CD4^LVFOXP3 in Participants With IPEX
Recruiting
Phase 1
Interventional Study
IPEX
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 4 Months – 35
- Sex
- Male
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 30 (estimated)
- Sponsor
- Bacchetta, Rosa, MD · Other
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About This Trial
This first-in-human, Phase 1 clinical trial will test the feasibility of the manufacturing and the safety of the administration of CD4\^LVFOXP3 in up to 30 evaluable human participants with IPEX and evaluate the impact of the CD4\^LVFOXP3 infusion on the disease.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Body weight greater than 8 kg, unless assessed as able to tolerate leukapheresis
* FOXP3 gene mutation
* Medical history of progressive symptoms of IPEX with persistency of some symptoms and/or signs requiring immune suppressive medication. The participant may or may not be on…
Contacts