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NS Pharma - RACER53-X
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
Hub 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.
The dystrophin gene has 79 pieces called exons. These link together to form a code which instructs the body to make dystrophin. If there is a fault, as in DMD, the sequence is broken and the code cannot be read. Exon skipping drugs complete the sequence and leads to a shortened dystrophin being produced that still contains the important pieces of this molecule.
Study Number: NCT04768062
Description by NS Pharma, Inc.
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.
Primary Outcome Measures
- 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
- Time to Stand Test (TTSTAND) [ Time Frame: baseline to 96 weeks of treatment ]
Change in Time to Stand
- Time to Run/Walk 10 Meters Test (TTRW) [ Time Frame: baseline to 96 weeks of treatment ]
Change in Time to Run/Walk 10 meters
- Six-minute Walk Test (6MWT) [ Time Frame: baseline to 96 weeks of treatment ]
Change in Six-minute Walk
- North Star Ambulatory Assessment (NSAA) [ Time Frame: baseline to 96 weeks of treatment ]
Change in North Star Ambulatory Assessment
- Time to Climb 4 Stairs Test (TTCLIMB) [ Time Frame: baseline to 96 weeks of treatment ]
Change in Time to Climb 4 Stairs
- Muscle Strength Measured by Hand-Held Dynamometer [ Time Frame: baseline to 96 weeks of treatment ]
Change in Muscle Strength Measured by Hand-Held Dynamometer
Can I take part?
Inclusion Criteria
- Patient has completed the NS-065/NCNP-01-301 study;
- 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;
- 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
- 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;
- 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;
- Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;
- Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.
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Fully recruited
NS Pharma, Inc.