Actively Recruiting
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
Led by Porteus, Matthew, MD · Updated on 2026-01-27
18
Participants Needed
1
Research Sites
364 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Fanconi Anemia confirmed by chromosome breakage tests and mutation in Fanconi gene
- Bone marrow failure with reduced blood cells on two separate occasions at least one month apart
- Age 2 years or older
- Available consenting donor matched at least 5 out of 10 HLA markers for stem cell collection
- Adequate organ function including kidney, lung, heart, liver, and coagulation parameters
- Life expectancy of at least 2 years
- Willingness to use effective contraception if of childbearing potential during treatment and recovery
- Ability to provide written informed consent or have a legal guardian provide consent
You will not qualify if you...
- Having a fully matched 10 out of 10 HLA-identical sibling donor available
- Active or uncontrolled infections at enrollment
- Positive for HIV-I/II or HTLV-I/II
- Receiving other investigational, chemotherapy, or radiation treatments within 14 days before enrollment
- Presence of active cancer, myelodysplastic syndrome, or high-risk bone marrow disease
- Use of androgens within the last 3 months
- Pregnant or breastfeeding women
- Lansky/Karnofsky performance score below 50%
- Any other medical condition or history posing significant safety risks or study compliance issues as judged by the investigator
- Inability to comply with study safety monitoring requirements
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Stanford University
Stanford, California, United States, 94305
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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