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

NCT02740972 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2021-12-07

Study results available
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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.

Conditions

Interventions

DRUG

NS-065/NCNP-01

DRUG

Placebo

Sponsors & Collaborators

  • Nippon Shinyaku Co., Ltd.

    collaborator INDUSTRY

  • Cooperative International Neuromuscular Research Group

    collaborator NETWORK

  • Therapeutic Research in Neuromuscular Disorders Solutions

    collaborator INDUSTRY

  • NS Pharma, Inc.

    lead INDUSTRY

Principal Investigators

  • Paula R. Clemens, MD · University of Pittsburgh

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
9 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-12-31
Primary Completion
2018-03-31
Completion
2018-04-30
FDA Drug
Yes

Countries

  • United States
  • Canada

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02740972 on ClinicalTrials.gov