Trial · NCT04060199

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

NCT04060199, Phase 3, Completed, NS Pharma, Inc.; results first posted 2024-12-11

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53). Trials, record NCT04060199. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04060199

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
NCT number: NCT04060199
Status: Completed
Phases: Phase 3
Enrollment: 77 participants, actual
Lead sponsor: NS Pharma, Inc.
Conditions: Duchenne Muscular Dystrophy
Interventions: Viltolarsen, Placebo
First posted: 2019-08-19
Last updated: 2024-12-11
Results first posted: 2024-12-11

Source ClinicalTrials.gov

Registered outcomes1

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in Time to Stand (TTSTAND) Velocity	baseline, Week 13, 25, 37, 49	The change from baseline for velocity converted from TTSTAND was compared between the viltolarsen-treated patients and the placebo-treated patients. TTSTAND was assessed as the time it takes the participant to go from lying flat on the floor to standing. The time measured for TTSTAND was converted to a velocity expressed as rise per second.

Source ClinicalTrials.gov

Outcomes1

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Time to Stand (TTSTAND) Velocity	baseline, Week 13, 25, 37, 49	The modified ITT (mITT) Population was set as the analysis population. The mITT Population was defined as all randomized patients who received at least 1 dose of IP and had a baseline assessment and at least 1 post baseline efficacy assessment. The change from baseline for velocity converted from TTSTAND was compared between the viltolarsen-treated patients and the placebo-treated patients using a mixed-effect model for repeated measures (MMRM) analysis.	rise/s	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group8

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in Time to Stand (TTSTAND) Velocity	Viltolarsen	Week 13	0.026	Standard Error 0.0103	rise/s
Change From Baseline in Time to Stand (TTSTAND) Velocity	Placebo	Week 13	0.013	Standard Error 0.0104	rise/s
Change From Baseline in Time to Stand (TTSTAND) Velocity	Viltolarsen	Week 25	0.027	Standard Error 0.0100	rise/s
Change From Baseline in Time to Stand (TTSTAND) Velocity	Placebo	Week 25	0.014	Standard Error 0.0100	rise/s
Change From Baseline in Time to Stand (TTSTAND) Velocity	Viltolarsen	Week 37	0.027	Standard Error 0.0107	rise/s
Change From Baseline in Time to Stand (TTSTAND) Velocity	Placebo	Week 37	0.027	Standard Error 0.0108	rise/s
Change From Baseline in Time to Stand (TTSTAND) Velocity	Viltolarsen	Week 49	0.009	Standard Error 0.0096	rise/s
Change From Baseline in Time to Stand (TTSTAND) Velocity	Placebo	Week 49	0.013	Standard Error 0.0096	rise/s

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Viltolarsen	38
Overall Study	STARTED	Placebo	39
Overall Study	COMPLETED	Viltolarsen	36
Overall Study	COMPLETED	Placebo	38
Overall Study	NOT COMPLETED	Viltolarsen	2
Overall Study	NOT COMPLETED	Placebo	1

Source ClinicalTrials.gov

Adverse events120

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	COVID-19	Infections and infestations	Viltolarsen	12	38	13
other	COVID-19	Infections and infestations	Placebo	12	39	13
other	Upper respiratory tract infection	Infections and infestations	Viltolarsen	10	38	18
other	Upper respiratory tract infection	Infections and infestations	Placebo	9	39	12
other	Nasopharyngitis	Infections and infestations	Viltolarsen	9	38	25
other	Nasopharyngitis	Infections and infestations	Placebo	7	39	16
other	Rhinitis	Infections and infestations	Viltolarsen	6	38	10
other	Rhinitis	Infections and infestations	Placebo	3	39	5
other	Gastroenteritis	Infections and infestations	Viltolarsen	3	38	4
other	Gastroenteritis	Infections and infestations	Placebo	4	39	4

1 to 10 of 120Source ClinicalTrials.gov

See all 120 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Viltolarsen	0	38
Total, all-cause mortality	Placebo	0	39
Total, other adverse events	Viltolarsen	33	38
Total, other adverse events	Placebo	37	39
Total, serious adverse events	Viltolarsen	2	38
Total, serious adverse events	Placebo	3	39

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Viltolarsen vs Placebo: deaths from any cause	Trial comparisons
Viltolarsen vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04060199

GET https://conductscience.org/api/trials/v1/NCT04060199/events?page=2

API reference For AI assistants