CD4^LVFOXP3 for IPEX Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests if using special immune cells can help patients with a rare immune disorder by replacing their faulty immune cells, aiming to restore immune balance and improve health.
Will I have to stop taking my current medications?
The trial protocol does not specify if you need to stop taking your current medications. However, it mentions that participants may or may not be on immunosuppressive medication when starting the study.
How is the CD4^LVFOXP3 treatment different from other treatments for IPEX Syndrome?
CD4^LVFOXP3 is unique because it involves gene therapy targeting the FOXP3 gene, which is crucial for the function of regulatory T cells that are defective in IPEX Syndrome. This approach aims to correct the underlying genetic cause of the disease, unlike other treatments that may only address symptoms.12345
What data supports the effectiveness of the treatment CD4^LVFOXP3 for IPEX Syndrome?
Who Is on the Research Team?
Jessie Alexander, MD
Principal Investigator
Stanford University
Are You a Good Fit for This Trial?
This trial is for individuals with IPEX Syndrome who have persistent symptoms despite medication, weigh over 8 kg, and can tolerate leukapheresis. Participants should have a FOXP3 gene mutation and be at least 50% functional on the Lansky/Karnofsky Scale. They cannot join if they have severe unrelated diseases, allergies to study treatments, certain cancer histories, are medically unstable or expected to live less than six months.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive CD4^LVFOXP3 infusion to replace defective Treg cells and restore immune control
Follow-up
Participants are monitored for safety, effectiveness, and long-term outcomes
What Are the Treatments Tested in This Trial?
Interventions
- CD4^LVFOXP3
Find a Clinic Near You
Who Is Running the Clinical Trial?
Bacchetta, Rosa, MD
Lead Sponsor
California Institute for Regenerative Medicine (CIRM)
Collaborator
Food and Drug Administration (FDA)
Collaborator