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A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

NCT04179409 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2023-10-26

Study results available
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Summary

This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12.

Conditions

Interventions

DRUG

Amondys 45

This drug is used to target skipping of exon 45 of the dystrophin gene.

DRUG

Exondys 51

This drug is used to target skipping of exon 51 of the dystrophin gene.

DRUG

Vyondys 53

This drug is used to target skipping of exon 53 of the dystrophin gene.

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    collaborator INDUSTRY

  • Kevin Flanigan

    lead OTHER

Principal Investigators

  • Kevin Flanigan, MD · Nationwide Children's Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
6 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-02-18
Primary Completion
2021-09-01
Completion
2023-09-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

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 NCT04179409 on ClinicalTrials.gov