CD4^LVFOXP3 in Participants With IPEX

ConditionIPEX

Trial statusRecruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexMale

Age4-35

SponsorBacchetta, Rosa, MD

About this trial

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 evaluate the impact of the CD4\^LVFOXP3 infusion on the disease.

Eligibility criteria

Qualifiers

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

Disqualifiers

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.

Trial design

Treatments tested in this trial

CD4^LVFOXP3

Treatment groups

30 Participants

are divided into 2 treatment groups

Sponsors and collaborators

Bacchetta, Rosa, MD

Lead sponsor

Stanford University

Sponsor institution

California Institute for Regenerative Medicine (CIRM)

Collaborator

Source ClinicalTrials.gov