Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

Sponsor

NS Pharma, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04060199

Collaborator

Nippon Shinyaku Co., Ltd. (Industry)

Enrollment

Locations

Arms

55.6

Anticipated Duration (Months)

1.9

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy	Drug: Viltolarsen Drug: Placebo	Phase 3

Detailed Description

This is 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. Eligible patients with out-of-frame deletion mutations amenable to exon 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 80 mg/kg Viltolarsen or placebo for up to 48 weeks.

The study will enroll approximately 74 patients amenable to exon 53 skipping. Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as 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 Steps Test (TTCLIMB) and Hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only).

Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.

Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of study drug.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

74 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official 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)

Actual Study Start Date :

Apr 14, 2020

Anticipated Primary Completion Date :

Nov 1, 2024

Anticipated Study Completion Date :

Dec 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Viltolarsen Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.	Drug: Viltolarsen IV infusion Other Names: NS-065/NCNP-01
Placebo Comparator: Placebo Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.	Drug: Placebo IV infusion

Arm

Intervention/Treatment

Experimental: Viltolarsen

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

Drug: Viltolarsen

IV infusion

Other Names:

NS-065/NCNP-01

Placebo Comparator: Placebo

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

Drug: Placebo

IV infusion

Outcome Measures

Primary Outcome Measures

TTSTAND [baseline to 48 weeks of treatment]
Change in Time to Stand (TTSTAND)

Secondary Outcome Measures

TTRW [baseline to 48 weeks of treatment]
Change in Time to Run/Walk 10 Meters Test (TTRW)
6MWT [baseline to 48 weeks of treatment]
Change in Six-minutes Walk Test (6MWT)
NSAA [baseline to 48 weeks of treatment]
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.
TTCLIMB [baseline to 48 weeks of treatment]
Change in Time to Climb 4 Steps Test (TTCLIMB)
Hand-held dynamometer [baseline to 48 weeks of treatment]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

4 Years to 7 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

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

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California Davis Medical Center	Sacramento	California	United States	95817
2	Ann and Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	United States	60611
3	Queensland Children's Hospital	Brisbane		Australia
4	Perth Children's Hospital	Nedlands		Australia
5	The Childrens Hospital at Westmead	Westmead		Australia
6	The Hospital for Sick Children (SickKids)	Toronto	Ontario	Canada
7	Alberta Children's Hospital	Calgary		Canada
8	CHU de Quebec Research Centre	Quebec City		Canada
9	Hospital de Niños Roberto del Rio	Santiago		Chile
10	Pontificia Universidad Católica de Chile	Santiago		Chile
11	Agia Sofia Children's Hospital	Athens		Greece
12	Hippokration General Hospital of Thessaloniki	Thessaloníki		Greece
13	Hong Kong Children's Hospital	Kowloon Bay		Hong Kong
14	Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore	Rome		Italy
15	Hyogo College of Medicine College Hospital	Hyōgo		Japan
16	Kumamoto University Hospital	Kumamoto		Japan
17	National Center of Neurology and Psychiatry	Tokyo		Japan
18	Pusan National University Yangsan Hospital	Pusan		Korea, Republic of
19	Seoul National University Hospital	Seoul		Korea, Republic of
20	Hospital Angeles Chihuahua	Chihuahua		Mexico
21	Instituto Nacional de Pediatria	Ciudad de mexico		Mexico
22	Radboud Universitair Medisch Centrum	Nijmegen	Gelderland	Netherlands
23	Leids Universitair Medisch Centrum	Leiden		Netherlands
24	New Zealand Clinical Research Ltd	Auckland		New Zealand
25	Christchurch Clinical Studies Trust	Christchurch		New Zealand
26	Rikshospitalet	Oslo		Norway
27	Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev	Moscow		Russian Federation
28	"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre	Saint Petersburg		Russian Federation
29	Tomsk National Research Medical Center of Russian Academy of Sciences	Tomsk		Russian Federation
30	Hospital Sant Joan de Deu	Barcelona		Spain
31	Hospital Universitario La Paz	Madrid		Spain
32	Kaohsiung Medical University Chung-Ho Memorial Hospital	Kaohsiung		Taiwan
33	National Taiwan University Hospital	Taipei		Taiwan
34	Yeditepe University Kosuyolu Hospital	Istanbul		Turkey
35	State Institution "Ukrainian Medical rehabilitation Center for Children with organic disorders of the nervous system of the Ministry of Health of Ukraine"	Kyiv		Ukraine
36	Birmingham Heartlands Hospital	Birmingham		United Kingdom
37	Royal Hospital for Children - Glasgow	Glasgow		United Kingdom
38	University College London Institute of Child Health	London		United Kingdom
39	Royal Manchester Children's Hospital	Manchester		United Kingdom

Sponsors and Collaborators

NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

NS Pharma, Inc.

ClinicalTrials.gov Identifier:

NCT04060199

Other Study ID Numbers:

NS-065/NCNP-01-301

First Posted:

Aug 19, 2019

Last Update Posted:

May 25, 2022

Last Verified:

Jun 1, 2021

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Muscular Dystrophies

Muscular Dystrophy, Duchenne

Study Results

No Results Posted as of May 25, 2022