RecruitingIPEX
CD4^LVFOXP3 in Participants With IPEX
Eligible age
0.3–35 yrs
Accepts
Men
Locations
1 state
Healthy volunteers
No
See if you qualify for this study
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About this study
This first-in-human, Phase 1 clinical trial will test the feasibility of the manufacturing and the safety of the administration of CD4\^LVFOXP3 in up to 30 evaluable human participants with IPEX and evaluate the impact of the CD4\^LVFOXP3 infusion on the disease.
Sponsor: Bacchetta, Rosa, MD
You may qualify if…
- ✓ Body weight greater than 8 kg, unless assessed as able to tolerate leukapheresis
- ✓ FOXP3 gene mutation
- ✓ Medical history of progressive symptoms of IPEX with persistency of some symptoms and/or signs requiring immune suppressive medication. The participant may or may not be on immunosuppression at time of starting the study.
- ✓ Uncontrolled IPEX disease but unable to tolerate immune suppressive medication
- ✓ Recurrent IPEX symptoms, requiring immune suppressive medications, in participants who have had prior allogeneic (allo) blood stem cell transplantation (HSCT).
- ✓ ≥ 50% Performance rating on Lansky/Karnofsky Scale
- ✓ Organ and marrow function within acceptable levels of function
- ✓ Absence of ongoing infections
You may not qualify if…
- ✕ Medical instability
- ✕ Less than 6 months life expectancy
- ✕ Inability to meet limits for steroid dosing
- ✕ Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant, and be willing to undergo transplant.
- ✕ Unrelated or comorbid disease
- ✕ Allergy to any study medication, product, or intervention
- ✕ Currently receiving another experimental treatment
- ✕ History of malignancy, unless disease free for at least 2 years, with the exception of non melanoma skin cancer or carcinoma in situ
Where it's recruiting
California
Palo Alto
Source: ClinicalTrials.gov · NCT05241444 · last updated 2025-05-22