CD4^LVFOXP3 in Participants With IPEX

NCTID	NCT05241444 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome (IPEX)
Disease Ontology Term	DOID:0090110
Compound Name	CD4^LVFOXP3
Sponsor	Bacchetta, Rosa, MD
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	30
Results Posted	Not Available

Target Gene/Variant	FOXP3
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD4+ T cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type	none
Dose 1	1.0E6 CD4+ cells/kg
Dose 2	3.0E6 CD4+ cells/kg
Dose 3	1.0E7 CD4+ cells/kg
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States