An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
Status:
Terminated
Trial end date:
2021-05-13
Target enrollment:
Participant gender:
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.