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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Viltolarsen
Placebo
Sponsored by
NS Pharma, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy

Eligibility Criteria

4 Years - 7 Years (Child)MaleDoes not accept 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

Sites / Locations

  • University of California Davis Medical CenterRecruiting
  • Ann and Robert H. Lurie Children's Hospital of ChicagoRecruiting
  • Queensland Children's HospitalRecruiting
  • Perth Children's HospitalRecruiting
  • The Childrens Hospital at WestmeadRecruiting
  • The Hospital for Sick Children (SickKids)Recruiting
  • Alberta Children's HospitalRecruiting
  • CHU de Quebec Research CentreRecruiting
  • Hospital de Niños Roberto del RioRecruiting
  • Pontificia Universidad Católica de ChileRecruiting
  • Agia Sofia Children's HospitalRecruiting
  • Hippokration General Hospital of ThessalonikiRecruiting
  • Hong Kong Children's HospitalRecruiting
  • Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro CuoreRecruiting
  • Hyogo College of Medicine College Hospital
  • Kumamoto University Hospital
  • National Center of Neurology and Psychiatry
  • Pusan National University Yangsan HospitalRecruiting
  • Seoul National University HospitalRecruiting
  • Hospital Angeles ChihuahuaRecruiting
  • Instituto Nacional de PediatriaRecruiting
  • Radboud Universitair Medisch CentrumRecruiting
  • Leids Universitair Medisch CentrumRecruiting
  • New Zealand Clinical Research Ltd
  • Christchurch Clinical Studies Trust
  • RikshospitaletRecruiting
  • Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev
  • "Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
  • Tomsk National Research Medical Center of Russian Academy of Sciences
  • Hospital Sant Joan de DeuRecruiting
  • Hospital Universitario La PazRecruiting
  • Kaohsiung Medical University Chung-Ho Memorial HospitalRecruiting
  • National Taiwan University HospitalRecruiting
  • Yeditepe University Kosuyolu HospitalRecruiting
  • State Institution "Ukrainian Medical rehabilitation Center for Children with organic disorders of the nervous system of the Ministry of Health of Ukraine"Recruiting
  • Birmingham Heartlands HospitalRecruiting
  • Royal Hospital for Children - GlasgowRecruiting
  • University College London Institute of Child HealthRecruiting
  • Royal Manchester Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Viltolarsen

Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

TTSTAND
Change in Time to Stand (TTSTAND)

Secondary Outcome Measures

TTRW
Change in Time to Run/Walk 10 Meters Test (TTRW)
6MWT
Change in Six-minutes Walk Test (6MWT)
NSAA
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
Change in Time to Climb 4 Steps Test (TTCLIMB)
Hand-held dynamometer
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.

Full Information

First Posted
August 15, 2019
Last Updated
May 23, 2022
Sponsor
NS Pharma, Inc.
Collaborators
Nippon Shinyaku Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT04060199
Brief Title
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2021
Overall Recruitment Status
Recruiting
Study Start Date
April 14, 2020 (Actual)
Primary Completion Date
November 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NS Pharma, Inc.
Collaborators
Nippon Shinyaku Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
74 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Viltolarsen
Arm Type
Experimental
Arm Description
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Intervention Type
Drug
Intervention Name(s)
Viltolarsen
Other Intervention Name(s)
NS-065/NCNP-01
Intervention Description
IV infusion
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
IV infusion
Primary Outcome Measure Information:
Title
TTSTAND
Description
Change in Time to Stand (TTSTAND)
Time Frame
baseline to 48 weeks of treatment
Secondary Outcome Measure Information:
Title
TTRW
Description
Change in Time to Run/Walk 10 Meters Test (TTRW)
Time Frame
baseline to 48 weeks of treatment
Title
6MWT
Description
Change in Six-minutes Walk Test (6MWT)
Time Frame
baseline to 48 weeks of treatment
Title
NSAA
Description
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.
Time Frame
baseline to 48 weeks of treatment
Title
TTCLIMB
Description
Change in Time to Climb 4 Steps Test (TTCLIMB)
Time Frame
baseline to 48 weeks of treatment
Title
Hand-held dynamometer
Description
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.
Time Frame
baseline to 48 weeks of treatment

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Trial Info
Phone
1-866-677-4276
Email
trialinfo@nspharma.com
Facility Information:
Facility Name
University of California Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Individual Site Status
Recruiting
Facility Name
Ann and Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
Queensland Children's Hospital
City
Brisbane
Country
Australia
Individual Site Status
Recruiting
Facility Name
Perth Children's Hospital
City
Nedlands
Country
Australia
Individual Site Status
Recruiting
Facility Name
The Childrens Hospital at Westmead
City
Westmead
Country
Australia
Individual Site Status
Recruiting
Facility Name
The Hospital for Sick Children (SickKids)
City
Toronto
State/Province
Ontario
Country
Canada
Individual Site Status
Recruiting
Facility Name
Alberta Children's Hospital
City
Calgary
Country
Canada
Individual Site Status
Recruiting
Facility Name
CHU de Quebec Research Centre
City
Quebec City
Country
Canada
Individual Site Status
Recruiting
Facility Name
Hospital de Niños Roberto del Rio
City
Santiago
Country
Chile
Individual Site Status
Recruiting
Facility Name
Pontificia Universidad Católica de Chile
City
Santiago
Country
Chile
Individual Site Status
Recruiting
Facility Name
Agia Sofia Children's Hospital
City
Athens
Country
Greece
Individual Site Status
Recruiting
Facility Name
Hippokration General Hospital of Thessaloniki
City
Thessaloníki
Country
Greece
Individual Site Status
Recruiting
Facility Name
Hong Kong Children's Hospital
City
Kowloon Bay
Country
Hong Kong
Individual Site Status
Recruiting
Facility Name
Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore
City
Rome
Country
Italy
Individual Site Status
Recruiting
Facility Name
Hyogo College of Medicine College Hospital
City
Hyōgo
Country
Japan
Individual Site Status
Withdrawn
Facility Name
Kumamoto University Hospital
City
Kumamoto
Country
Japan
Individual Site Status
Withdrawn
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
Country
Japan
Individual Site Status
Withdrawn
Facility Name
Pusan National University Yangsan Hospital
City
Pusan
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Seoul National University Hospital
City
Seoul
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Hospital Angeles Chihuahua
City
Chihuahua
Country
Mexico
Individual Site Status
Recruiting
Facility Name
Instituto Nacional de Pediatria
City
Ciudad de mexico
Country
Mexico
Individual Site Status
Recruiting
Facility Name
Radboud Universitair Medisch Centrum
City
Nijmegen
State/Province
Gelderland
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Leids Universitair Medisch Centrum
City
Leiden
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
New Zealand Clinical Research Ltd
City
Auckland
Country
New Zealand
Individual Site Status
Completed
Facility Name
Christchurch Clinical Studies Trust
City
Christchurch
Country
New Zealand
Individual Site Status
Withdrawn
Facility Name
Rikshospitalet
City
Oslo
Country
Norway
Individual Site Status
Recruiting
Facility Name
Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev
City
Moscow
Country
Russian Federation
Individual Site Status
Active, not recruiting
Facility Name
"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
City
Saint Petersburg
Country
Russian Federation
Individual Site Status
Active, not recruiting
Facility Name
Tomsk National Research Medical Center of Russian Academy of Sciences
City
Tomsk
Country
Russian Federation
Individual Site Status
Active, not recruiting
Facility Name
Hospital Sant Joan de Deu
City
Barcelona
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Universitario La Paz
City
Madrid
Country
Spain
Individual Site Status
Recruiting
Facility Name
Kaohsiung Medical University Chung-Ho Memorial Hospital
City
Kaohsiung
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
National Taiwan University Hospital
City
Taipei
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Yeditepe University Kosuyolu Hospital
City
Istanbul
Country
Turkey
Individual Site Status
Recruiting
Facility Name
State Institution "Ukrainian Medical rehabilitation Center for Children with organic disorders of the nervous system of the Ministry of Health of Ukraine"
City
Kyiv
Country
Ukraine
Individual Site Status
Recruiting
Facility Name
Birmingham Heartlands Hospital
City
Birmingham
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Royal Hospital for Children - Glasgow
City
Glasgow
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
University College London Institute of Child Health
City
London
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Royal Manchester Children's Hospital
City
Manchester
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

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

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