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NCT02740972 · ClinicalTrials.gov registry record · Phase 2

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

A Phase 2 study, sponsored by NS Pharma.

Completed
Registry status
Phase 2
Development phase
16
Enrollment target

NCT02740972: Completed Phase 2 study, sponsored by NS Pharma.

NCT02740972 is a Phase 2 study that has completed, run by NS Pharma. The registered enrollment target is 16 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (88% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT02740972, a Phase 2 study, has completed, sponsored by NS Pharma.

COMPLETED
Registry status
Phase 2
Development phase
16 participants
Enrollment target

Study Summary

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.

Primary Outcome

Treatment emergent adverse events (TEAEs) were summarized for Period 1 by comparing low dose to high dose to placebo and for Period 2 between the low dose cohort and the high dose cohort. TEAEs were summarized both at the patient level for number of TEAEs, highest severity, relationship, action and outcome and at the TEAE level (summarizing events) by organ system and preferred term TEAE as well as severity, relationship, action and outcome. The Medical Dictionary for Regulatory Activities (Med

Interventions

  • DRUG Placebo
  • DRUG NS-065/NCNP-01

Trial Details

FieldValue
Enrollment Target 16 participants
Start Date 2016-12
Est. Completion 2018-04
Phase Phase 2
NS Pharma

11 total trials

What the finished NCT02740972 record still lists

NCT02740972 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (88% lower).

The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.

NCT02740972 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT02740972 about?

NCT02740972 is a clinical study titled "Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)". 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 a...

What is the current status of trial NCT02740972?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 16 participants. The study started on 2016-12. Estimated completion is 2018-04.

What interventions are being tested in trial NCT02740972?

The interventions under investigation include: Placebo (DRUG), NS-065/NCNP-01 (DRUG).

Who is sponsoring clinical trial NCT02740972?

This trial is sponsored by NS Pharma, which has 11 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT02740972's enrollment target sits among peer trials

16 1908th of 2000 higher than 82 of 2,000 other Phase 2 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT02740972, the US trial registry maintained by the National Library of Medicine. NCT02740972 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.