NCT04784052

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

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Porteus, Matthew, MD · Academic / Other
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Fanconi Anemia

Interventions

Type	Name	Description
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
DRUG	Rituximab	1 dose of rituximab will be given at the end of conditioning

Timeline

• Start date: 2021-12-07
• Primary completion: 2027-12-01
• Completion: 2028-12-01
• First posted: 2021-03-05
• Last updated: 2026-01-27

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study