This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)

• Register: EUCTR
• Last refreshed on: 30 January 2023
• Main ID: EUCTR2019-002076-13-GR
• Date of registration: 24/09/2020
• Prospective Registration: Yes
• Primary sponsor: NS Pharma, Inc.
• Public title: This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
• Scientific title: A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53
• Date of first enrolment: 12/11/2020
• Target sample size: 74
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-002076-13
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Canada, Chile, China, Greece, Hong Kong, Italy, Japan, Korea, Republic of, Mexico, Netherlands, New Zealand, Norway, Russian Federation, Spain, Sweden, Taiwan, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: Kyle Haas
• Affiliation: Medpace

• Name: Kyle Haas
• Affiliation: Medpace

Key inclusion & exclusion criteria

Inclusion criteria:
1. Participant’s parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act
HIPAA) authorization, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to local requirements;
2. Participant has a confirmed diagnosis of DMD defined as:
a. Participant is male with clinical signs compatible with DMD; and
b. Participant has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame including determination of unambiguously defined exon boundaries (using techniques such as Multiplex ligation-dependent Probe Amplification [MLPA], comparative genomic hybridization [CGH] array or other techniques with similar capability);
3. Participant is = 4 years and <8 years of age at time of first infusion in the study;
4. Participant is able to walk independently without assistive devices;
5. Participant is able to complete the TTSTAND without assistance in <10 seconds, as assessed at the Screening Visit and the Pre-infusion Visit;
6. Participant and parent(s)/guardian(s) are willing and able to comply with scheduled visits, study drug administration plan, and study procedures;
7. Participant must be on a stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and is expected to remain on stable dose of GC treatment for the duration of the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 74
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Participant has current or history of chronic systemic fungal or viral infections;
2. Participant has had an acute illness within 4 weeks prior to the first dose of study drug based on the Principal Investigator’s judgment/discretion;
3. Participant has evidence of symptomatic cardiomyopathy (Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary);
4. Participant has an allergy or hypersensitivity to the study drug or to any of its constituents;
5. Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator;
6. Participant has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect 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;
7. Participant has had surgery within the 3 months prior to the first anticipated administration of study drug or surgery is planned for anytime during the duration of the study;
8. Participant has positive test results for hepatitis B antigen, hepatitis C antibody or human immunodeficiency virus (HIV) antibody at screening (Note: A positive hepatitis C antibody result is acceptable if accompanied by a negative hepatitis C RNA test and normal bilirubin and gamma glutamyl transferase results.);
9. Participant is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the halflife of a medication, whichever is longer;
10. Participant was previously enrolled in an interventional study of viltolarsen.
11. Participant 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 study drug.
12. Participant has taken any gene therapy.
13. Participant is currently taking idebenone, anabolic steroids (e.g., oxendolone), or products containing resveratrol or adenosine triphosphate, or has taken such within 3 months prior to first dose of study drug. Coenzyme Q10 or creatine are permitted only if the participant is receiving a stable dose for at least 3 months prior to the first dose of study drug and for the duration of the study;
14. There is no exclusion criterion #14. This criterion was removed from the protocol with Amendment 4 (version 3.0, dated 08 January 2021); however, the numbering was maintained to avoid documentation errors;
15. Participant has hydronephrosis, hydroureter, renal or urinary tract calculi, or ureteral stenosis by medical history or renal ultrasound.





Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Intervention(s)

Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: VILTOLARSEN
CAS Number: 2055732-84-6
Current Sponsor code: NS-065/NCNP-01
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

Main Objective: To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in
ambulant boys ages 4 to <8 years with DMD using the Time to Stand Test (TTSTAND) as a measure of strength and function.

Primary end point(s): TTSTAND

Secondary Objective: -To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg in ambulant boys ages 4 to <8 years with DMD over a 48-week treatment period vs. placebo controls using hierarchical strength and endurance outcomes
-To evaluate the safety and tolerability of viltolarsen administered intravenously at weekly doses of 80 mg/kg in ambulant boys ages 4 to <8 years with DMD

Timepoint(s) of evaluation of this end point: At 48 weeks treatment

Secondary Outcome(s)

Secondary end point(s): -Hierarchical analysis of the following strength and endurance measures:
- Time to Run/Walk 10 Meters Test (TTRW)
- Six-minute Walk Test (6MWT)
- North Star Ambulatory Assessment (NSAA)
- Time to Climb 4 Stairs Test (TTCLIMB)
- Quantitative muscle strength measured by hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only)

Timepoint(s) of evaluation of this end point: At 48 weeks treatment (first 5 bullets)

At each study visit (last 10 bullets)

Secondary ID(s)

2019-002076-13-GB

127474

NS-065/NCNP-01-301

Source(s) of Monetary Support

Nippon Shinyaku Co., Ltd

NS Pharma, Inc.

Ethics review

Status: Approved
Approval date: 16/10/2020
Contact: