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:

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

Stanford is currently accepting patients for this trial.

Stanford Investigator(s):

Matthew Porteus

Intervention(s):

genetic: nula-cel Drug Product

Eligibility

Inclusion Criteria:

* ≥12 to ≤ 40 years
* Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures:
* recurrent severe VOC (≥ 4 episodes in the preceding 2 years)
* ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year)
* Lansky/Karnofsky performance status of ≥ 80

Exclusion Criteria:

* Available 10/10 HLA-matched sibling donor
* Prior HSCT or gene therapy
* Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder
* Clinically significant and active bacterial, viral, fungal or parasitic infection
* Pregnancy or breastfeeding in a postpartum female
* Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment

Ages Eligible for Study

12 Years - 40 Years

Genders Eligible for Study

ALL

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Elisabeth Merkel, RN
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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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