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NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
NCT05996003 · View on ClinicalTrials.gov ↗
Study Summary
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.
Conditions Studied
Interventions
- DRUG NS-089/NCNP-02
Study Locations (20)
Pennsylvania
- The Children's Hospital of Philadelphia (CHOP) — Philadelphia
- University of Pittsburgh School of Medicine — Pittsburgh
Colorado
- Children's Hospital Colorado — Aurora
Florida
- Rare Disease Research, LLC - FL — Kissimmee
Georgia
- Rare Disease Research — Atlanta
Illinois
- Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago
Kansas
- University of Kansas Medical Center (KUMC) — Kansas City
Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati
Texas
- UT Southwestern/Children's Health — Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2024-02-22 |
| Est. Completion | 2026-09-11 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05996003
The ClinicalTrials.gov registry entry for NCT05996003 describes a study currently listed as recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is NS Pharma, which has 6 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 3 conditions, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention — of which NS-089/NCNP-02 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05996003 reports 20 study locations spanning 19 distinct geographic areas — top geographies include Pennsylvania, Colorado, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05996003 about?
NCT05996003 is a clinical study titled "NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)". This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The stu...
What is the current status of trial NCT05996003?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 20 participants. The study started on 2024-02-22. Estimated completion is 2026-09-11.
What conditions does trial NCT05996003 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy, DMD, Exon 44. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05996003?
The interventions under investigation include: NS-089/NCNP-02 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05996003?
This trial is sponsored by NS Pharma, which has 6 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05996003 being conducted?
This trial has 20 study locations across Colorado, Florida, Georgia, Illinois, Kansas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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