A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)

• 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
• Affiliation: Medpace

• Name: Kyle Haas
• 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)

Timepoint(s) of evaluation of this end point: At 96 weeks treatment

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

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)

127474

NS-065/NCNP-01-302

2021-000122-10-NL

Source(s) of Monetary Support

Nippon Shinyaku Co., Ltd

NS Pharma, Inc.

Ethics review

Status: Approved
Approval date: 05/09/2022
Contact: