Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial.
Rajni Agarwal, Alice Bertaina, Charmaine Soco...
https://pubmed.ncbi.nlm.nih.gov/40696207Actively Recruiting
Phase 1
Phase 2
Age: 2Years +
All Genders
ID04784052
TCRαβ+ T-cell/CD19+ B-cell Depleted Stem Cell Transplant with Reduced Intensity Conditioning Containing JSP191 (Briquilimab) for Fanconi Anemia
Led by Porteus, Matthew, MD · Updated on 2026-01-27
18
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new cell therapy for people with Fanconi Anemia to improve donor stem cell and immune system recovery after transplant. This trial aims to reduce the side effects seen with standard chemotherapy by using depleted donor stem cells and testing an experimental antibody treatment called JSP-191 as part of the preparative conditioning. The study is conducted in phases 1 and 2 to assess safety and effectiveness. Participants receive donor stem cells that have been depleted of certain T cells using the CliniMACS System. Before the transplant, they undergo a reduced-intensity conditioning regimen that may include JSP-191 combined with other drugs such as rATG, cyclophosphamide, fludarabine, and rituximab. There are two groups: one receives conditioning with JSP-191 and the other without it. Treatment is given through infusions over several days before the stem cell transplant. During the study, participants are monitored from the start of conditioning through at least two years after the stem cell infusion. Researchers assess side effects, donor cell engraftment in the bone marrow, immune system recovery, and survival without disease. Blood tests, lung and heart function tests, and safety evaluations are done regularly. The trial tracks how well the new therapy supports blood and immune cell recovery while monitoring for complications like graft-versus-host disease.
CONDITIONS
Brief Title
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
Who Can Participate
Age: 2Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Diagnosed with Fanconi Anemia confirmed by chromosome breakage studies and gene mutation
- Bone marrow failure with reduced blood counts on at least two occasions at least one month apart
- Age 2 years or older
- Have a consenting donor matched at 5/10 or more HLA markers available for stem cell donation
- Adequate organ function including kidney, lung, heart, liver, and blood clotting within defined limits
- Life expectancy of at least 2 years
- Willing to use effective contraception if of childbearing potential during the study
- Able to provide informed consent or have a legal guardian consent to participate
You will not qualify if you...
- Having a 10/10 HLA-identical sibling donor available and consenting for donation
- Any acute or uncontrolled infection at enrollment
- Positive for HIV-I/II or HTLV-I/II
- Receiving other investigational treatments, chemotherapy, or radiation within 14 days before enrollment
- Active cancer, myelodysplastic syndrome, or high-risk bone marrow disease
- Use of androgens in the last 3 months
- Pregnant or breastfeeding women who do not wish to stop nursing
- Poor performance status with Lansky/Karnofsky score below 50%
- Any medical condition or history that poses safety risks or affects study integrity as judged by the investigator
- Unable to comply with safety monitoring requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Run-in Period
Duration - Up to 30 days
Participants receive a reduced-intensity conditioning regimen including a single IV dose of JSP191, 3 daily doses of rATG, 4 daily doses of cyclophosphamide, 4 daily doses of fludarabine, and 1 dose of rituximab before the stem cell transplant.
Multiple visits during conditioning period
Treatment
Duration - 1 day
Participants receive an infusion of stem cells depleted of αβ+ T cells and CD19+ B cells using a device prior to the transplant.
1 day (in-person) for stem cell infusion
Post-treatment Follow-up
Duration - Up to 2 years
Participants are monitored for safety, engraftment, immune recovery, and potential complications such as graft-versus-host disease for up to 2 years after the stem cell infusion.
Regular follow-up visits during 2-year period
Trial Site Locations
Total: 1 location
1
Stanford University
Stanford, California, United States, 94305
Actively Recruiting
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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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