About this trial
This study aims to help patients who need a stem cell transplant but do not have a fully matched sibling donor. It focuses on those with serious or life-threatening diseases. The goal is to use a special device to remove certain cells that can cause problems after the transplant. This helps make the procedure safer for patients.
The trial's ID number is NCT02356653. It is in the early phase of testing, which means it is looking at how well the treatment works and how safe it is. The study aims to include about 100 participants.
Who can join
Patients up to 30 years old can join. Both men and women are welcome. To participate, patients must not have a matched sibling donor and must have a living donor ready. They should have specific illnesses that can be helped by a stem cell transplant. The medical team will check to see who meets these requirements.
Conditions
Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
The study team makes the final call on eligibility.
What happens in the study
Everyone in the study will receive a transplant using stem cells processed with a special device. This device helps to remove certain T cells that may cause issues after the transplant. Participants will be monitored for how well they do after the procedure, especially in the first year.
Where
This trial is running at 1 location in the United States. It is currently recruiting.
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania