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Gene Therapy Trial Report

Summary

CD4^LVFOXP3 in Participants With IPEX


NCTID NCT05241444 (View at clinicaltrials.gov)
Description
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
Enrollment Count 30 (ESTIMATED)
Results Posted Not Available

Therapy Information


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

Study Record Dates


Current Stage Phase1
Submit Date 2022-01-12
Completion Date 2037-02
Last Update 2025-05-22

Participation Criteria


Eligible Age 4 Months - 35 Years
Standard Ages Child, Adult
Sexes Eligible for Study MALE
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 immunosuppression at time of starting the study. * Uncontrolled IPEX disease but unable to tolerate immune suppressive medication * Recurrent IPEX symptoms, requiring immune suppressive medications, in participants who have had prior allogeneic (allo) blood stem cell transplantation (HSCT). * ≥ 50% Performance rating on Lansky/Karnofsky Scale * Organ and marrow function within acceptable levels of function * Absence of ongoing infections * Must be able to consent if an adult Exclusion Criteria: * Medical instability * Less than 6 months life expectancy * Inability to meet limits for steroid dosing * Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant, and be willing to undergo transplant. * Unrelated or comorbid disease * Allergy to any study medication, product, or intervention * Currently receiving another experimental treatment * History of malignancy, unless disease free for at least 2 years, with the exception of non melanoma skin cancer or carcinoma in situ
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates

Resources/Links