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

NCT07697118 · tracked via the Priya Life Science France tracker

Sponsor

Assistance Publique - Hôpitaux de Paris

Phase

Phase 1/2

Started

2026-09

Last updated

2026-07-13

Condition(s) studied

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

Investigational drug(s) / intervention(s)

FOXP3-T4 drug productILT-101

FOXP3-T4 drug product: Each patient will receive a single IV infusion of FOXP3-T4, autologous gene-modified CD4+ T-transduced ex vivo with a self-inactivating lentiviral vector (LV-EF1a.FOXP3-LNGFR)

ILT-101: The first two patients included in the study will not receive IL-2 treatment. The others will receive the first injection the FOXP3-T4 treatment and if needed IL2-treatment. For IL2 treatment, patients will receive a daily dose for 5 days, followed by an administration per week for 3 months (ILT-101)

Study summary

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.

Eligibility

Sex

MALE

Min age

1 Year

Max age

45 Years

Healthy volunteers

Inclusion Criteria: * 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 Exclusion Criteria: * 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.

Primary outcome measure(s)

Frequency of clinical AEs and pathological variations of laboratory parameters — Up to 24 months post-infusion
Number of clinical AEs
Severity of clinical AEs and pathological variations of laboratory parameters — Up to 24 months post-infusion
Grading will be performed according to the CTCAE (Common Terminology Criteria for Adverse Events) current version. Severity is rated on a scale of Grade 1 (Mild) to Grade 5 (Death).
Incidence of clinically detectable lymphoproliferation and abnormal clonal dominance — Up to 24 months post-infusion
This is measured via Vector Insertion Site Analysis (VISA)
Incidence of clinically detectable lymphoproliferation and abnormal clonal dominance — Beyond 3 months to 24 months post-infusion
This is measured by proliferation of LNGFR+ cells
Detection of Replication-Competent Lentivirus (RCL) — Up to 24 months post-infusion
Persistence of recirculating LNGFR+among CD4+ T cells — at 3 months post-infusion
Percentage of LNGFR+ among CD4+ T cells

Trial sites (1)

Facility	City	Region	Status
Department of Biotherapy, Hopital Necker Enfants malades	Paris	Île-de-France Region

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07697118 on ClinicalTrials.gov ↗ ← All trials in France