Back to results

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

Primary Purpose

Duchenne Muscular Dystrophy

Status

Active

Phase

Phase 4

Locations

International

Study Type

Interventional

Intervention

Viltolarsen

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

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.
Patient completed the NS-065/NCNP-01-202 study and was judged by the investigator as appropriate to participate in the VILT-502 study.
Patient and parent or legal guardian are willing and able to comply with scheduled visits, study treatment administration plan, and study procedures.

Exclusion Criteria:

Patient has an allergy or hypersensitivity to the study drug or to any of its constituents.
Patient has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator.
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.
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.
Patient took any other investigational drugs after completing the NS-065/NCNP-01-202 study.
Patient plans to participate in another clinical trial.
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.

Sites / Locations

UC Davis
Lurie Children's Hospital
Duke University Medical Center
Children's Hospital of Richmond at VCU
Alberta Children's Hospital

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Non-interventional (United States) / Low-interventional (Canada) : Viltolarsen

Arm Description

Patients will receive viltolarsen (recommended dose 80mg/kg/week) during a treatment period up to 120 months.

Outcomes

Primary Outcome Measures

Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0

Change in Time to Stand (TTSTAND)

Change in Time to Run/Walk 10 meters (TTRW)

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.

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT04687020

First Posted

December 16, 2020

Last Updated

October 18, 2022

Sponsor

NS Pharma, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04687020

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Active, not recruiting

Study Start Date

June 10, 2021 (Actual)

Primary Completion Date

September 2032 (Anticipated)

Study Completion Date

October 2032 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

NS Pharma, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. Study Description

Brief 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.

Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

9 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Non-interventional (United States) / Low-interventional (Canada) : Viltolarsen

Arm Type

Other

Arm Description

Patients will receive viltolarsen (recommended dose 80mg/kg/week) during a treatment period up to 120 months.

Intervention Type

Drug

Intervention Name(s)

Viltolarsen

Intervention Description

Received during weekly intravenous infusions

Primary Outcome Measure Information:

Title

Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0

Time Frame