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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04060199




Registration number
NCT04060199
Ethics application status
Date submitted
15/08/2019
Date registered
19/08/2019
Date last updated
19/02/2020

Titles & IDs
Public title
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
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)
Secondary ID [1] 0 0
NS-065/NCNP-01-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Viltolarsen
Treatment: Drugs - Placebo

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

Placebo Comparator: Placebo - Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.


Treatment: Drugs: Viltolarsen
IV infusion

Treatment: Drugs: Placebo
IV infusion

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
TTSTAND - Change in Time to Stand (TTSTAND)
Timepoint [1] 0 0
baseline to 48 weeks of treatment
Secondary outcome [1] 0 0
TTRW - Change in Time to Run/Walk 10 Meters Test (TTRW)
Timepoint [1] 0 0
baseline to 48 weeks of treatment
Secondary outcome [2] 0 0
6MWT - Change in Six-minutes Walk Test (6MWT)
Timepoint [2] 0 0
baseline to 48 weeks of treatment
Secondary outcome [3] 0 0
NSAA - Change in North Star Ambulatory Assessment (NSAA)
The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
Timepoint [3] 0 0
baseline to 48 weeks of treatment
Secondary outcome [4] 0 0
TTCLIMB - Change in Time to Climb 4 Steps Test (TTCLIMB)
Timepoint [4] 0 0
baseline to 48 weeks of treatment
Secondary outcome [5] 0 0
Hand-held dynamometer - The force generated for each muscle strength (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only) will be measured by Hand-held dynamometer.
Timepoint [5] 0 0
baseline to 48 weeks of treatment

Eligibility
Key inclusion criteria
- Male = 4 years and < 8 years of age

- Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon
53 to restore the dystrophin mRNA reading frame

- Able to walk independently without assistive devices

- TTSTAND < 10 seconds

- 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

- Other inclusion criteria may apply
Minimum age
4 Years
Maximum age
7 Years
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
- Current or history of chronic systemic fungal or viral infections

- Acute illness within 4 weeks prior to the first dose of study drug

- Evidence of symptomatic cardiomyopathy (Note: Asymptomatic cardiac abnormality on
investigation would not be exclusionary)

- Allergy or hypersensitivity to the study drug or to any of its constituents

- Severe behavioral or cognitive problems that preclude participation in the study, in
the opinion of the investigator

- 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;

- Surgery within the 3 months prior to the first dose of study drug or surgery is
planned for anytime during the duration of the study

- Participant has positive test results for hepatitis B antigen, hepatitis C antibody or
human immunodeficiency virus (HIV)

- 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
half-life of a medication, whichever is longer

- Previously enrolled in an interventional study of viltolarsen

- 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

- Having taken any gene therapy

- Other exclusion criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Oregon
Country [5] 0 0
Canada
State/province [5] 0 0
Calgary
Country [6] 0 0
Italy
State/province [6] 0 0
Milano
Country [7] 0 0
Japan
State/province [7] 0 0
Hyogo
Country [8] 0 0
Japan
State/province [8] 0 0
Kumamoto
Country [9] 0 0
Japan
State/province [9] 0 0
Tokyo
Country [10] 0 0
Korea, Republic of
State/province [10] 0 0
Pusan
Country [11] 0 0
Korea, Republic of
State/province [11] 0 0
Seoul
Country [12] 0 0
New Zealand
State/province [12] 0 0
Auckland
Country [13] 0 0
Russian Federation
State/province [13] 0 0
Tomsk
Country [14] 0 0
Spain
State/province [14] 0 0
Barcelona
Country [15] 0 0
Sweden
State/province [15] 0 0
Göteborg
Country [16] 0 0
Taiwan
State/province [16] 0 0
Kaohsiung
Country [17] 0 0
Taiwan
State/province [17] 0 0
Taipei

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
NS Pharma, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Nippon Shinyaku Co., Ltd.
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to
placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
Trial website
https://clinicaltrials.gov/show/NCT04060199
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Info
Address 0 0
Country 0 0
Phone 0 0
201-986-3860
Fax 0 0
Email 0 0
trialinfo@nspharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04060199