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:

JSP191 Antibody Targeting Conditioning in SCID Patients

A Phase 1/2 study to evaluate the safety, tolerability, and efficacy of an antibody conditioning regimen, known as JSP191, in patients with Severe Combined Immune Deficiency undergoing blood stem cell transplantation

Stanford is currently accepting patients for this trial.

Intervention(s):

biological: Humanized anti-CD117 Monoclonal Antibody (JSP191)

Eligibility

Key Inclusion Criteria:

1. Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including but not limited to the following subtypes:

1. T-, B+, NK-: IL-2Rcγ deficient, JAK3-deficient (no longer enrolling)
2. T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
3. T-, B+, NK+: IL7Rα deficient, CD3 subunit deficient, CD45 deficient (no longer enrolling) OR Variant SCID with absent or low T cell function, Omenn syndrome, Leaky SCID, Reticular dysgenesis, Adenosine deaminase deficiency, and Purine nucleoside phosphorylase deficiency may be included after consultation with the medical monitor.
2. Patients with human leukocyte antigen (HLA) matched related or unrelated donors
3. Adequate end organ function as defined in study protocol
4. Age ≤ 12 years
5. Prior donor of appropriate age (≥ 5 years old) available for re-collection of stem cells
6. Previous allogeneic Hematopoietic Cell Transplantation HCT (≥ 6 months post initial transplant) with poor graft function

Key Exclusion Criteria:

1. Patients with any acute or uncontrolled infections
2. Patients receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy
3. Patients with active malignancies
4. Active GVHD within 6 months prior to enrollment, or on immunosuppressive therapy for GVHD

Ages Eligible for Study

3 Months - 12 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
650-721-0644
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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