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

NCT04956289 | Completed Phase 2 Results FDA Drug

• 1 other identifier: NS-065/NCNP-01-211
• Study Type: interventional
• Target: 20
• Locations: 6 countries
• Sites: 8

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (9)

• 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".

  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".

  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".

  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".

  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".

  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".

  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".

  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".

  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".

  baseline to up to 48 weeks of treatment

Study Arms (1)

Viltolarsen

EXPERIMENTAL

Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.

Drug: Viltolarsen

Interventions

Viltolarsen DRUG

Received during weekly intravenous infusions

Also known as: NS-065/NCNP-01

Viltolarsen

Eligibility Criteria

• Age: 8 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• 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.

You may not qualify if:

• 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;
• 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.

Sponsors & Collaborators

• NS Pharma, Inc.: lead
• Nippon Shinyaku Co., Ltd.: collaborator

Study Sites (8)

Children's Hospital of Richmond at VCU

Richmond, Virginia, 23219, United States

Location

The Third Medical Center of PLA General Hospital

Beijing, 1000053, China

Location

Hunan Children's Hospital

Changsha, 410021, China

Location

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

Roma, 00168, Italy

Location

Russian National Research Medical University

Moscow, 117997, Russia

Location

Saint Petersburg State Paediatric Medical University

Saint Petersburg, 194100, Russia

Location

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

Location

Yeditepe University Kosuyolu Hospital

Istanbul, 34718, Turkey (Türkiye)

Location

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

Results Point of Contact

• Title: Medical Affairs
• Organization: NS Pharma, Inc.

trialinfo@nspharma.com

+1(201) 986-3860

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2021
• First Posted: July 9, 2021
• Study Start: July 1, 2021
• Primary Completion: June 20, 2023
• Study Completion: July 13, 2023
• Last Updated: August 20, 2024
• Results First Posted: August 20, 2024

Record last verified: 2024-06

Locations

TU (1); ES (1); CN (2); IT (1); RU (2); US (1)