Gene therapy for treating IPEX
Phase 1 Study of Autologous CD4^LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome
This study is testing a new gene therapy to see if it can help children and young adults with IPEX by improving their immune system and reducing their symptoms.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 4 Months to 35 Years |
| Sex | Male |
| Sponsor | Stanford University Academic / other |
| Locations | 1 site (Palo Alto, California) |
| Trial ID | NCT05241444 on ClinicalTrials.gov |
What this trial studies
This Phase 1 clinical trial aims to evaluate the safety and feasibility of administering CD4^LVFOXP3 to participants with IPEX, a rare autoimmune condition. The treatment is designed to replace defective regulatory T cells, potentially restoring immune system control and alleviating IPEX symptoms. The study will assess the quality of the manufactured CD4^LVFOXP3, its safety in children and young adults, and its effects on overall health and medication reduction. Up to 30 participants will be involved in this first-in-human trial.
Who should consider this trial
Good fit: Ideal candidates include children and young adults with a confirmed FOXP3 gene mutation and persistent symptoms of IPEX requiring immunosuppressive medication.
Not a fit: Patients with medical instability or those eligible for a matched blood stem cell transplant may not benefit from this treatment.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the health and quality of life for patients with IPEX by restoring immune function.
How similar studies have performed: While this approach is novel, similar gene therapy strategies have shown promise in other autoimmune conditions, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Palo Alto, California
- Lucile Packard Children's Hospital — Palo Alto, California, United States (Recruiting)
Study contacts
- Principal investigator: Jessie Alexander, MD — Stanford University
- Study coordinator: Rosa Bacchetta, MD
- Email: rosab@stanford.edu
- Phone: 650-498-8369
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.