Duchenne Muscular DystrophyPhase 2NCT04956289

Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

A Phase 2 study testing Viltolarsen in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 8 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years. Read more about this study

Who is it for?: Ages 8 years and older, 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: 20 participants
Where: 8 sitesin 6 countries
Study dates: 1 Jul 2021to 13 Jul 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 8 years and older
Men only
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Patient (if age 18 years or older) or patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients younger than age 18 years will be asked to give written or verbal assent according to local requirements;
Patient has a confirmed diagnosis of DMD defined as:
Patient is male with clinical signs compatible with DMD; and
Patient has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin messenger ribonucleic acid reading frame including determination of unambiguously defined exon boundaries (using techniques such as multiplex ligation-dependent probe amplification, comparative genomic hybridization array, or other techniques with similar capability);
Patient is more than 8 years of age at time of first infusion in the study;
Patient has a Brooke scale rating of 3 or better OR an upright FVC 30% or greater at Screening;

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Patient, if sexually active, is willing to abstain from sexual intercourse or employ a barrier or medical method of contraception during and for 3 months following completion of IP administration;
Patient and patient's parent(s)/guardian(s) (if patient is <18 years of age) and/or caregiver(s) are willing and able to comply with scheduled visits, IP administration plan, and study procedures;
Patient must be on a stable dose of glucocorticoid (GC) or not treated with GC for at least 3 months prior to the first dose of IP and is expected to remain on stable dose of GC treatment or off GC for the duration of the study.
Other inclusion criteria may apply

Who cannot join

Patient has had an acute illness within 4 weeks prior to the first dose of IP;
Patient has evidence of symptomatic cardiomyopathy (New York Heart Association Class III or higher);
Patient requires ventilation support while awake during the day;
Patient has an allergy or hypersensitivity to IP or 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 a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormalities that could affect patient safety, make it unlikely that treatment and follow-up will be correctly completed, or impair the assessment of study results, in the opinion of the investigator;

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Patient has had surgery within 3 months prior to the first anticipated administration of IP or has known plans to have surgery during the Treatment Period;
Patient has positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus antibody at Screening;
Patient has been diagnosed with asthma that requires chronic treatment with a long-acting beta agonist;
Patient has relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana products by smoking or vaping within 3 months prior to treatment with IP;
Patient is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of IP or within 5 times the half-life of a medication, whichever is longer;
Patient has taken any gene therapy;
Patient is currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of IP;
Patient has hydronephrosis, hydroureter, renal or urinary tract calculi, or ureteral stenosis by renal ultrasound;
Patient was previously enrolled in an interventional study of viltolarsen.
Other exclusion criteria may apply

Trial locations and contacts

8 sites in 6 countries.

Country

City, postcode or hospital

8 sites in 6 countries

China2 sites

The Third Medical Center of PLA General Hospital
Beijing, 1000053
Hunan Children's Hospital
Changsha, 410021

Other trials in China

Russia2 sites

Russian National Research Medical University
Moscow, 117997
Saint Petersburg State Paediatric Medical University
Saint Petersburg, 194100

Other trials in Russia

United States1 site

Children's Hospital of Richmond at VCU
Richmond, Virginia, 23219

Other trials in United States

Italy1 site

Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Roma, 00168

Other trials in Italy

Spain1 site

Hospital Sant Joan de Deu
Barcelona, 08950

Other trials in Spain

Turkey (Türkiye)1 site

Yeditepe University Kosuyolu Hospital
Istanbul, 34718

Other trials in Turkey (Türkiye)

About this study

Official title: A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls

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
Also called NS-065/NCNP-01
Received during weekly intravenous infusions

Study groups in NCT04956289
Study group	Role	What they receive
Viltolarsen	Experimental	Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks. 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 Emergent Adverse Events
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment
Number of Participants With Treatment Emergent Adverse Events by Maximum Severity
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment
Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment
Number of Participants With Treatment Emergent Adverse Events by Worst Outcome
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Maximum Severity
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Worst Outcome
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment
Number of Participants With Adverse Event of Special Interest
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Measured: baseline to up to 48 weeks of treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Jul 202113 Jul 2023

First posted9 Jul 2021
Study start1 Jul 2021
Primary completion20 Jun 2023
Study completion13 Jul 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 20 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04956289
Sponsor's protocol ID: NS-065/NCNP-01-211
Status last verified: Jun 2024
First submitted: 30 Jun 2021
Last updated: 20 Aug 2024
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy

All Duchenne Muscular Dystrophy trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls
ClinicalTrials.gov ID: NCT04956289
Acronym / sponsor ID: NS-065/NCNP-01-211
Conditions: Duchenne Muscular Dystrophy
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 20 participants (actual)
Lead sponsor: NS Pharma, Inc.Industry (pharma, biotech, devices)
Collaborators: Nippon Shinyaku Co., Ltd.
Study start: 1 Jul 2021 (actual)
Primary completion: 20 Jun 2023 (actual)
Study completion: 13 Jul 2023 (actual)
First posted: 9 Jul 2021 (actual)
Results: Posted 20 Aug 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Viltolarsen	Experimental	Drug: ViltolarsenPatients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.

Who may qualify

Ages: 8 years and older
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (10 listed)

Patient (if age 18 years or older) or patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients younger than age 18 years will be asked to give written or verbal assent according to local requirements;
Patient has a confirmed diagnosis of DMD defined as:
Patient is more than 8 years of age at time of first infusion in the study;
Patient has a Brooke scale rating of 3 or better OR an upright FVC 30% or greater at Screening;
Patient, if sexually active, is willing to abstain from sexual intercourse or employ a barrier or medical method of contraception during and for 3 months following completion of IP administration;

Key exclusion criteria (16 listed)

Patient has had an acute illness within 4 weeks prior to the first dose of IP;
Patient has evidence of symptomatic cardiomyopathy (New York Heart Association Class III or higher);
Patient requires ventilation support while awake during the day;
Patient has an allergy or hypersensitivity to IP or any of its constituents;
Patient has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator;

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
ViltolarsenAlso: NS-065/NCNP-01Received during weekly intravenous infusions	Drug	Viltolarsen

Primary outcome

Number of Participants With Treatment Emergent Adverse Events
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Number of Participants With Treatment Emergent Adverse Events by Maximum Severity
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Number of Participants With Treatment Emergent Adverse Events by Worst Outcome
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Maximum Severity
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Worst Outcome
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Number of Participants With Adverse Event of Special Interest
Time frame: baseline to up to 48 weeks of treatment
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Results and safety

Results posted to the registry on 20 Aug 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Viltolarsen 80mg/kg	20	20

Primary outcome: Number of Participants With Treatment Emergent Adverse Events

Time frame: baseline to up to 48 weeks of treatment. Count of participants. Unit: Participants

Measure	Viltolarsen 80mg/kg n = 20
Value	19

Population analysed: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10).

Primary outcome: Number of Participants With Treatment Emergent Adverse Events by Maximum Severity

Time frame: baseline to up to 48 weeks of treatment. Count of participants. Unit: Participants

Measure	Viltolarsen 80mg/kg n = 19
Mild	7
Moderate	12
Severe	0

Primary outcome: Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product

Time frame: baseline to up to 48 weeks of treatment. Count of participants. Unit: Participants

Measure	Viltolarsen 80mg/kg n = 19
Dose not changed	16
Dose reduced	0
Drug interrupted	2
Drug withdrawn	0
Not applicable	1

Primary outcome: Number of Participants With Treatment Emergent Adverse Events by Worst Outcome

Time frame: baseline to up to 48 weeks of treatment. Count of participants. Unit: Participants

Measure	Viltolarsen 80mg/kg n = 19
Recovered or resolved	17
Recovered or resolved with sequelae	0
Recovering or resolving	0
Not recovered or resolved	2
Fatal	0

Adverse events

Time frame: Up to 48 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Viltolarsen 80mg/kg	0 of 20 (0%)	19 of 20 (95%)	0 of 20

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Duchenne Muscular Dystrophy

RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
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Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy

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.

8 sites in 6 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Viltolarsen in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 8 sites in 6 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 10 inclusion and 16 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: finished, as of the sponsor's last update on 20 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in China, Russia, United States, Italy, Spain and 1 more country. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 Emergent Adverse Events (time frame: baseline to up to 48 weeks of treatment). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04956289.

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 20 Aug 2024.