Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T … (NCT07697118) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.
France5 participantsStarted 2026-09
Plain-language 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.
Who can participate
Age range
1 Year – 45 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 w…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Frequency of clinical AEs and pathological variations of laboratory parameters
Timeframe: Up to 24 months post-infusion
2
Severity of clinical AEs and pathological variations of laboratory parameters
Timeframe: Up to 24 months post-infusion
3
Incidence of clinically detectable lymphoproliferation and abnormal clonal dominance
Timeframe: Up to 24 months post-infusion
4
Incidence of clinically detectable lymphoproliferation and abnormal clonal dominance
Timeframe: Beyond 3 months to 24 months post-infusion
5
Detection of Replication-Competent Lentivirus (RCL)
Timeframe: Up to 24 months post-infusion
6
Persistence of recirculating LNGFR+among CD4+ T cells