Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
brief summary
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to participants who were randomized to the Usual Care treatment group of the HOPE-Duchenne study (NCT02485938) and completed 12 months of follow-up. The trial will assess the safety and efficacy of two intravenous administrations of CAP-1002, each separated by three months.
detailed description
Participants with documented enrollment in the Usual Care treatment group of the HOPE-Duchenne study and completion of study follow-up through Month 12 were eligible for this study.
Participants will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of CAP-1002.
All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and at Month 3. Participants will be observed in the outpatient setting for at least two hours post-infusion and then discharged the same day if medically cleared by the site Investigator.
official title
Open-Label Extension of the Halt Cardiomyopathy Progression in Duchenne (HOPE-Duchenne) Trial (CAP-1002-DMD-03)