Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

Duchenne Muscular Dystrophy Clinical Trial

Official title:

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04687020
• Other study ID #: VILT-502
• Status: Active, not recruiting
• Phase: Phase 4
• Start date: June 10, 2021
• Est. completion date: October 2032

Study information

• Verified date: October 2022
• Source: NS Pharma, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 9
• Est. completion date: October 2032
• Est. primary completion date: September 2032
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Patient, patient's parent or legal guardian have provided written informed consent/medical record release authorization prior to any extension study-specific procedures, and the patient has provided assent appropriate for his age and developmental status.

2. Patient completed the NS-065/NCNP-01-202 study and was judged by the investigator as appropriate to participate in the VILT-502 study.

3. Patient and parent or legal guardian are willing and able to comply with scheduled visits, study treatment administration plan, and study procedures.

Exclusion Criteria:

1. Patient has an allergy or hypersensitivity to the study drug or to any of its constituents.

2. Patient has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator.

3. Patient has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and observation will be correctly completed or impair the assessment of study results, in the opinion of the investigator.

4. Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completing the NS-065/NCNP-01-202 study.

5. Patient took any other investigational drugs after completing the NS-065/NCNP-01-202 study.

6. Patient plans to participate in another clinical trial.

7. Patient was judged by the investigator and/or the Sponsor as not appropriate to participate in the study for reasons other than #1 - #6 above.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Viltolarsen
Received during weekly intravenous infusions

Locations

Country	Name	City	State
Canada	Alberta Children's Hospital	Calgary	Alberta
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	UC Davis	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
NS Pharma, Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0		baseline to up to 120 months of treatment
Primary	Change in Time to Stand (TTSTAND)		baseline to up to 120 months of treatment
Primary	Change in Time to Run/Walk 10 meters (TTRW)		baseline to up to 120 months of treatment
Primary	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.	baseline to up to 120 months of treatment
Primary	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.	baseline to up to 120 months of treatment

External Links

•   Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)