HOPE-OLE
15 evidence topics · 6 sources
Objective
Exploratory safety and efficacy objective
Location and study date
Sponsor-reported first-visit and last-visit dates
| Milestone | Reported date |
|---|---|
| First participant first visit | “21 Jun 2018” |
| Last participant last visit | “06 Mar 2019” |
Planned study centers
Study design
Open-label extension design
Eligibility criteria
Treatment
Intravenous dose per administration
Number of administrations
Repeat-dose visit
Study outcomes
Rationale for using serious adverse events as a primary endpoint
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
Planned enrollment
Actual treated population
Description of analysis sets
Analysis-set definitions
| Analysis set | Definition excerpt |
|---|---|
| Intention to treat | “meet the enrollment criteria and are enrolled in the study” |
| Safety | “all subjects who receive any amount of IP.” |
Descriptive analyses
Results
Participant disposition
Completion of enrollment and treatment
Baseline characteristics
No evidence found.
Efficacy results
No evidence found.
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
Serious anaphylactic reaction: participants, percentage, and event count
Study limitations
Summary
HOPE-OLE treated 8 participants who had previously been allocated to usual care in HOPE-Duchenne. It was an open-label extension without a concurrent randomized comparator. The regimen comprised two intravenous administrations of 75 million cells, with the repeat dose at month 3, rather than the 150-million-cell regimen used in HOPE-2 and HOPE-3. The small, selected population and different exposure limit its contribution to efficacy assessment and extrapolation to later dosing regimens.