CD4^LVFOXP3 in Participants With IPEX

Trial ID or NCT#

NCT05241444

Status

recruiting iconRECRUITING

Purpose

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 36 evaluable human participants with IPEX and evaluate the impact of the CD4^LVFOXP3 infusion on the disease.

Official Title

Phase 1 Study of Autologous CD4^LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome

Eligibility Criteria

Ages Eligible for Study: 4 Months to 35 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - 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:
  1. - 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 cardiac, renal, pulmonary, liver, or hematologic 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 All Criteria
Show Less

Investigator(s)

Maria Grazia Roncarolo
George D. Smith Professor of Stem Cell and Regenerative Medicine and Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Rosa Bacchetta
Shweta S. Namjoshi MD MPH
Clinical Associate Professor, Pediatrics - Gastroenterology
Orly R. Klein, MD
Rajni Agarwal

Contact us to find out if this trial is right for you.

Contact

SCGT Clinical Trials Program
650-723-6661

SEND MESSAGE

View on ClinicalTrials.gov