CD4^LVFOXP3 in Participants With IPEX
Trial ID or NCT#
Status
Purpose
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.
Official Title
Phase 1 Study of Autologous CD4^LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome
Eligibility 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
- * 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 cardiac, renal, pulmonary, liver, or hematologic 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
Investigator(s)
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Contact
SCGT Clinical Trials Program
650-723-6661
View on ClinicalTrials.gov