CD4^LVFOXP3 in Participants With IPEX


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Trial ID: NCT05241444


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.

Official Title

Phase 1 Study of Autologous CD4^LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome

Stanford Investigator(s)

Maria Grazia Roncarolo

George D. Smith Professor of Stem Cell and Regenerative Medicine and Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)

Shweta S. Namjoshi MD MPH

Clinical Associate Professor, Pediatrics - Gastroenterology


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 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


biological: CD4^LVFOXP3


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Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
SCGT Clinical Trials Program