IPEX syndrome is a primary immunodeficiency caused by hemizygous mutations in the gene FOXP3, Patients affected by IPEX syndrome, proven by molecular diagnosis, which encodes an essential transcription factor required to maintain immunological tolerance by thymus-derived regulatory T (Treg) cells., with any active autoimmune complications or controlled under immunosuppressive therapy can be enrolled in this trial.
Conditions
Brief summary
The primary endpoint of the study is safety and efficacy up to 24 months following IV infusion of FOXP3-T4 alone or combined with low doses of IL-2 treatment.
Detailed description
The efficacy endpoint is assessed until 24 months following the infusion of the FOXP3-T4 treatment alone or combined with low-dose of IL-2 treatment
Interventions
DRUGFOXP3-T4
DRUGILT-101 liquide
Sponsors
Assistance Publique Hopitaux De Paris
Eligibility
Sex/Gender
Male
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint of the study is safety and efficacy up to 24 months following IV infusion of FOXP3-T4 alone or combined with low doses of IL-2 treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| The efficacy endpoint is assessed until 24 months following the infusion of the FOXP3-T4 treatment alone or combined with low-dose of IL-2 treatment | — |
Outcome results
None listed