Duchenne Muscular DystrophyPhase 4NCT04687020
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
A Phase 4 study testing Viltolarsen in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 5 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Viltolarsen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 10 Jun 2021to Oct 2032
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
United States4 sites
Lurie Children's Hospital
Chicago, Illinois, 60611
Duke University Medical Center
Durham, North Carolina, 27710
Children's Hospital of Richmond at VCU
Richmond, Virginia, 23230
UC Davis
Sacramento, California, 95817
About this study
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.
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Viltolarsen
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ViltolarsenDrug
Received during weekly intravenous infusions
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Viltolarsen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0
Measured: baseline to up to 120 months of treatment
Change in Time to Stand (TTSTAND)
Measured: baseline to up to 120 months of treatment
Change in Time to Run/Walk 10 meters (TTRW)
Measured: 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.
Measured: 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.
Measured: baseline to up to 120 months of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2020
- Study start10 Jun 2021
- Primary completionSep 2032 (estimated)
- Study completionOct 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- NS Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04687020
- Sponsor's protocol ID
- VILT-502
- Status last verified
- Oct 2022
- First submitted
- 16 Dec 2020
- Last updated
- 19 Oct 2022
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
- ClinicalTrials.gov ID
- NCT04687020
- Acronym / sponsor ID
- VILT-502
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (actual)
- Lead sponsor
- NS Pharma, Inc.Industry (pharma, biotech, devices)
- Study start
- 10 Jun 2021 (actual)
- Primary completion
- Sep 2032 (estimated)
- Study completion
- Oct 2032 (estimated)
- First posted
- 29 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Non-interventional (United States) / Low-interventional (Canada) : Viltolarsen | Other | Drug: ViltolarsenPatients will receive viltolarsen (recommended dose 80mg/kg/week) during a treatment period up to 120 months. |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ViltolarsenReceived during weekly intravenous infusions | Drug | Non-interventional (United States) / Low-interventional (Canada) : Viltolarsen |
Primary outcome
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.
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2022.
- Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)Link provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Viltolarsen in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 5 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 19 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0 (time frame: baseline to up to 120 months of treatment). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2022.