Status:
RECRUITING
CD4^LVFOXP3 in Participants With IPEX
Lead Sponsor:
Bacchetta, Rosa, MD
Collaborating Sponsors:
California Institute for Regenerative Medicine (CIRM)
Conditions:
IPEX
Eligibility:
MALE
4-35 years
Phase:
PHASE1
Brief Summary
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 e...
Detailed Description
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...
Eligibility Criteria
Inclusion
- 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
- 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 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
Key Trial Info
Start Date :
March 22 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 1 2037
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT05241444
Start Date
March 22 2022
End Date
February 1 2037
Last Update
May 22 2025
Active Locations (1)
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1
Lucile Packard Children's Hospital
Palo Alto, California, United States, 94305