The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
171
Casimersen solution for IV infusion
Golodirsen solution for IV infusion
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
Time frame: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Neuromuscular Research Center
Phoenix, Arizona, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of California Los Angeles
Los Angeles, California, United States
Stanford Neuroscience Health Center
Palo Alto, California, United States
Rady Children's Hospital- San Diego
San Diego, California, United States
UF Health: University of Florida Clinical Research Center
Gainesville, Florida, United States
NW Florida Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States
University of Iowa Childrens Hospital
Iowa City, Iowa, United States
...and 40 more locations