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

NCT04687020 | Active Not Recruiting Phase 4 FDA Drug

• 1 other identifier: VILT-502
• Study Type: interventional
• Target: 9
• Locations: 2 countries
• Sites: 5

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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (5)

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

  baseline to up to 120 months of treatment

• Change in Time to Stand (TTSTAND)

  baseline to up to 120 months of treatment

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

  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.

  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.

  baseline to up to 120 months of treatment

Study Arms (1)

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

OTHER

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

Drug: Viltolarsen

Interventions

Viltolarsen DRUG

Received during weekly intravenous infusions

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

Eligibility Criteria

• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• NS Pharma, Inc.: lead

Study Sites (5)

UC Davis

Sacramento, California, 95817, United States

Location

Lurie Children's Hospital

Chicago, Illinois, 60611, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Children's Hospital of Richmond at VCU

Richmond, Virginia, 23230, United States

Location

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

Location

Related Links

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

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

viltolarsen

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 16, 2020
• First Posted: December 29, 2020
• Study Start: June 10, 2021
• Primary Completion (Estimated): September 1, 2032
• Study Completion (Estimated): October 1, 2032
• Last Updated: October 19, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (4)