RECRUITING

CD4^LVFOXP3 in Participants With IPEX

Conditions

IPEX Treg cells Regulatory T cells Gene therapy autoimmunity Immune Dysregulation Polyendocrinopathy Enteropathy X-linked FOXP3 Lentiviral CD271 NGFR immune suppression

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2022-03-22

Study Completion:2037-02

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05241444

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.

Ages Eligible for Study:4 Months to 35 Years

Sexes Eligible for Study:MALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion 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

Inclusion Criteria

Exclusion 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

Contacts and Locations

Study Contact

Rosa Bacchetta, MD

CONTACT

650-498-8369

rosab@stanford.edu

Principal Investigator

Jessie Alexander, MD

PRINCIPAL_INVESTIGATOR

Stanford University

Study Locations (Sites)

Lucile Packard Children's Hospital

Palo Alto, California, 94305

United States

Collaborators and Investigators

Sponsor: Bacchetta, Rosa, MD

Jessie Alexander, MD, PRINCIPAL_INVESTIGATOR, Stanford University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-03-22

Study Completion Date2037-02

Study Record Updates

Study Start Date2022-03-22

Study Completion Date2037-02

Terms related to this study

Keywords Provided by Researchers

Treg cells
Regulatory T cells
Gene therapy
autoimmunity
Immune Dysregulation Polyendocrinopathy Enteropathy X-linked
FOXP3
Lentiviral
CD271
NGFR
immune suppression

Additional Relevant MeSH Terms

IPEX