TCRαβ+ T-cell/CD19+ B-cell Depleted Hematopoietic Grafts and a Reduced Intensity Preparative Conditioning Regimen Containing JSP191 (Briquilimab) to Achieve Engraftment and Blood Reconstitution in Patients With Fanconi Anemia

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 with and without using an experimental antibody 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.

Eligibility

Age range

2+ years

Sex

All

Healthy volunteers

No

Interventions

• Drug
  JSP191

  Participants will receive a single IV dose at start of conditioning

• Device
  CliniMACS Prodigy System

  The device used to remove the αβ+T cells from donor stem cell transplant before being given to the recipient

• Biological
  Depleted Stem Cell Transplant

  TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic cells will be administered by IV after completion of conditioning regimen.

• Biological
  Rabbit Anti-Thymoglobulin (rATG)

  3 consecutive daily doses of rATG will be given by IV during conditioning

• Drug
  Cyclophosphamide

  4 consecutive daily doses of cyclophosphamid will be given by IV during conditioning

• Drug
  Fludarabine

  4 consecutive daily doses of fludarabine will be given by IV during conditioning

Location