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

October 18, 2022 updated by: NS Pharma, Inc.
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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

9

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • Alberta Children's Hospital
  • United States
    • California
      • Sacramento, California, United States, 95817
        • UC Davis
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Lurie Children's Hospital
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Virginia
      • Richmond, Virginia, United States, 23230
        • Children's Hospital of Richmond at VCU

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: 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.
Drug: Viltolarsen
Received during weekly intravenous infusions

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0
Time Frame: baseline to up to 120 months of treatment
baseline to up to 120 months of treatment
Change in Time to Stand (TTSTAND)
Time Frame: baseline to up to 120 months of treatment
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
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.
baseline to up to 120 months of treatment
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.
baseline to up to 120 months of treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

NS Pharma, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 10, 2021

Primary Completion (Anticipated)

September 1, 2032

Study Completion (Anticipated)

October 1, 2032

Study Registration Dates

First Submitted

December 16, 2020

First Submitted That Met QC Criteria

December 23, 2020

First Posted (Actual)

December 29, 2020

Study Record Updates

Last Update Posted (Actual)

October 19, 2022

Last Update Submitted That Met QC Criteria

October 18, 2022

Last Verified

October 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VILT-502

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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