CD4^LVFOXP3 in Participants With IPEX

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.

Conditions

IPEX

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Study Overview

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Study Details

Study overview

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.

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

CD4^LVFOXP3 in Participants With IPEX

Condition
IPEX
Intervention / Treatment

-

Contacts and Locations

Palo Alto

Lucile Packard Children's Hospital, Palo Alto, California, United States, 94305

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

4 Months to 35 Years

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Bacchetta, Rosa, MD,

Jessie Alexander, MD, PRINCIPAL_INVESTIGATOR, Stanford University

Study Record Dates

2037-02