University of Miami Miller School of Medicine clinical investigators from its Diabetes Research Institute and Vertex Pharmaceuticals initiated a potentially path breaking clinical trial for an investigational cell therapy with the potential to restore normal glucose control in those suffering from type 1 diabetes (T1D) with severe hypoglycemia and impaired hypoglycemia awareness. The University of Miami Health System was the first trial site activated to test VX-880, developed originally from the work of a lab at Harvard University. With Fast Track Designation via the U.S. Food and Drug Administration—a program involving expedited review processes to accelerate treatments for urgent and unmet medical needs—the study evaluates the safety, tolerability and efficacy of this regenerative therapy-based infusion in certain T1D patients. The story of VX-880 started in the Harvard laboratory of Dr. Doug Melton and came to Vertex Pharmaceutical with the acquisition of Dr. Melton’s Semma Therapeutics.
TrialSite provides the network a brief breakdown of this important study.
An autoimmune disorder, T1D emerges as the body’s immune system attacks its own insulin-producing islet cells. Upon complete destruction of these cells, patients must take daily injections of insulin or use an insulin pump to replace the hormone. As one of the largest and most comprehensive research centers dedicated to curing diabetes, South Florida’s Diabetes Research Institute is at the forefront of investigating biological cures involving the restoration of natural insulin production and normalization of blood sugar levels without imposition of other risks.
The Lab of Dr. Doug Melton
The program is based on the groundbreaking studies of Dr. Doug Melton’s lab, who in their research developed this particular line of insulin-producing pancreatic cells derived from stem cells covered by TrialSite back in June, 2019. Dr. Melton, on a mission to find a cure for TD1, founded Semma Therapeutics which was acquired by Vertex the sponsor of the present study. Dr. Melton was confident that a cure for diabetes probably depends on the ability of one’s own body to make and deliver insulin.
TrialSite is a big fan of Dr. Melton and his lab work that led to this transformational investigational approach. Melton’s conviction, determination and mission to cure the disease reflects the true potential and spirit of research that TrialSite seeks to bring to everyone.
He has always been convinced nature shows us a way toward a cure for T1D. Each step that he’s taken along the preclinical research way has only reinforced his determination that the disease can be cured.
Fast Forward the Principal Investigator Today
Camillo Ricordi, MD, Professor of Surgery, also directs the Diabetes Research Institute and the Cell Transplant Center at the University of Miami Miller School of Medicine and happens to serve as the Chair for the Steering Committee as well as Principal Investigator for the VX-880 clinical trial, representing this Miami-based trial site. He shared with South Florida Hospital News: “It’s a remarkable time for T1D research efforts worldwide as this investigational treatment enters the clinic.” Dr. Ricordi went on to describe in a bit more detail why this study has some momentum, declaring, “The field’s experience with the limited cadaveric islet transplants available, where some patients have experienced prolonged insulin independence for years, provides important proof-of-concept for the potential of cell therapy to be transformative for patients living with T1D.”
This Phase 1/2 study (NCT04786262) just recently started with the first trial site opening up at the University of Miami Health System and a few others, including University of Pennsylvania and Massachusetts General Hospital to follow.
The study team is evaluating the safety, tolerability and efficacy of VX-880 infusion in participants in T1D and impaired awareness of hypoglycemia (IAH) and severe hypoglycemia.
Participation in this study necessitates that the participant have a clinical history of T1D of at least five years. Additionally, they must be between the ages of 18 and 65 years of age and have experienced multiple episodes of severe hypoglycemia and impaired hypoglycemia awareness 12 months prior to enrollment.
The study includes two primary endpoints and two secondary endpoints including the following:
|Primary Outcome Measures||Secondary Outcome Measures|
|Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From VX-880 infusion to end of study (up to 5 years) ]||Proportion of Participants who are Insulin Independent [ Time Frame: At 1 year after VX-880 infusion ]|
|Proportion of Participants Free of Severe Hypoglycemic Events With Either a Glycosylated Hemoglobin (HbA1c) <7.0% or a ≥ 1% Reduction in HbA1c From Baseline [ Time Frame: At 1 year after VX-880 infusion||Changes in Stimulated C-peptide [ Time Frame: At 1 year after VX-880 infusion ]|
The First Trial Site
The Diabetes Research Institute in Miami represents the first trial site to treat patients in what Dr. Ricordi declared is an “…unprecedented, first-in-human clinical trial.” He emphasized for South Florida Hospital News that this effort represents all that the DRI and “…our entire translational and clinical efforts,” including “the cGMP Advanced Cell and Biologic Product Manufacturing Facility.”
About Diabetes Research Institute
The Diabetes Research Institute Foundation’s mission centers on the appropriate funding of the Diabetes Research Institute (DRI) to cure diabetes now. DRI at the University of Miami Miller School of Medicine leads the world in cure-focused research. As one of the largest and most comprehensive research centers dedicated to curing diabetes, DRI is aggressively working to develop a biological cure by restoring natural insulin production and normalizing blood sugar levels without imposing other risks.
Here, researchers have already demonstrated that transplanted islet cells allow patients to live without the need for insulin therapy. Some study participants have maintained insulin independence for over a decade. The DRI now builds upon these promising outcomes through its BioHub Strategy, a multidisciplinary, three-pronged approach for addressing the major challenges that stand in the way of a cure, including 1) elimination of the need for any-rejection drugs; 2) reset the immune system to block autoimmunity ;and 3) develop an unlimited supply of insulin-producing cells.
The DRI Team
Dr. Ricordi also gave thanks to team members for this important investigational undertaking including:
cGMP Advanced Cell and Biologic Product Manufacturing Facility
∙ Xiaojing Wang MD, Associate Scientist
∙ Clarissa Lanero
∙ The CCTP Clinical Cell Transplant Program
∙ Kemraj Hirani, MPharm, PhD, RPh, CCRP, CIP, RAC, MBA
∙ Luis Roque, Quality Assurance Auditor
∙ Burlett Masters, Research Support Specialist
Interventional Radiologist (infuses the cells in the subjects)
∙ Prasoon P. Mohan, MD, Associate Professor- Clinical Interventional Radiology Program director for the Interventional Radiology Residency and Fellowship
Camillo Ricordi, MD, Professor of Surgery also directs the Diabetes Research Institute and the Cell Transplant Center at the University of Miami Miller School of Medicine
Call to Action: This study’s estimated primary completion date isn’t till January 2024 but TrialSite will monitor the study for updates. If you have T1D and want personalized updates, register a profile and request a T1D group in TrialSite Patient Centers.