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

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ClinicalTrials.gov Identifier: NCT04687020
Recruitment Status : Recruiting
First Posted : December 29, 2020
Last Update Posted : June 30, 2021
Sponsor:
Information provided by (Responsible Party):
NS Pharma, Inc.

Tracking Information
First Submitted Date  ICMJE December 16, 2020
First Posted Date  ICMJE December 29, 2020
Last Update Posted Date June 30, 2021
Actual Study Start Date  ICMJE June 10, 2021
Estimated Primary Completion Date November 2031   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 23, 2021)
  • 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.
Original Primary Outcome Measures  ICMJE
 (submitted: December 23, 2020)
  • Number of participants with treatment related Adverse Events as assessed by CTCAE v5.0 [ 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.
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
Official Title  ICMJE Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: Viltolarsen
Received during weekly intravenous infusions
Study Arms  ICMJE 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.
Intervention: Drug: Viltolarsen
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 23, 2020)
16
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2031
Estimated Primary Completion Date November 2031   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Trial info trialinfo@nspharma.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04687020
Other Study ID Numbers  ICMJE VILT-502
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party NS Pharma, Inc.
Study Sponsor  ICMJE NS Pharma, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account NS Pharma, Inc.
Verification Date June 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP