Our Mission

The Western States Sickle Cell Disease Clinical Trials Network (WestSCDCTN)
Organizational Structure of the WeST SCD CTN

The Western States Sickle Cell Disease Clinical Trials Network (WeST SCD CTN) is a Clinical Trials Unit (CTU) in the national American Society of Hematology (ASH) Research Collaborative (RC) Sickle Cell Disease Clinical Trials Network, one of the first 10 sites who will be enrolling children and adults living with sickle cell disease as part of an unprecedented national effort to streamline operations and facilitate data sharing to expedite the development of new treatments for those living with the disease. Each of the 10 Network consortia is a "hub and spoke" model, consisting of a CTU that serves as the lead clinical trial site, or hub, providing support to its various clinical research sites (CRS), or spokes, within each consortium. The Network currently represents approximately 24,000 individuals living with sickle cell disease.

The CTU of the WeSt SCD CTN is the Northern California Comprehensive Sickle Cell Center (NCCSCC), which for 40 years has been an NIH-funded, multidisciplinary center partnered with the sickle cell community to improve treatment through clinical research trials. The NCCSCC will provide leadership and the scientific, administrative, and infrastructure support to the WeSt SCD CTN.

We have participated in multi-center trials for years and have formed a regional network that is synergistic with the goals of expanding clinical trials sites, increasing efficiencies and coordination, cost savings, and increasing access to research and effective therapies to patients with sickle cell disease (SCD). We have a robust network of community-based organizations (CBOs) engaged with our network and supporting the research collaboration.

History of the NCCSCC

The NCCSCC at UCSF Benioff Children's Hospital Oakland (BHCO) has decades of success in leading multisite clinical trials in SCD and thalassemia. Historically, our research focus has included: (1) novel therapeutics; (2) early detection and treatment of complications; (3) psychobiologic effects of SCD; (4) red cell membrane and vasculopathy; (5) lung injury; (6) cellular therapies; (7) arginine/nitric oxide dysregulation; (8) genetic modifiers of complications; (9) oxidative stress and antioxidant therapy; and (10) development of biomarkers. We also have a large, stable patient population who are motivated to participate in research.

Over 7 years ago, we developed a network of sickle cell programs throughout the western states to improve access to new therapies and in 2013, were funded along with the Center for Inherited Blood Disorders by the HRSA Sickle Cell Disease Treatment Demonstration Program. This grant supports the Pacific Sickle Cell Regional Collaborative (PSCRC) (Appendix 1) with its core partners within the Western United States. The PSCRC has developed and implemented strategies to expand access and improve care for patients with SCD. Core members of the PSCRC have partnered with the Agency for Health Research and Quality (AHRQ) to monitor improvements in quality care. Centers from this collaborative network have demonstrated successful standardized quality data collection and meaningful engagement of community advocacy groups and some have been chosen to be clinical research sites (CRSs).

In 2016, the NCCSCC and its northern California partners were funded by the NHLBI to join the Sickle Cell Care Coordination Initiative (SCCCI). This collaboration of experts in SCD clinical trials, implementation scientists, engaged stakeholders (CBOs, primary care, Medicare/Medicaid managed care plans, community clinics, and patient/family representatives) are working together to address individual, organizational, and systemic barriers to sickle cell care and clinical research prior to implementing clinical trials within this national consortium. The NHLBI launched the CURE Sickle Cell Initiative to speed up movement of the most promising curative therapies into clinical trials. Cellular therapy should be considered by families with other clinical trials initiated by the ASH RC network. Therefore, the WeSt SCD CTN has developed a working partnership with California Institute of Regenerative Medicine (CIRM) (Appendix 3) to accelerate the development of cell and gene therapy treatments. We are proposing a novel approach - including the CIRM Alpha Cell Stem Cell Clinic Programs as a virtual protocol-specific site (PS) within the WeSt SCD CTN.

Research Experience

During the past 5 years, we have been a Principal or Co-Investigator in the majority of SCD Phase I-III intervention trials. A detailed analysis of 36 recent interventional trials and the 457 patients involving 7 Phase I, 21 Phase II, and 8 Phase III trials are in Appendix 4. These studies include trials of glutamine, NKTT120, polaxamer, Selg1, regadenoson, hydroxyurea, GBT440, transfusion, Mirasol-treated whole blood, SC411 (DHA), GM1-1070, IMR687, arginine, prasugrel, simvastatin, ketamine, encephaloduroarteriosynangiosis for moyamoya, crizanlizumab, vitamin B6, vaporized cannabis, and iron chelators. We have also been investigators in many stem cell cellular therapy trials -- including adult severe sickle cell disease, gene therapy with Lentiglobin 3305, the Cordin ex vivo stem cell, and the adult haplo transplant trial.