BridgeMD
THERIPEX
EU CTIS ID: 2025-523305-15-00
What this study is testing
The primary objective of this clinical trial is to assess the initial safety and efficacy of treatment with FOXP3-T4 alone or combined with low-dose IL-2 treatment, including the procedure and infusion with LV-FOXP3-LNGFR lentiviral vector gene-modified induced CD4+ Treg-cells in IPEX patients.
- Human Pharmacology (Phase I)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1) Male patients only 2) Patients aged from 1-45 years of age. The first three patients will be aged between 10 – 45 years of age.
- 3) Patient with IPEX syndrome caused by mutation of the FOXP3 gene
- 4) Patients are eligible from the second line of treatment onward, even those under controlled disease
- 5) Patient with recurrent IPEX symptoms, under immune suppressive medications
- 6)Patient for whom HSCT is not feasible or when no suitable compatible donor is available 7)Patients who have had prior allogeneic blood stem cell transplantation (HSCT) with engraftment failure defined as no intake of donor cells
- 8) Parental, guardian’s patient signed informed consent
You likely can't join if
- 1) Unwillingness to return for follow-up during the 2-year study and during the 15 years of long-term follow-up study
- 3) Patient on AME (state medical aid) (unless exemption from affiliation)
- 4) Diagnosis of a significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
- 4) Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant and be willing to undergo transplant
- 7) HIV-1 or 2 or HTLV1 infections
- 8) Patients with severe IPEX clinical presentation needing a rapid allogeneic HSCT treatment within 3 months
See the full eligibility criteria
- 1) Male patients only 2) Patients aged from 1-45 years of age. The first three patients will be aged between 10 – 45 years of age.
- 3) Patient with IPEX syndrome caused by mutation of the FOXP3 gene
- 4) Patients are eligible from the second line of treatment onward, even those under controlled disease
- 5) Patient with recurrent IPEX symptoms, under immune suppressive medications
- 6)Patient for whom HSCT is not feasible or when no suitable compatible donor is available 7)Patients who have had prior allogeneic blood stem cell transplantation (HSCT) with engraftment failure defined as no intake of donor cells
- 8) Parental, guardian’s patient signed informed consent
- 9) For patients of childbearing age: willing to use an effective method of contraception during the trial and for at least 12 months post-infusion
- 10) Affiliation to a French or European social security scheme
- 1) Unwillingness to return for follow-up during the 2-year study and during the 15 years of long-term follow-up study
- 3) Patient on AME (state medical aid) (unless exemption from affiliation)
- 4) Diagnosis of a significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
- 4) Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant and be willing to undergo transplant
- 7) HIV-1 or 2 or HTLV1 infections
- 8) Patients with severe IPEX clinical presentation needing a rapid allogeneic HSCT treatment within 3 months
- 9) hypersensitivity to IL-2 or any component of the formulation
- 6) Patients with uncontrolled or ongoing active infections
- 2) Patient with short life expectancy
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.