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Duchenne Muscular DystrophyPhase 2NCT04433234

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

A Phase 2 study testing DS-5141b in people with Duchenne Muscular Dystrophy, run by Daiichi Sankyo Co., Ltd. at 2 sites in Japan.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101. Read more about this study

Who is it for?
Ages 5 years and older, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
DS-5141b
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
8 participants
Where
2 sitesJapan
Study dates
30 Jun 2020to 31 Jan 2031
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 5 years and older
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Has competed a study of DS5141-A-J101

Who cannot join

  • Significant safety issues in a study of DS5141-A-J101
  • Patient who does not consent to use appropriate contraception
  • Patient not appropriate to participant in the study as determined by the Investigator

Trial locations and contacts

2 sites in 1 country.

Japan2 sites

  • Kobe University Hospital

    Hyōgo, 650-0017

  • National Center of Neurology and Psychiatry

    Tokyo, Kodaira-Shi, 187-8551

Other trials in Japan

About this study

Official title: A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.

Being tested
DS-5141b
Keywords
Duchenne muscular dystrophy, DMD, Oligonucleotides, antisense, Exon skipping, DS-5141b
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.

  • DS-5141bDrug

    Also called Renadirsen

    Administered via subcutaneous injection once weekly

Study groups in NCT04433234
Study groupRoleWhat they receive
DS-5141b 2.0 mg/kgExperimentalParticipants who will receive DS-5141b 2.0 mg/kg once weekly.
Receives: Drug: DS-5141b
DS-5141b 6.0 mg/kgExperimentalParticipants who will receive DS-5141b 6.0 mg/kg once weekly.
Receives: Drug: DS-5141b

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Adverse Events (AEs)

    Measured: From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)

  2. Change in distance walked during 6-minute walk test (6MWT)

    Measured: Every 3 months (within approximately 2 years)

  3. Change in time to stand (TTSTAND)

    Measured: Every 3 months (within approximately 2 years)

  4. Change in time in Timed up and go test

    Measured: Every 3 months (within approximately 2 years)

  5. Change in time in 10-meter Run/Walk test

    Measured: Every 3 months (within approximately 2 years)

  6. Change in score in the North Star Ambulatory Assessment (NSAA)

    Measured: Every 3 months (within approximately 2 years)

  7. Change in score in the Performance of Upper Limb (PUL)

    Measured: Every 3 months (within approximately 2 years)

  8. Change in Left Ventricular Ejection Fraction percentage (LVEF %)

    Measured: Every 6 months (within approximately 2 years)

  9. Change in in Forced Vital Capacity (FVC) (percent predicted)

    Measured: Every 6 months (within approximately 2 years)

  10. Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment

    Measured: Every 3 months (within approximately 2 years)

Secondary outcomes (1)

  1. Concentration of DS-5141a in plasma

    Measured: Every 3 months (within approximately 2 years)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Jun 2020
  2. Study start30 Jun 2020
  3. Primary completion31 Jan 2027 (estimated)
  4. Study completion31 Jan 2031 (estimated)

Resources

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
8 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04433234
Sponsor's protocol ID
DS5141-A-J201
Other IDs
205321
Status last verified
Oct 2026
First submitted
12 Jun 2020
Last updated
6 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing DS-5141b in people with Duchenne Muscular Dystrophy, run by Daiichi Sankyo 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 3 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 6 Oct 2026. 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 (AEs) (time frame: From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04433234.

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 6 Oct 2026.

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