Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04687020|
Recruitment Status : Enrolling by invitation
First Posted : December 29, 2020
Last Update Posted : November 22, 2021
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Viltolarsen||Phase 4|
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 (Clinical Trial)|
|Estimated Enrollment :||16 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)|
|Actual Study Start Date :||June 10, 2021|
|Estimated Primary Completion Date :||November 2031|
|Estimated Study Completion Date :||November 2031|
Non-interventional (United States) / Low-interventional (Canada) : Viltolarsen
Patients will receive viltolarsen (recommended dose 80mg/kg/week) during a treatment period up to 120 months.
Received during weekly intravenous infusions
- 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) [ Time Frame: baseline to up to 120 months of treatment ]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.
- Loss of Ambulation (LOA) [ Time Frame: baseline to up to 120 months of treatment ]Loss of Ambulation (LOA) is defined by the inability to complete the Time to Run/Walk 10 meters (TTRW) in less than 30 seconds.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04687020
|United States, Illinois|
|Lurie Children's Hospital|
|Chicago, Illinois, United States, 60611|
|United States, Virginia|
|Children's Hospital of Richmond at VCU|
|Richmond, Virginia, United States, 23230|
|Alberta Children's Hospital|
|Calgary, Alberta, Canada, T3B 6A8|