The purpose of this study is to evaluate the safety and efficacy of FOXP3-T4 (an autologous gene therapy) alone or in combination with low-dose IL-2 for the treatment of IPEX syndrome. The therapy involves the transplantation of autologous CD4+ T-cells transduced ex vivo with the LV-EF1a-FOXP3-LNGFR lentiviral vector. The study follows a staggered approach: the first two patients will receive FOXP3-T4 monotherapy. Subsequent patients will receive FOXP3-T4 followed if needed by low-dose IL-2 treatment consisting of a daily dose for 5 days, then weekly administrations for 3 months.
The study aims to stabilize autoimmune manifestations and potentially cure the underlying disease, ultimately allowing for the discontinuation of ongoing immunosuppressive treatments.
Male patients only
Patients aged from 1 - 45 years of age (the first three patients will be aged between 10 - 45 years of age)
Patient with IPEX syndrome caused by mutation of the FOXP3 gene
Patients are eligible from the second line of treatment onward, even those under controlled disease
Unwillingness to return for follow-up during the 2-year study and during the 15 years of long term follow up study.
Patient with short life expectancy
Patient on AME (state medical aid) (unless exemption from affiliation).
Diagnosis of a significant psychiatric disorder of the patient that could seriously impede the ability to participate in the study.
Assistance Publique - Hôpitaux de Paris
Lead sponsor
URC-CIC Paris Descartes Necker Cochin
Collaborator