Advancing Regenerative Medicine

Stanford's Alpha Clinic supports the advancement of regenerative therapies in the following ways

Real World Consortium

We are pursuing real-world outcomes data in the post-CAR pediatric and adult settings.

Collaborate with the Alpha Clinic Network to develop a charter
Offer consultation on regulatory and IRB protocol best practices
Consult on development of tools and databases for sample tracking and real-world outcomes data analysis
Provide guidance on multi-site data sharing template agreements and standards

Expand Toxicity Guidelines and Safety

We are working with the Alpha Clinic network to build a California Cell Therapy Safety Monitoring Committee (CCTSMC)

Safety guidance for IITs conducted through the CIRM CLIN2 award mechanism and designated as “high-risk”. This includes first-in-human testing, first-in-child testing and therapy using genetically manipulated cells.
Develop best practices and guidelines for toxicity grading and management.
Offer consultation and expertise on safety-related issues, including serious adverse events (SAEs), subject deviations, internal and external audit/monitoring reports, dose escalation decisions based on available AEs and dose limiting toxicity (DLT) determinations, and study halt/termination based on protocol-specified safety rules.

Maintain a Minimum Number of Clinical trials

Current Cell and Gene Therapy Clinical Trial List:

CD4^LVFOXP3 in Participants With IPEX

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.

Stanford is currently accepting patients for this trial.

Stanford Investigator(s):

Intervention(s):

biological: CD4^LVFOXP3

Eligibility

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 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 - 35 Years

Genders Eligible for Study

MALE

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
SCGT Clinical Trials Program
650-723-6661
I'm interested

Link to All Stanford Clinical Trials

Stanford Affiliates

The Alpha Clinic Network

We are part of a state-wide network funded by the California Institute for Regenerative Medicine

This network includes:

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