Depleted Donor Stem Cell Transplant for Fanconi Anemia
This study is for children and adults with Fanconi Anemia (a genetic disorder affecting bone marrow). It aims to find a safer way to perform stem cell transplants by using donor stem cells that have been specially prepared to remove certain immune cells (called TCRαβ+ T-cells and CD19+ B-cells) using a device called CliniMACS Prodigy System. Participants will also receive a conditioning regimen that includes drugs like Cyclophosphamide and Rabbit Anti-Thymoglobulin (rATG), and some will also receive an experimental antibody treatment called JSP191. The goal is to reduce side effects compared to standard chemotherapy and help the new stem cells engraft (take hold) better. The study will enroll 18 participants and will track serious side effects and successful engraftment for up to two years after the transplant.
- Study design
- This study is an interventional trial with a planned enrollment of 18 participants. The phase is not specified.
- What's involved
- You would receive a conditioning regimen, a depleted stem cell infusion, and be followed for up to 2 years after the cell infusion.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 2 years after the cell infusion.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Rajni Agarwal, MD · PRINCIPAL_INVESTIGATOR · Stanford University
Who to contact
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What this trial measures
- Number of participants without grade 3 and 4 treatment-emergent adverse events (TEAEs) (infusion related reactions).From start of conditioning regimen administration until cell infusion (up to 30 days)
Recorded and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
- Number of participants without grade 3 and 4 treatment-emergent adverse events (TEAEs) (infusion related reactions) following infusion of TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic graft transplantationUp to 2 years post-cell infusion
- Number of participants able to achieve donor engraftmentAssessed at Day +42 post-cell infusion
Participants have achieved engraftment when absolute Neutrophil Count (ANC) is above 500/mm\^3 for three consecutive laboratory values obtained on different days post cell transplantation with \>1% CD15 donor chimerism
- Number of participants who are able to have donor engraftment persist at the same rate or better compared to alternative hematopoietic cell transplant regimens for this patient populationAssessed at Day +100 post-cell infusion