In short
Clinical trials are studying Autologous Cord Blood-Derived Dendritic Cells Loaded With Wt1 Peptide Pool in children and young adults with acute myeloid leukemia. The trials are looking at safety and whether the vaccine may help lower relapse risk after cord blood transplant. They focus on patients with WT1-positive AML.
Key points
- Clinical trials are studying Autologous Cord Blood-Derived Dendritic Cells Loaded With Wt1 Peptide Pool in pediatric and young adult patients with acute myeloid leukemia. The trial is a phase I/II study, and the listed phase is Phase 1. It focuses on patients after cord blood transplant, especially those with WT1-positive AML. The main goal in the early part is safety, measured by dose-limiting toxicities. The later part looks at whether the vaccine may improve one-year relapse-free survival compared with historic controls. The trial is currently suspended.
Trial overview
The clinical trial titled U-DANCE-anti-AML is a phase I/II post-cord blood HCT dendritic cell vaccination study directed against WT1 for pediatric and young adult acute myeloid leukemia. It is listed as an interventional study and has a target enrollment of 54 participants. The trial status is suspended.
The study investigates Autologous Cord Blood-Derived Dendritic Cells Loaded With Wt1 Peptide Pool as a WT1-loaded CBDC vaccine given by intravenous injection. The trial is focused on AML, which is described in the source as cancer of the blood and bone marrow.
Who the trial is for
This trial is aimed at pediatric and young adult patients with AML. The brief summary also says the study uses a historic cohort of patients who received cord blood transplant but did not receive the vaccine, showing that the trial is centered on the period after transplant.
The condition is described more specifically as WT1+ AML in the study summary and primary outcome description. In simple terms, this means the leukemia group being studied has the WT1 target that the trial is built around.
What the trial is studying
The main question in Part A is whether the vaccine dose is safe after cord blood transplant. Safety is checked by looking for dose-limiting toxicities, which are serious side effects that may limit treatment.
Part B looks at activity, meaning whether the study treatment may help patients stay free of relapse longer. The trial summary says the goal is to increase the one-year WT1+ AML relapse-free survival rate from 50% to 70% compared with historic controls.
The source also states that the vaccine is given after cord blood transplantation, which is a transplant that uses blood from an umbilical cord to help rebuild blood-forming cells. This tells us the study is not testing the disease itself, but a post-transplant vaccination approach.
Trial design and phase
The trial is described as a phase I/II study in the title, while the structured data lists the phase as Phase 1. This means the study starts with early safety testing and then includes a second part that looks more closely at activity.
The summary explains that the study uses a Simon 2-stage design for the second part. In simple language, this is a step-by-step plan that checks early results before moving ahead.
The first part of the study follows patients from the first vaccination until 84 days after the third CBDC vaccination for the safety check. The first 6 patients receiving the dose judged safe are also included in the first stage of the phase 2 design.
Outcomes being measured
The main safety outcome is the occurrence of DLTs from the first vaccination until 84 days after the third vaccination. This outcome helps the researchers decide whether the dose can be used safely in the next part of the study.
The main activity outcome is the one-year WT1+ AML relapse-free survival rate from the time of the first vaccination. This means the study checks how many patients remain free from leukemia coming back during the first year after vaccination.
The study compares this result with a historic group of patients from 2010 to 2015 who had cord blood transplant but did not receive a CBDC vaccination. This comparison helps researchers see whether the trial group does better than earlier patients who had standard transplant care alone.
Status and comparison group
The trial is currently listed as suspended. In trial language, this means the study is not actively moving forward at the time shown in the source.
Instead of using a live control group in the same study, the researchers use historic controls from earlier years. These are past patient records used as a comparison group, which is common in some early studies when a randomized control group is not listed.
Key patient terms
- Interventional study: a study where participants receive a treatment or procedure that the researchers are testing.
- WT1: the target used in this trial to guide the vaccine approach.
- Relapse-free survival: the time a patient stays free from the cancer coming back.
- Primary outcome: the main result the study is designed to measure.
- Phase 1: an early trial phase that mainly checks safety and dose.