HOPE-Duchenne
19 evidence topics · 7 sources
Objective
Safety and efficacy objective
Location and study date
HOPE-Duchenne: location
Enrollment period
| Milestone | Reported date |
|---|---|
| Enrollment began | “January 7, 2016” |
| Enrollment ended | “August 15, 2016” |
Study design
Early randomized controlled design
Eligibility criteria
Cardiac disease in the studied population
Minimum age in the sponsor study summary
Treatment
HOPE-Duchenne: intracoronary administration
Study outcomes
Rationale for using serious adverse events as the primary endpoint
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
HOPE-Duchenne: actual enrollment
Target sample size
Description of analysis sets
Primary efficacy analysis population
HOPE-Duchenne: upper-limb analysis qualification
Results
Participant disposition
Discontinuation due to adverse events
Baseline characteristics
Efficacy results
Health-related quality of life and patient-reported outcomes
Exploratory questionnaire findings: nominal comparisons favouring CAP-1002 unless stated otherwise
| Instrument and comparison | Reported p value |
|---|---|
| Pediatric Outcomes Data Collection Instrument: patient-reported global function, month 3 | “0.02” |
| Pediatric Outcomes Data Collection Instrument: patient-reported happiness, month 12 | “0.03” |
| Pediatric Outcomes Data Collection Instrument: parent-reported happiness, month 12 | “0.0003” |
| Pediatric Quality of Life Inventory: patient-reported worry, month 6 | “0.047” |
| Pediatric Quality of Life Inventory: patient-reported worry, month 12 | “0.043” |
| Pediatric Quality of Life Inventory: patient-reported communication, month 6 | “0.043” |
| Pediatric Quality of Life Inventory: patient-reported communication, month 12 | “0.01” |
| Pediatric Quality of Life Inventory: treatment barriers, week 6, favouring usual care | “0.01” |
Safety results
Periprocedural major adverse cardiac events in each group
Sponsor safety overview
Study limitations
Summary
HOPE-Duchenne was an open-label phase I/II trial with 25 participants and a single intracoronary administration rather than the repeated intravenous regimen used in later trials. The upper-limb responder analysis and definition of the lower-functioning subgroup were post hoc. Statistical testing did not adjust for multiplicity, so the nominal questionnaire and functional findings should not be interpreted as independently confirmed effects. Its size, open-label design, and different route and dosing schedule limit extrapolation to the proposed repeated-infusion treatment.