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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04768062




Registration number
NCT04768062
Ethics application status
Date submitted
19/02/2021
Date registered
24/02/2021
Date last updated
14/02/2024

Titles & IDs
Public title
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
Scientific title
A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
Secondary ID [1] 0 0
NS-065/NCNP-01-302
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

Experimental: Viltolarsen - Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks.


Treatment: Drugs: Viltolarsen
Received during weekly intravenous infusions
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03
Timepoint [1] 0 0
baseline to up to 96 weeks of treatment
Secondary outcome [1] 0 0
Time to Stand Test (TTSTAND)
Timepoint [1] 0 0
baseline to 96 weeks of treatment
Secondary outcome [2] 0 0
Time to Run/Walk 10 Meters Test (TTRW)
Timepoint [2] 0 0
baseline to 96 weeks of treatment
Secondary outcome [3] 0 0
Six-minute Walk Test (6MWT)
Timepoint [3] 0 0
baseline to 96 weeks of treatment
Secondary outcome [4] 0 0
North Star Ambulatory Assessment (NSAA)
Timepoint [4] 0 0
baseline to 96 weeks of treatment
Secondary outcome [5] 0 0
Time to Climb 4 Stairs Test (TTCLIMB)
Timepoint [5] 0 0
baseline to 96 weeks of treatment
Secondary outcome [6] 0 0
Muscle Strength Measured by Hand-Held Dynamometer
Timepoint [6] 0 0
baseline to 96 weeks of treatment

Eligibility
Key inclusion criteria
1. Patient has completed the NS-065/NCNP-01-301 study;

2. 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 will be asked to give
written or verbal assent according to local requirements;

3. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled
visits, investigational product (IP) administration plan, and study procedures.
Minimum age
No limit
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Patient had an adverse event in Study NS-065/NCNP-01-301 that, in the opinion of the
investigator and/or the sponsor, precludes safe use of viltolarsen for the patient in
this study;

2. Patient had a treatment which was made for the purpose of dystrophin or
dystrophin-related protein induction after completion of Study NS-065/NCNP-01-301;

3. Patient took any other investigational drug(s) during or after completion of Study
NS-065/NCNP-01-301;

4. Patient is judged by the investigator and/or the sponsor not to be appropriate to
participate in the extension study for any reason.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/04/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2025
Actual
Sample size
Target
Accrual to date
Final
74
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [2] 0 0
The Childrens Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
- Brisbane
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Quebec City
Country [2] 0 0
Chile
State/province [2] 0 0
Santiago
Country [3] 0 0
China
State/province [3] 0 0
Beijing
Country [4] 0 0
China
State/province [4] 0 0
Changsha
Country [5] 0 0
China
State/province [5] 0 0
Shanghai
Country [6] 0 0
China
State/province [6] 0 0
Shenzhen
Country [7] 0 0
Czechia
State/province [7] 0 0
Nový Hradec Králové
Country [8] 0 0
Greece
State/province [8] 0 0
Athens
Country [9] 0 0
Greece
State/province [9] 0 0
Thessaloníki
Country [10] 0 0
Italy
State/province [10] 0 0
Rome
Country [11] 0 0
Japan
State/province [11] 0 0
Tokyo
Country [12] 0 0
Korea, Republic of
State/province [12] 0 0
Pusan
Country [13] 0 0
Korea, Republic of
State/province [13] 0 0
Seoul
Country [14] 0 0
Mexico
State/province [14] 0 0
Ciudad de mexico
Country [15] 0 0
Netherlands
State/province [15] 0 0
Leiden
Country [16] 0 0
Netherlands
State/province [16] 0 0
Nijmegen
Country [17] 0 0
New Zealand
State/province [17] 0 0
Auckland
Country [18] 0 0
Norway
State/province [18] 0 0
Oslo
Country [19] 0 0
Russian Federation
State/province [19] 0 0
Moscow
Country [20] 0 0
Russian Federation
State/province [20] 0 0
Saint Petersburg
Country [21] 0 0
Russian Federation
State/province [21] 0 0
Tomsk
Country [22] 0 0
Spain
State/province [22] 0 0
Barcelona
Country [23] 0 0
Spain
State/province [23] 0 0
Madrid
Country [24] 0 0
Turkey
State/province [24] 0 0
Istanbul
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Birmingham
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Glasgow
Country [27] 0 0
United Kingdom
State/province [27] 0 0
London

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
This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who
have completed the 48-week treatment period of either viltolarsen or placebo in Study
NS-065/NCNP-01-301.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04768062
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