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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.

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Viltolarsen Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
Actual Study Start Date : June 10, 2021
Estimated Primary Completion Date : November 2031
Estimated Study Completion Date : November 2031


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0 [ Time Frame: baseline to up to 120 months of treatment ]
  2. Change in Time to Stand (TTSTAND) [ Time Frame: baseline to up to 120 months of treatment ]
  3. Change in Time to Run/Walk 10 meters (TTRW) [ Time Frame: baseline to up to 120 months of treatment ]
  4. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04687020


Contacts
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Contact: Trial info trialinfo@nspharma.com

Locations
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United States, Virginia
Children's Hospital of Richmond at VCU Recruiting
Richmond, Virginia, United States, 23230
Sponsors and Collaborators
NS Pharma, Inc.
Additional Information:
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Responsible Party: NS Pharma, Inc.
ClinicalTrials.gov Identifier: NCT04687020    
Other Study ID Numbers: VILT-502
First Posted: December 29, 2020    Key Record Dates
Last Update Posted: June 30, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked