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Duchenne Muscular DystrophyPhase 1/2NCT06053814

NS-050/NCNP-03 in Boys With DMD (Meteor50)

A Phase 1/2 study testing NS-050/NCNP-03 in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 19 sites in 5 countries.

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 Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping. Read more about this study

Who is it for?
Ages 4 to 15 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
NS-050/NCNP-03
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
20 participants
Where
19 sitesin 5 countries
Study dates
18 Sep 2024to Mar 2028
Phase
Phase 1/2
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 4 to 15 years
  • Men only
  • No healthy volunteers
  • Child

Who may be able to join

  • Male ≥ 4 years and <16 years of age;
  • Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame;
  • Able to walk independently without assistive devices;
  • Able to complete the TTSTAND without assistance in <20 seconds;
  • Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.
  • Other inclusion criteria may apply.

Who cannot join

  • Evidence of symptomatic cardiomyopathy;
  • Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug;
  • Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug;
  • Surgery within the 3 months prior to the first dose of study drug or planned during the study duration;
  • Having taken any gene therapy.
  • Other exclusion criteria may apply.

Trial locations and contacts

19 sites in 5 countries.

19 sites in 5 countries

United States6 sites

  • Children's Hospital Colorado

    Aurora, Colorado, 80011

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • University of Kansas Medical Center

    Kansas City, Kansas, 66103

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213

Other trials in United States

Japan5 sites

  • National Center of Neurology and Psychiatry

    Kodaira, Tokyo, 187-8551

  • National Hospital Organization Nagara Medical Center

    Nagara, Gifu, 502-8558

  • Hyogo Medical University Hospital

    Nishinomiya, Hyōgo, 663-8501

  • Miyagi Children's Hospital

    Sendai, Miyagi, 989-3126

  • NHO Osaka Toneyama Medical Center

    Toyonaka, Osaka, 560-8552

Other trials in Japan

Canada3 sites

  • Alberta Children's Hospital

    Calgary, Alberta

  • London Health Sciences Centre

    London, Ontario

  • British Columbia Children's Hospital

    Vancouver, British Columbia

Other trials in Canada

Turkey (Türkiye)3 sites

  • Ankara Bilkent City Hospital

    Ankara

  • Istanbul University

    Istanbul

  • Yeditepe University Kosuyolu Hospital

    Istanbul

Other trials in Turkey (Türkiye)

South Korea2 sites

  • Seoul National University Hospital

    Seoul

  • Pusan National University Yangsan Hospital

    Yangsan, Gyeongsangnam-do

Other trials in South Korea

About this study

Official title: A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys With Duchenne Muscular Dystrophy (DMD)

This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.

Being tested
NS-050/NCNP-03, Placebo
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-050/NCNP-03Drug

    NS-050/NCNP-03 solution for IV infusion.

  • PlaceboDrug

    NS-050/NCNP-03 placebo-matching solution for IV infusion.

Study groups in NCT06053814
Study groupRoleWhat they receive
Part 1: NS-050/NCNP-03ExperimentalParticipants will be randomized and receive NS-050/NCNP-03 intravenous (IV) infusions once weekly for 2 weeks at each of MAD levels (1.95, 5, 10, 20, 40, and 80 mg/kg).
Receives: Drug: NS-050/NCNP-03
Part 1: PlaceboPlacebo comparatorParticipants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels.
Receives: Drug: Placebo
Part 2: NS-050/NCNP-03ExperimentalParticipants will receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the Data and Safety Monitoring Board (DSMB) at the conclusion of Part 1.
Receives: Drug: NS-050/NCNP-03

What is being measured

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

  1. Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs)

    TEAEs will be summarized both at the patient level for number of TEAEs, highest severity, relationship, action, and outcome, and at the TEAE level (summarizing events) by system organ class (SOC) and preferred term (PT) as well as severity, relationship, action, and outcome.

    The most recent version of the Medical Dictionary for Regulatory Activities (MedDRA) will be used for coding TEAEs.

    Measured: Baseline up to Week 24

  2. Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03

    Blood samples will be collected at the designated time frame. Pharmacokinetic (PK) parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods.

    Measured: Day 1 (1st dose) for each dose level

  3. Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03

    Urine samples will be collected at the designated time frame. PK parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods.

    Measured: Day 1 (1st dose) for each dose level

  4. Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot)

    Measured: Baseline, Week25

Secondary outcomes (11)

  1. Part 2: Change from baseline in skeletal muscle dystrophin protein by mass spectrometry

    Measured: Baseline, Week25

  2. Part 2: Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining

    Measured: Baseline, Week25

  3. Part 2: Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin

    Measured: Baseline, Week25

  4. Part 2: North Star Ambulatory Assessment (NSAA) score

    The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.

    Measured: Baseline, Week13, Week25

  5. Part 2: Time to Stand (TTSTAND)

    Measured: Baseline, Week13, Week25

  6. Part 2: Time to Run/Walk 10 Meters (TTRW)

    Measured: Baseline, Week13, Week25

  7. Part 2: Time to Climb 4 Stairs (TTCLIMB)

    Measured: Baseline, Week13, Week25

  8. Part 2: Total distance of 6 Minute Walk Test (6MWT)

    Measured: Baseline, Week13, Week25

  9. Part 2: Muscle strength measured by Quantitative Muscle Testing (QMT)

    Measured: Baseline, Week13, Week25

  10. Part 2: Grip/Pinch Strength

    Measured: Baseline, Week13, Week25

  11. Part 2: Performance of Upper Limb (PUL) 2.0. score

    The PUL 2.0 provides both a total score and sub-scores for the 3 domains (shoulder, middle, and distal) that in DMD are progressively involved with a proximal to distal gradient. The PUL includes 22 items with an entry item to define the starting functional level. The 22 items are subdivided into the high level shoulder dimension (6 items), middle level elbow dimension (9 items), and distal wrist and hand dimension (7 items). For weaker patients, a low score on the entry item (0 2) means high level items do not need to be performed. Scoring options vary across the scale between 0-1 and 0-2 according to performance. Each dimension can be scored separately with a maximum score of 12 for the high level shoulder dimension, 17 for the middle level elbow dimension, and 13 for the distal wrist and hand dimension. A total score can be achieved by adding the 3 level scores (maximum total score of 42).

    Measured: Baseline, Week13, Week25

Timeline

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

  1. First posted26 Sep 2023
  2. Study start18 Sep 2024
  3. Primary completionMar 2028 (estimated)
  4. Study completionMar 2028 (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 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06053814
Sponsor's protocol ID
NS-050/NCNP-03-101
Status last verified
Jun 2026
First submitted
19 Sep 2023
Last updated
16 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing NS-050/NCNP-03 in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 19 sites in 5 countries.

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 6 inclusion and 6 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 16 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States, Japan, Canada, Turkey (Türkiye), South Korea. 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. Some participants in this study may receive a placebo. 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?

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?

Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs) (time frame: Baseline up to Week 24). The study lists 4 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 NCT06053814.

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 16 Jun 2026.

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