Deramiocel is described as a heart-derived cellular therapy under investigation for patients with advanced Duchenne muscular dystrophy (DMD). The HOPE-3 trial was conducted to evaluate this investigational therapy in a late-phase, confirmatory setting. The PubMed entry identifies the trial as a phase 3, randomised, double-blind, placebo-controlled study published in Lancet.
The public citation classifies HOPE-3 as a phase 3, randomised, double-blind, placebo-controlled clinical trial. The PubMed record lists the article title, authors, institutional affiliations, DOI, and publication date. The accessible portion of the source does not include the detailed trial protocol, randomisation ratio, blinding procedures, dosing schedule, or follow-up duration. Those methodological specifics were not reported in the source text provided here.
The PubMed entry lists the lead author Craig M McDonald and a multi-institutional author group representing paediatric neurology, cardiology, and rare-disease centres across the USA and internationally. Multiple clinical sites and a long collaborator list labelled “HOPE-3 Investigators” are included. The source does not provide the number of enrolled participants, inclusion or exclusion criteria, baseline characteristics, or geographic distribution of trial sites in the accessible text.
The investigational product evaluated is Deramiocel, described in the title as a heart-derived cell therapy. The PubMed record does not include a description of manufacture, cell source, cell dose, route or schedule of administration, or comparator/placebo composition in the accessible content. Those details were not reported in the source text available here.
The article is identified as a phase 3 efficacy and safety trial, implying predefined primary and secondary clinical endpoints. However, the accessible PubMed content does not list the specific primary endpoint(s) (for example functional, respiratory, cardiac, or survival measures), secondary endpoints, or statistical analysis plan. The source text provided does not report those endpoint definitions or prespecified analyses.
The PubMed entry shown here provides bibliographic and authorship information but does not include the trial results, effect sizes, confidence intervals, p values, or responder analyses. Specific efficacy outcomes, numerical results, and statistical conclusions are not present in the accessible portion of the source and therefore cannot be summarized from this record.
Although phase 3 trials routinely report adverse events and safety profiles, the PubMed text available here does not include any safety data, adverse event frequencies, serious adverse events, or tolerability findings for Deramiocel. These safety outcomes were not reported in the accessible source content.
The article lists a broad group of authors and collaborators. Lead and senior authors include clinicians and researchers affiliated with Davis Health System/University of California, Cincinnati Children’s Hospital, Vanderbilt University Medical Center, Capricor Therapeutics, Children’s National Hospital, and multiple paediatric centres. The record also names numerous HOPE-3 investigators and site personnel. The PubMed entry includes institutional affiliations and electronic contact for the corresponding author.
The trial report is cited as published in Lancet, 2026 Aug 22;408(10556):721–733, with DOI 10.1016/S0140-6736(26)01385-1 and an electronic publication date of 2026 Jul 29. The PubMed page links to full text options via Elsevier Science. No abstract content or trial data are available in the portion of the PubMed record provided here beyond bibliographic information.
This PubMed record confirms the existence of a multicentre phase 3 randomized, double-blind, placebo-controlled trial (HOPE-3) of Deramiocel in advanced Duchenne muscular dystrophy and provides full authorship and publication metadata. However, the accessible source content does not include trial methods in detail, participant numbers, outcome definitions, efficacy results, or safety data. Because those key clinical details are not present in this PubMed entry excerpt, no conclusions about efficacy or safety can be drawn from this source alone. To interpret the trial findings and clinical implications, the full Lancet article or complete abstract must be consulted for methodology, endpoints, numerical results, and adverse event reporting.