Trial Search Results

CD4^LVFOXP3 in Participants With IPEX

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.

Stanford is currently accepting patients for this trial.

Lead Sponsor:

Bacchetta, Rosa, MD

Collaborator: California Institute for Regenerative Medicine (CIRM)


  • Biological: CD4^LVFOXP3


Phase 1


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 still requiring immune suppressive medication

   - 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 (e.g., cervix, bladder, breast)

Ages Eligible for Study

4 Months - 35 Years

Genders Eligible for Study


Now accepting new patients

Contact Information

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