Actively Recruiting

Phase 1
Age: 4Months - 35Years
MALE
NCT05241444

CD4^LVFOXP3 in Participants With IPEX

Led by Bacchetta, Rosa, MD · Updated on 2025-05-22

30

Participants Needed

1

Research Sites

775 weeks

Total Duration

On this page

Sponsors

B

Bacchetta, Rosa, MD

Lead Sponsor

C

California Institute for Regenerative Medicine (CIRM)

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

Official Title

CD4^LVFOXP3 in Participants With IPEX

Who Can Participate

Age: 4Months - 35Years
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Body weight greater than 8 kg, unless able to tolerate leukapheresis
  • FOXP3 gene mutation
  • History of progressive IPEX symptoms requiring immune suppressive medication, with or without current immunosuppression
  • Uncontrolled IPEX disease but unable to tolerate immune suppressive medication
  • Recurrent IPEX symptoms requiring immune suppressive medications after prior allogeneic blood stem cell transplantation
  • Performance rating of at least 50% on Lansky/Karnofsky Scale
  • Organ and marrow function within acceptable limits
  • No ongoing infections
  • Ability to consent if an adult
Not Eligible

You will not qualify if you...

  • Medical instability
  • Life expectancy less than 6 months
  • Inability to meet steroid dosing limits
  • Eligible and willing for HLA matched sibling or unrelated donor blood stem cell transplant
  • Unrelated or comorbid diseases
  • Allergy to any study medication, product, or intervention
  • Currently receiving another experimental treatment
  • History of malignancy unless disease-free for at least 2 years, except non-melanoma skin cancer or carcinoma in situ

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Lucile Packard Children's Hospital

Palo Alto, California, United States, 94305

Actively Recruiting

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Research Team

R

Rosa Bacchetta, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

2

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