In short
Clinical trials are investigating ALLOGENEIC ADIPOCYTE-DERIVED MESENCHYMAL STROMAL CELLS TRANSDUCED WITH A LENTIVIRAL PROVIRUS VECTOR CONTAINING THE HUMAN CXCR4 AND IL-10 GENES for acute graft-versus-host disease (GVHD). The trial data focus on safety and tolerability in people whose GVHD did not respond to standard treatment or who could not receive ruxolitinib.
Key points
- Clinical trials are investigating ALLOGENEIC ADIPOCYTE-DERIVED MESENCHYMAL STROMAL CELLS TRANSDUCED WITH A LENTIVIRAL PROVIRUS VECTOR CONTAINING THE HUMAN CXCR4 AND IL-10 GENES for acute graft-versus-host disease. The trial data focus on patients whose disease did not respond to corticosteroids and ruxolitinib, or who could not receive ruxolitinib. This was a Phase 1 study, so the main goal was to assess safety and tolerability. The primary endpoint was serious adverse reactions, including unexpected serious reactions during infusion and follow-up. The study planned to enroll 15 patients. Its status was Withdrawn.
Trial overview
This clinical trial studied ALLOGENEIC ADIPOCYTE-DERIVED MESENCHYMAL STROMAL CELLS TRANSDUCED WITH A LENTIVIRAL PROVIRUS VECTOR CONTAINING THE HUMAN CXCR4 AND IL-10 GENES for acute graft-versus-host disease (acute GVHD).
The study was designed as an interventional trial, which means the researchers planned to give a study treatment and watch what happened.
Who was studied
The trial focused on patients who had developed acute GVHD that was refractory, meaning it did not respond well to treatment.
More specifically, it included patients whose disease was refractory to corticosteroids and ruxolitinib, or patients who were not eligible to receive ruxolitinib.
In simple terms, this was a group of people with difficult-to-treat disease and limited treatment options.
Trial goals and study phase
This was a Phase 1 trial.
Phase 1 studies usually look first at safety and tolerability, which means whether the treatment can be given without causing too much harm and whether patients can handle it reasonably well.
The brief summary said the study aimed to analyze the safety and tolerability of the administration of the study treatment in patients with acute GVHD who had few remaining options.
Main endpoints
The primary outcome was safety, measured by serious adverse reactions after sequential infusions of the study drug during the full follow-up period.
The trial also looked for serious unexpected adverse reactions at the time of infusion or during follow-up.
These endpoints show that the study was mainly checking for harmful reactions and how patients responded over time.
Trial status and enrollment
The trial status was Withdrawn, which means it did not continue as planned.
The planned enrollment was 15 patients.
The trial title described the treatment as a new generation of mesenchymal stromal cells that ectopically express CXCR4 and IL-10, and the intervention was given by intravenous infusion.