HOPE-3 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys and young men with Duchenne muscular dystrophy. The study randomized 106 participants across 20 leading U.S. clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for a 12-month period.
The average age of participants was approximately 15 years, and all were on a stable corticosteroid regimen throughout the study. Baseline demographics were well balanced between treatment arms, approximately 90 percent were receiving cardiac medications at baseline, and over 75 percent had a clinical diagnosis of cardiomyopathy. Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience. >>> What is Deramiocel? What Does CAP-1002 Do?
Topline Efficacy Results
| Endpoint | % Slowing of Progression3 (Deramiocel vs. Placebo) | p-value |
| Performance of Upper Limb (PUL v2.0) Total Score¹ (Primary, n=105) | 54% | p=0.029 |
| Left Ventricular Ejection Fraction (LVEF %)² (Key Secondary, n=83) | 91% | p=0.041 |
¹ n reflects the number of patients in the ITT population with evaluable PUL v2.0 assessments at 12 months.
² n reflects the number of patients in the ITT population with centrally reviewed and evaluable cardiac MRI LVEF assessments at 12 months.
3 Percent slowing is calculated as the treatment difference divided by the placebo change from baseline.
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Salut, je suis une maman d’un enfant de 15ans qui est atteint d’une myopathie de Duchêne j’ai besoin d’un aide pour les essais cliniques merci
Salut je suis un parent de 2 enfants jumeaux de 9 ans qui sont atteints d’une myopathie de douchéne delition d’exons de l’exon 3 au 73 sont ombilants j’ai besoin d’un aide pour les essais cliniques et merci très bien
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