DMT02 (80 mg/kg) will be administered once weekly for 288 weeks (The total treatment period is 312 weeks including Part2 of Study NCNP/DMT02).
| Study group | Role | What they receive |
|---|---|---|
| NS-089/NCNP-02 40 mg/kg | Experimental | Not described Receives: Drug: NS-089/NCNP-02 |
| NS-089/NCNP-02 80 mg/kg | Experimental | Not described Receives: Drug: NS-089/NCNP-02 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Measured: Up to Week 319
Secondary outcomes (10)
Expression of dystrophin protein (Western blot)
Measured: Week 99
Percentage of exon 44-skipped mRNA of dystrophin
Measured: Week 99
North Star Ambulatory Assessment (NSAA)
Measured: Up to Week 315
Time to stand test
Measured: Up to Week 315
Time to run/walk 10 meters test
Measured: Up to Week 315
Six-minute walk test/Two-minute walk test
Measured: Up to Week 315
Timed Up & Go test
Measured: Up to Week 315
Quantitative muscle strength assessment
Measured: Up to Week 315
Performance of Upper Limb test
Measured: Up to Week 315
Change in serum creatine kinase concentration from baseline
Measured: Up to Week 315
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2021
- Study start23 Jun 2021
- Primary completion30 Apr 2027 (estimated)
- Study completion30 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nippon Shinyaku Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05135663
- Sponsor's protocol ID
- NS089/NCNP02-P2OE
- Other IDs
- jRCT2031210162
- Status last verified
- Jul 2025
- First submitted
- 5 Oct 2021
- Last updated
- 20 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT05135663
- Acronym / sponsor ID
- NS089/NCNP02-P2OE
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Nippon Shinyaku Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 23 Jun 2021 (actual)
- Primary completion
- 30 Apr 2027 (estimated)
- Study completion
- 30 Apr 2027 (estimated)
- First posted
- 26 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Patient completed Study NCNP/DMT02
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NS-089/NCNP-02Also: BrogidirsenThe 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). | Drug | NS-089/NCNP-02 40 mg/kg |
| NS-089/NCNP-02Also: BrogidirsenThe same dose administered in as Part 2 of Study NCNP/DMT02 (80 mg/kg) will be administered once weekly for 288 weeks (The total treatment period is 312 weeks including Part2 of Study NCNP/DMT02). | Drug | NS-089/NCNP-02 80 mg/kg |
Primary outcome
Incidence of adverse events
Time frame: Up to Week 319
10 secondary outcomes
Expression of dystrophin protein (Western blot)
Time frame: Week 99
Percentage of exon 44-skipped mRNA of dystrophin
Time frame: Week 99
North Star Ambulatory Assessment (NSAA)
Time frame: Up to Week 315
Time to stand test
Time frame: Up to Week 315
Time to run/walk 10 meters test
Time frame: Up to Week 315
Six-minute walk test/Two-minute walk test
Time frame: Up to Week 315
Timed Up & Go test
Time frame: Up to Week 315
Quantitative muscle strength assessment
Time frame: Up to Week 315
Performance of Upper Limb test
Time frame: Up to Week 315
Change in serum creatine kinase concentration from baseline
Time frame: Up to Week 315
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NS-089/NCNP-02 in people with Duchenne Muscular Dystrophy (DMD), run by Nippon Shinyaku Co., Ltd. at 2 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 20 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: Up to Week 319). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2025.