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This study examines CD4^LVFOXP3, a gene-modified cell product, in participants with IPEX syndrome caused by a FOXP3 gene mutation. It enrolls patients with a FOXP3 gene mutation who have progressive IPEX symptoms and require immunosuppressive medication. The study is being conducted in Phase 1 and is currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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