PROMOVI
12 evidence topics · 2 sources
Objective
Domains evaluated
Location and study date
Study design
Eligibility criteria
Ambulatory participant age range
Treatment
Study outcomes
Rationale for using Six-Minute walk test distance as the primary endpoint
Primary endpoint: change from baseline to week 96
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
Results
Participant disposition
Baseline characteristics
Eteplirsen baseline age
| Characteristic | Quoted result |
|---|---|
| Age, years, mean and standard deviation | “9.1±2.0” |
Efficacy results
Eteplirsen PROMOVI trial: week 96
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
Most frequent treatment-related adverse events
Study limitations
Summary
PROMOVI was open label and included 79 eteplirsen-treated and 30 untreated participants. Completion was reported for 78 of 79 treated participants and 15 of 30 untreated participants. The investigators considered the untreated cohort inappropriate for comparison because of genotype-related differences in clinical trajectory. The reported seven-fold dystrophin increase is relative to baseline and is not interchangeable with an absolute percentage of normal dystrophin. This was not a direct comparison with z-rostudirsen.