Gene therapy aims to tame rare immune disorder
NCT ID NCT07697118
First seen Jul 13, 2026 · Last updated Jul 14, 2026 · Updated 1 time
Summary
This trial tests a new gene therapy called FOXP3-T4 for people with IPEX syndrome, a rare genetic condition that causes severe immune problems. The therapy involves taking a patient's own immune cells, modifying them in the lab to work better, and giving them back via a single infusion. Some participants may also receive low-dose IL-2 to boost the therapy. The goal is to stabilize symptoms and reduce the need for lifelong immune-suppressing drugs.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- FOXP3-T4 (gene-modified immune cells) and low-dose IL-2
- What this could lead to
- If successful, this could provide a long-term treatment option for IPEX syndrome, potentially allowing patients to reduce or stop immunosuppressive drugs.
- What could go wrong
- This is an early-phase trial with only 5 participants, so results may not apply broadly. Risks include immune reactions, lymphoproliferation, or the therapy not working as expected.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Department of Biotherapy, Hopital Necker Enfants malades
Paris, Île-de-France Region, 75015, France
Contact Phone: •••-•••-•••• Email: •••••@•••••