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An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

NCT04708314 · Status: TERMINATED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2026-04-03

No results posted yet for this study

Summary

This is an open-label study to evaluate the safety and tolerability of golodirsen injection in Non-ambulant DMD patients with confirmed genetic mutations amenable to treatment by exon 53 skipping (Golodirsen).

Golodirsen 30 mg/kg will be administered as an intravenous (IV) infusion over approximately 35 to 60 minutes once a week during the treatment period (up to 96 weeks). After the treatment period, patients can go into a safety extension period (not to exceed 48 weeks) until the patient is able to transition to commercially available drug or a separate golodirsen study.

Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory assessments, including pulmonary function tests (PFTs), upper extremity testing, and other measurements of functional status, will occur at functional assessment visits every 12 weeks over the first year of treatment and approximately every 24 weeks over the second year of treatment.

Conditions

Interventions

DRUG

Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]

Vyondys 53

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    collaborator INDUSTRY

  • Rare Disease Research, LLC

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
7 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-10-31
Primary Completion
2021-05-13
Completion
2021-05-13
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 NCT04708314 on ClinicalTrials.gov