Overview

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing JSP191

Status:
Not yet recruiting

Trial end date:
2028-04-01

Target enrollment:

Participant gender:

Summary

The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor after using an experimental treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method). Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.

Phase:

Phase 1/Phase 2

Details

Lead Sponsor:

Maria Grazia Roncarolo

Treatments:

Antilymphocyte Serum
Cyclophosphamide
Fludarabine
Rituximab
Thymoglobulin