MedTrace Logo

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

NCT04784052 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-01-27

No results posted yet for this study

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

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

Sponsors & Collaborators

  • Porteus, Matthew, MD

    lead OTHER

Principal Investigators

  • Rajni Agarwal, MD · Stanford University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-12-07
Primary Completion
2027-12-31
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04784052 on ClinicalTrials.gov