Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Public ClinicalTrials.gov record NCT02740972. 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 II, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.
Study identification
- NCT ID
- NCT02740972
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 16 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years to 9 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
- Dec 2016 (Actual)
- Primary completion
- Mar 2018 (Actual)
- Completion
- Apr 2018 (Actual)
- Last update posted
- Dec 7, 2021
2016 – 2018
United States locations
- U.S. sites
- 6
- U.S. states
- 6
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UC Davis | Sacramento | California | 95817 | — |
| University of Florida Health | Gainesville | Florida | — | — |
| Lurie Children's Hospital | Chicago | Illinois | — | — |
| Washington University | St Louis | Missouri | — | — |
| Duke University Medical Center | Durham | North Carolina | — | — |
| Children's Hospital of Richmond at VCU | Richmond | Virginia | — | — |
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 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02740972, 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 Dec 7, 2021 · Synced Oct 3, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02740972 live on ClinicalTrials.gov.