The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.
The VILT-502 study is an open-label, single-arm study to assess the long-term safety and effectiveness of viltolarsen, an exon skipping therapy for the treatment of DMD. Patients who complete the Phase II long-term extension study and meet the additional inclusion and exclusion criteria of the present protocol will be invited to enroll. Viltolarsen will be administered through weekly IV infusions, at the study site or at home. The VILT-502 study will be conducted as a non-interventional study in the US, and as a low-intervention clinical trial in Canada where viltolarsen is not yet commercially available, owing to differences in the stage of regulatory approval in the two countries.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Received during weekly intravenous infusions
UC Davis
Sacramento, California, United States
Lurie Children's Hospital
Chicago, Illinois, United States
Duke University Medical Center
Durham, North Carolina, United States
Children's Hospital of Richmond at VCU
Richmond, Virginia, United States
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0
Time frame: baseline to up to 120 months of treatment
Change in Time to Stand (TTSTAND)
Time frame: baseline to up to 120 months of treatment
Change in Time to Run/Walk 10 meters (TTRW)
Time frame: baseline to up to 120 months of treatment
Change in Performance of Upper Limb (PUL)
The Performance of the Upper Limb (PUL) scale is a specifically designed for assessing upper limb function in ambulant and non-ambulant patients with DMD. It consists of 22 items subdivided into shoulder level (6 items), mid-level (9 items) and distal level (7 items) dimension. The item ranges from score 0 -no useful hand function -to score 6 full shoulder abduction.
Time frame: baseline to up to 120 months of treatment
Loss of Ambulation (LOA)
Loss of Ambulation (LOA) is defined by the inability to complete the Time to Run/Walk 10 meters (TTRW) in less than 30 seconds.
Time frame: baseline to up to 120 months of treatment
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Alberta Children's Hospital
Calgary, Alberta, Canada