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Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

NCT04060199 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 77

Last updated 2024-12-11

Study results available
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Summary

The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.

Conditions

Interventions

DRUG

Viltolarsen

IV infusion

DRUG

Placebo

IV infusion

Sponsors & Collaborators

  • Nippon Shinyaku Co., Ltd.

    collaborator INDUSTRY

  • NS Pharma, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2020-04-14
Primary Completion
2023-10-19
Completion
2023-10-19
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • Chile
  • China
  • Greece
  • Hong Kong
  • Italy
  • Mexico
  • Netherlands
  • New Zealand
  • Norway
  • Russia
  • South Korea
  • Spain
  • Taiwan
  • Turkey (Türkiye)
  • Ukraine
  • United Kingdom

Study Locations

More Related Trials

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 NCT04060199 on ClinicalTrials.gov