Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04060199|
Recruitment Status : Recruiting
First Posted : August 19, 2019
Last Update Posted : April 1, 2022
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Viltolarsen Drug: Placebo||Phase 3|
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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||74 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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 :||April 14, 2020|
|Estimated Primary Completion Date :||November 2024|
|Estimated Study Completion Date :||December 2024|
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Other Name: 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.
- TTSTAND [ Time Frame: baseline to 48 weeks of treatment ]Change in Time to Stand (TTSTAND)
- TTRW [ Time Frame: baseline to 48 weeks of treatment ]Change in Time to Run/Walk 10 Meters Test (TTRW)
- 6MWT [ Time Frame: baseline to 48 weeks of treatment ]Change in Six-minutes Walk Test (6MWT)
- NSAA [ Time Frame: 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 [ Time Frame: baseline to 48 weeks of treatment ]Change in Time to Climb 4 Steps Test (TTCLIMB)
- Hand-held dynamometer [ Time Frame: 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.
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): NCT04060199
|Contact: Trial Infofirstname.lastname@example.org|