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CD4^LVFOXP3 in Participants With IPEX
RecruitingPhase 1
Phase 1 Study of Autologous CD4^LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome
Who can join
4 Months – 35 Years · Men
Full eligibility criteria
Inclusion Criteria: * 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 * Must be able to consent if an adult Exclusion Criteria: * 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
About the 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.
What is being tested
- CD4^LVFOXP3 (biological)
Sponsor: Bacchetta, Rosa, MD · Participants: 30 · Started: Mar 22, 2022
Contact the study team
- Rosa Bacchetta, MD · Phone: 650-498-8369
Official record on ClinicalTrials.gov — NCT05241444
Locations in the U.S.
| California | Lucile Packard Children's Hospital, Palo Alto |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.