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NCT04768062

NS Pharma - RACER53-X

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

Locations:
enrollingByInvitation
London - GOSH
,
enrollingByInvitation
Manchester
,
enrollingByInvitation
Birmingham
,
enrollingByInvitation
Glasgow
,

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

  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

 
Can I take part?

 
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.
 

Age

5 - 8 years

Mutation Specific

Mutation Specific

Muscle Biopsy

Not Required

Ambulation

ambulant