Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.

Starting soon · Phase 1/Phase 2

Conditions studied: The Immune Dysregulation Polyendocrinopathy Enteropathy X-linked Syndrome is a Primary Immunodeficiency Caused by Pathogenic Variants in Forkhead Box Protein 3

In brief

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.

Key facts

Study ID

NCT07697118

Run by

Assistance Publique - Hôpitaux de Paris

People needed

5

Starts

2026-09-01

Expected to finish

2029-01-01

Last updated by the study team

2026-07-13

Who can join

Age: 1 and older, up to 45. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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
• Patient with recurrent IPEX symptoms, under immune suppressive medications
• Patient for whom HSCT is not feasible or when no suitable compatible donor is available
• Patients who have had prior allogeneic blood stem cell transplantation (HSCT) with engraftment failure defined as no intake of donor cells
• Patient or parental, guardian's patient signed informed consent
• Male participants of reproductive potential with a partner of childbearing potential (WOCBP): willing to use an effective method of contraception during the trial and for at least 12 months post-infusion
• Affiliation to a French or European social security scheme

You may not qualify if…

• 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.
• Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant (HLA 10/10) and be willing to undergo transplant.
• Patients with uncontrolled or ongoing active infections.
• HIV-1 or 2 or HTLV-1 infections.
• Patients with severe IPEX clinical presentation needing a rapid allogeneic HSCT treatment within 3 months.
• Patients with known history of hypersensitivity to IL-2 or any component of the formulation (mannitol, sodium lauryl sulfate, monosodium phosphate dehydrate, disodium phosphate dehydrate, glucose.

Where it is running

• Department of Biotherapy, Hopital Necker Enfants malades — Paris, Île-de-France Region, France

Full record on ClinicalTrials.gov

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