Education and Training
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.
Stanford Investigator(s):
Intervention(s):
- biological: CD4^LVFOXP3
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 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
Ages Eligible for Study
4 Months - 35 Years
Genders Eligible for Study
Male
Now accepting new patients
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
SCGT Clinical Trials Program
650-723-6661
I'm interested