HOPE-3 evaluated deramiocel (allogeneic cardiosphere-derived cells) vs. placebo in 106 participants aged ≥10 years with advanced DMD, administered IV every 3 months for 12 months, with skeletal and cardiac function as outcomes.
Deramiocel significantly improved the primary endpoint (total PUL2.0 score) vs. placebo at 12 months: least-squares mean percentage change favoured deramiocel by **4.55%** (95% CI 0.47–8.63; p=0.029), with a safety profile similar to placebo.
- Relatively small sample size (n=106) for a phase 3 trial. - 12-month follow-up may be insufficient to capture long-term cardiac or functional outcomes in a progressive disease. - Cardiac outcomes are described but specific cardiac endpoint results are not reported in the abstract.
Deramiocel, given quarterly IV in an outpatient setting, slows upper limb function decline in advanced DMD regardless of the underlying mutation. Clinicians managing advanced DMD patients should be aware of this mutation-agnostic option as it moves toward potential regulatory review.
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