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Duchenne Muscular DystrophyPhase 1/2NCT04129294

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

A Phase 1/2 study testing NS-089/NCNP-02 in people with Duchenne Muscular Dystrophy, run by National Center of Neurology and Psychiatry, Japan at 1 site in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-089/NCNP-02 in subjects diagnosed with Duchenne muscular dystrophy (DMD), and to determine the dosage for subsequent studies. Read more about this study

Who is it for?
Ages 4 to 17 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
NS-089/NCNP-02
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteJapan
Study dates
2 Dec 2019to 31 May 2022
Phase
Phase 1/2
Are there results?
None posted in the registry

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 4 to 17 years
  • Men only
  • No healthy volunteers
  • Child

Who may be able to join

  • Has an out of frame deletion(s) that could be corrected by skipping exon 44 as confirmed by any of methodology at the time of visit 1. If not confirmed by any of methodology that evaluates the relative copy number of all exons (i.e. MLPA etc), must be confirmed through these techniques by the time of visit 3.
  • DNA sequencing of exon 44 confirms that no DNA polymorphisms occur that could compromise duplex formation between NS-089/NCNP-02 and pre-mRNA.
  • Male and >= 8 years and < 17 years of age at the time of obtaining informed consent and/or assent. Subjects aged >= 4 years and < 8 years can be enrolled according to the circumstances.
  • Able to give informed consent in writing signed by parent(s) or legal guardian who is able to understand all of the study procedure requirements. If applicable, able to give informed assent in writing signed by the subject.
  • Life expectancy of at least 1 year
  • Able to ambulate. Non-ambulant subject can be enrolled according to the circumstances.
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  • Have intact muscles, which have adequate quality for biopsy. (No lacks or severe atrophy of biceps brachii or tibialis anterior muscle)
  • QTc <450 msec (based on 12-lead ECGs), or <480 msec for subject with Bundle Branch Block.
  • Glucocorticoid-naive patients, or patients who have used systemic glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.

Who cannot join

  • Has participated in other pharmacological clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.
  • A forced vital capacity (FVC) < 50% of predicted.
  • Continuous use of artificial respirator (except for use of NPPV while sleeping)
  • A left ventricular ejection fraction (EF) < 40% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
  • Surgery within the last 3 months prior to the first anticipated administration of study medication or planned for anytime between visit 1 of Part 1 and the last visit of Part 2.
  • Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at screening.
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  • Current diagnosis of any immune deficiency or autoimmune disease.
  • Current diagnosis of any active or uncontrolled infection, cardiomyopathy, or liver or renal disease.
  • Use of any other investigational agents and/or experimental agents within 3 months prior to the first anticipated administration of study medication.
  • History of any severe drug allergy.

Trial locations and contacts

1 site in 1 country.

Japan1 site

  • National Center of Neurology and Psychiatry

    Kodaira, Tokyo, 1878551

Other trials in Japan

About this study

Official title: Exploratory Study of NS-089/NCNP-02 in Duchenne Muscular Dystrophy

This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-089/NCNP-02 in subjects diagnosed with Duchenne muscular dystrophy (DMD), and to determine the dosage for subsequent studies.

Being tested
NS-089/NCNP-02
Keywords
Duchenne Muscular Dystrophy
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

    NS-089/NCNP-02 for Infusion is packaged as 50 mg/mL with 3 mL per vial. Study dosages will be infused over a 1 hour period at the following dose levels.

    "[Part 1] NS-089/NCNP-02 is administered at dose levels 1 and 3 in Cohort 1 and at dose levels 2 and 4 in Cohort 2.

    Dose level 1: 1.62 mg/kg once weekly for 2 weeks; Dose level 2: 10 mg/kg once weekly for 2 weeks; Dose level 3: 40 mg/kg once weekly for 2 weeks; Dose level 4: 80 mg/kg once weekly for 2 weeks [Part 2] Based on the results from Part 1, two dosages are selected as study dosages in Part 2. Each selected dose are administered once a week for 24 weeks."

Study groups in NCT04129294
Study groupRoleWhat they receive
NS-089/NCNP-02ExperimentalNS-089/NCNP-02
Receives: Drug: NS-089/NCNP-02

What is being measured

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

  1. Adverse event and adverse drug reaction [Safety and Tolerability]

    adverse event and adverse drug reaction

    Measured: At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period)

Secondary outcomes (10)

  1. Expression of dystrophin protein

    Expression of dystrophin protein

    Measured: At the end of the treatment period (24 weeks) of Part 2

  2. NSAA

    North Star Ambulatory Assessment

    Measured: At the end of the treatment period (24 weeks) of Part 2

  3. TTSTAND

    Time to Stand Test

    Measured: At the end of the treatment period (24 weeks) of Part 2

  4. TTRW

    Time to Run/Walk 10 Meters test

    Measured: At the end of the treatment period (24 weeks) of Part 2

  5. 6MWT and 2MWT

    Six-Minute Walk Test (6MWT) and Two-Minute Walk Test (2MWT)

    Measured: At the end of the treatment period (24 weeks) of Part 2

  6. TUG

    Timed Up \& Go (TUG) test

    Measured: At the end of the treatment period (24 weeks) of Part 2

  7. PUL

    Performance of Upper Limb test

    Measured: At the end of the treatment period (24 weeks) of Part 2

  8. Detection of exon 44-skipped mRNA of dystrophin in muscle tissue

    Detection of exon 44-skipped mRNA of dystrophin in muscle tissue

    Measured: At the end of the treatment period (24 weeks) of Part 2

  9. NS-089/NCNP-02 concentration of the blood plasma

    NS-089/NCNP-02 concentration of the blood plasma

    Measured: At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period)

  10. Serum Creatine kinase concentration

    Serum Creatine kinase concentration

    Measured: At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period)

Timeline

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

  1. First posted16 Oct 2019
  2. Study start2 Dec 2019
  3. Primary completion31 May 2022
  4. Study completion31 May 2022

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04129294
Sponsor's protocol ID
NCNP/DMT02
Other IDs
UMIN000038505
Status last verified
Sep 2022
First submitted
15 Oct 2019
Last updated
29 Sep 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Komaki H, Takeshita E, Kunitake K, Ishizuka T, Shimizu-Motohashi Y, Ishiyama A, Sasaki M, Yonee C, Maruyama S, Hida E, Aoki Y. Phase 1/2 trial of brogidirsen: Dual-targeting antisense oligonucleotides for exon 44 skipping in Duchenne muscular dystrophy. Cell Rep Med. 2025 Jan 21;6(1):101901. doi: 10.1016/j.xcrm.2024.101901. Epub 2025 Jan 9.

    PubMed 39793573

  • Ishizuka T, Komaki H, Asahina Y, Nakamura H, Motohashi N, Takeshita E, Shimizu-Motohashi Y, Ishiyama A, Yonee C, Maruyama S, Hida E, Aoki Y. Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial. Neuropsychopharmacol Rep. 2023 Jun;43(2):277-286. doi: 10.1002/npr2.12335. Epub 2023 Apr 3.

    PubMed 37326950

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing NS-089/NCNP-02 in people with Duchenne Muscular Dystrophy, run by National Center of Neurology and Psychiatry, Japan at 1 site 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 9 inclusion and 10 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: finished, as of the sponsor's last update on 29 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Adverse event and adverse drug reaction [Safety and Tolerability] (time frame: At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period)). 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 NCT04129294, which also lists 2 linked publications.

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 29 Sep 2022.

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