IPEX Clinical Trial
Official title:
Phase 1 Study of Autologous CD4^LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome
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 36 evaluable human participants with IPEX and evaluate the impact of the CD4^LVFOXP3 infusion on the disease.
Treatment with CD4^LVFOXP3 is expected to replace the defective Treg cells of the participants, and restore control of the immune system and therefore ameliorate symptoms of IPEX. We expect to learn the following from this study: 1. That CD4^LVFOXP3 can be consistently produced and be of expected quality to be used in humans, 2. That CD4^LVFOXP3 are safe in children and young adults with IPEX, and determine its effects, both good and bad, 3. That CD4^LVFOXP3 can improve overall health and allow reduction of medication/s. This Phase 1 (feasibility and safety) trial will gather data about CD4^LVFOXP3 in vivo persistency and early signs of impact on symptoms of IPEX. ;
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