Evicenter
P&T meetings

Deramiocel (CAP-1002)

Duchenne muscular dystrophy

Launch
Not announced
Last updated
92 sources

Clinical evidence

HOPE-2-OLE

26 evidence topics · 9 sources

Objective
Location and study date
Study initiation and country
Study design
Eligibility criteria
Treatment
Study outcomes
Rationale for using performance of the upper limb 2.0 combined total score as the primary efficacy endpoint
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
Results
Participant disposition
Baseline characteristics
Extension baseline characteristics
CharacteristicReported value
Sex and ambulation“all non-ambulant males”
Mean age“16.5 yr”
Age range, years“12–23”
Mean PUL 2.0 total score“20.4”
Mean LVEF, percent“51.2”
Efficacy results
Three-year modeled change as presented in the five-year update
GroupModeled PUL 2.0 change at three years
Deramiocel“−3.46”
External comparator“-7.19”
HOPE-2 and open-label extension: 36-month PUL v2.0 analysis
MeasureReported result
Deramiocel mean decline“4.0-pt”
External-comparator mean decline“7.7 points”
Difference“Δ=3.7”
Reported p value“p <0.001”
Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results
Safety in the 36-month poster
MeasureNumber and percentage
Participants with any treatment-emergent adverse event“13 (100.0%)”
Participants with a severe treatment-emergent adverse event“5 (38.5%)”
Participants with a treatment-related adverse event“9 (69.2%)”
Participants with a treatment-related serious adverse event“0”
Study limitations
Summary

The extension enrolled 13 participants who had completed the parent trial; all received deramiocel, and 9 continued to month 60. The absence of a concurrent randomized control group, selection of prior-trial completers, and attrition limit causal interpretation. External-comparator analyses depend on modeling and cross-study comparability. The five-year comparator estimate is a projection, not an observed five-year randomized comparison. The earlier poster described the 3.46-point and 7.19-point estimates as yearly declines, whereas the later presentation labels them as three-year modeled changes; these descriptions should not be combined as if they used the same time scale. Off-treatment comparisons used linear interpolation, and analyses were exploratory and unadjusted for multiplicity.