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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04060199
Ethics application status
Date submitted
15/08/2019
Date registered
19/08/2019

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
Timepoint [1] 0 0
baseline to 48 weeks of treatment
Secondary outcome [1] 0 0
TTRW
Timepoint [1] 0 0
baseline to 48 weeks of treatment
Secondary outcome [2] 0 0
6MWT
Timepoint [2] 0 0
baseline to 48 weeks of treatment
Secondary outcome [3] 0 0
NSAA
Timepoint [3] 0 0
baseline to 48 weeks of treatment
Secondary outcome [4] 0 0
TTCLIMB
Timepoint [4] 0 0
baseline to 48 weeks of treatment
Secondary outcome [5] 0 0
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
Sex
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/04/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
19/10/2023
Sample size
Target
Accrual to date
Final
77
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [2] 0 0
Perth Children's Hospital - Nedlands
Recruitment hospital [3] 0 0
The Childrens Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
- Brisbane
Recruitment postcode(s) [2] 0 0
- Nedlands
Recruitment postcode(s) [3] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
Canada
State/province [3] 0 0
Ontario
Country [4] 0 0
Canada
State/province [4] 0 0
Calgary
Country [5] 0 0
Canada
State/province [5] 0 0
Quebec City
Country [6] 0 0
Chile
State/province [6] 0 0
Santiago
Country [7] 0 0
China
State/province [7] 0 0
Beijing
Country [8] 0 0
China
State/province [8] 0 0
Changsha
Country [9] 0 0
China
State/province [9] 0 0
Shanghai
Country [10] 0 0
China
State/province [10] 0 0
Shenzhen
Country [11] 0 0
Greece
State/province [11] 0 0
Athens
Country [12] 0 0
Greece
State/province [12] 0 0
Thessaloníki
Country [13] 0 0
Hong Kong
State/province [13] 0 0
Kowloon Bay
Country [14] 0 0
Italy
State/province [14] 0 0
Rome
Country [15] 0 0
Korea, Republic of
State/province [15] 0 0
Pusan
Country [16] 0 0
Korea, Republic of
State/province [16] 0 0
Seoul
Country [17] 0 0
Mexico
State/province [17] 0 0
Chihuahua
Country [18] 0 0
Mexico
State/province [18] 0 0
Ciudad de mexico
Country [19] 0 0
Netherlands
State/province [19] 0 0
Gelderland
Country [20] 0 0
Netherlands
State/province [20] 0 0
Leiden
Country [21] 0 0
New Zealand
State/province [21] 0 0
Auckland
Country [22] 0 0
Norway
State/province [22] 0 0
Oslo
Country [23] 0 0
Russian Federation
State/province [23] 0 0
Moscow
Country [24] 0 0
Russian Federation
State/province [24] 0 0
Saint Petersburg
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Tomsk
Country [26] 0 0
Spain
State/province [26] 0 0
Barcelona
Country [27] 0 0
Spain
State/province [27] 0 0
Madrid
Country [28] 0 0
Taiwan
State/province [28] 0 0
Kaohsiung
Country [29] 0 0
Taiwan
State/province [29] 0 0
Taipei
Country [30] 0 0
Turkey
State/province [30] 0 0
Istanbul
Country [31] 0 0
Ukraine
State/province [31] 0 0
Kyiv
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Birmingham
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Glasgow
Country [34] 0 0
United Kingdom
State/province [34] 0 0
London
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Manchester

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
Trial website
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04060199