NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Public ClinicalTrials.gov record NCT05996003. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT05996003
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 20 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years to 14 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 22, 2024 (Actual)
- Primary completion
- Feb 26, 2027 (Estimated)
- Completion
- Feb 26, 2027 (Estimated)
- Last update posted
- Sep 15, 2026
2024 – 2027
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Rare Disease Research | Atlanta | Georgia | 30329 | — |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| University of Kansas Medical Center (KUMC) | Kansas City | Kansas | 66160 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Columbia University Pediatric Neuromuscular Center | New York | New York | 10032 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| The Children's Hospital of Philadelphia (CHOP) | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh School of Medicine | Pittsburgh | Pennsylvania | 15224 | — |
| UT Southwestern/Children's Health | Dallas | Texas | 75207 | — |
| Virginia Commonwealth University Health System | Richmond | Virginia | 23298 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 18 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05996003, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 15, 2026 · Synced Oct 27, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT05996003 live on ClinicalTrials.gov.