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

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ClinicalTrials.gov Identifier: NCT04768062
Recruitment Status : Recruiting
First Posted : February 24, 2021
Last Update Posted : July 2, 2021
Sponsor:
Collaborator:
Nippon Shinyaku Co., Ltd.
Information provided by (Responsible Party):
NS Pharma, Inc.

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Viltolarsen Phase 3

Detailed Description:

This Phase 3 study is a 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. Patients will receive viltolarsen administered IV at weekly doses of 80 mg/kg.

Study NS-065/NCNP-01-302 will be comprised of a 96-week treatment period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 74 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : April 13, 2021
Estimated Primary Completion Date : June 2026
Estimated Study Completion Date : June 2026


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 96 weeks.
Drug: Viltolarsen
Received during weekly intravenous infusions
Other Name: NS-065/NCNP-01




Primary Outcome Measures :
  1. Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03 [ Time Frame: baseline to up to 96 weeks of treatment ]

Secondary Outcome Measures :
  1. Time to Stand Test (TTSTAND) [ Time Frame: baseline to 96 weeks of treatment ]
    Change in Time to Stand

  2. Time to Run/Walk 10 Meters Test (TTRW) [ Time Frame: baseline to 96 weeks of treatment ]
    Change in Time to Run/Walk 10 meters

  3. Six-minute Walk Test (6MWT) [ Time Frame: baseline to 96 weeks of treatment ]
    Change in Six-minute Walk

  4. North Star Ambulatory Assessment (NSAA) [ Time Frame: baseline to 96 weeks of treatment ]
    Change in North Star Ambulatory Assessment

  5. Time to Climb 4 Stairs Test (TTCLIMB) [ Time Frame: baseline to 96 weeks of treatment ]
    Change in Time to Climb 4 Stairs

  6. Muscle Strength Measured by Hand-Held Dynamometer [ Time Frame: baseline to 96 weeks of treatment ]
    Change in Muscle Strength Measured by Hand-Held Dynamometer



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04768062


Contacts
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Contact: Trial Info trialinfo@nspharma.com

Locations
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Italy
Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore Not yet recruiting
Rome, Italy
Japan
National Center of Neurology and Psychiatry Recruiting
Tokyo, Japan
Korea, Republic of
Pusan National University Yangsan Hospital Not yet recruiting
Pusan, Korea, Republic of
Seoul National University Hospital Not yet recruiting
Seoul, Korea, Republic of
New Zealand
Auckland Clinical Studies Ltd. Not yet recruiting
Auckland, New Zealand
Spain
Hospital Sant Joan de Deu Not yet recruiting
Barcelona, Spain
Turkey
Yeditepe University Kosuyolu Hospital Not yet recruiting
Istanbul, Turkey
Sponsors and Collaborators
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.
Additional Information:
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Responsible Party: NS Pharma, Inc.
ClinicalTrials.gov Identifier: NCT04768062    
Other Study ID Numbers: NS-065/NCNP-01-302
First Posted: February 24, 2021    Key Record Dates
Last Update Posted: July 2, 2021
Last Verified: June 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked