NCT04708314

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

An Open-Label Study to Evaluate the Safety of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

• Status: Terminated
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Rare Disease Research, LLC · Academic / Other
• Sex: Male
• Age: 7 Years
• Healthy volunteers: Not accepted

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

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]	Vyondys 53

Timeline

• Start date: 2020-10-31
• Primary completion: 2021-05-13
• Completion: 2021-05-13
• First posted: 2021-01-13
• Last updated: 2026-04-03

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study