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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 21 May 2024
Main ID:  EUCTR2021-000122-10-NO
Date of registration: 24/02/2022
Prospective Registration: Yes
Primary sponsor: NS Pharma, Inc.
Public title: A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)
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) - RACER-53X
Date of first enrolment: 05/09/2022
Target sample size: 74
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2021-000122-10
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no  
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 Czech Republic Greece Hong Kong Italy
Japan Korea, Republic of Mexico Netherlands New Zealand Norway Russian Federation Spain
Taiwan Turkey Ukraine United Kingdom United States
Contacts
Name: Kyle Haas   
Address:  Medpace Way 5375 Cincinnati United States
Telephone: +151357999111270
Email: regsubmissions@medpace.com
Affiliation:  Medpace
Name: Kyle Haas   
Address:  Medpace Way 5375 Cincinnati United States
Telephone: +151357999111270
Email: regsubmissions@medpace.com
Affiliation:  Medpace
Key inclusion & exclusion criteria
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.
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. 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.


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-

Primary Outcome(s)
Primary end point(s): - Vital signs
- Physical examination
- Clinical laboratory tests
o Hematology and clinical chemistry
o Urinalysis
o Urine cytology
- Antibodies to dystrophin and viltolarsen
- 12-lead electrocardiogram (ECG)
- Renal ultrasound
- Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Main Objective: To evaluate the safety and tolerability of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg in boys who have completed the NS-065/NCNP-01-301 study
Secondary Objective: Secondary Objective:
To compare the efficacy of viltolarsen administered IV at weekly doses of 80 mg/kg in boys who have completed the NS-065/NCNP-01-301 study over a 96-week treatment period versus natural history controls using strength and endurance outcomes.

Exploratory Objectives:
- To evaluate health-related quality of life impact of viltolarsen treatment on patient's DMD
- To evaluate preservation of ambulation of patients with DMD
Timepoint(s) of evaluation of this end point: At 96 weeks treatment
Secondary Outcome(s)
Secondary end point(s): Secondary:
- Time to Stand Test (TTSTAND)
- 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)

Exploratory:
- Pediatric Outcome Data Collection Instrument (PODCI)
- Personal Adjustment and Role Skills Scale, 3rd edition (PARS III) Questionnaire
-Loss of ambulation
Timepoint(s) of evaluation of this end point: At 96 weeks treatment
Secondary ID(s)
2021-000122-10-NL
NS-065/NCNP-01-302
127474
Source(s) of Monetary Support
Nippon Shinyaku Co., Ltd
NS Pharma, Inc.
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 05/09/2022
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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