Curing Diabetes

Established Type 1 Diabetes

Pancreatic Islet Transplantation into the Anterior Chamber of the Eye

Clinical trial is currently recruiting legally blind patients with type 1 diabetes to evaluate the effects of transplanting pancreatic islets into the eye’s anterior chamber to potentially reduce insulin requirements and stabilize blood glucose levels.

Status: RECRUITING*

Study Type: Interventional

Population: Legally blind patients with T1D

  • 18-70 years of age
  • onset of T1D at <40 years of age
  • insulin-dependence for >5 years at the time of enrollment
  • reduced awareness of hypoglycemia

Intervention: A single dose of islets will be infused into the anterior chamber of the eye through a self-sealing incision of the peripheral cornea. The procedure is projected to take approximately 20-30 minutes.

Comparison: Single group assignment study. There is no comparator treatment.

Outcome: Reduction of glucose variability; reduction of total insulin requirements; increase of basal C-peptide; HbA1C levels

Timing: The endpoints will be assessed at six months post transplantation

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT02846571

Contact: Call 305-243-5321 or email islet@med.miami.edu

Sponsor and Principal Investigator: Midhat H. Abdulreda PhD, University of Miami

Collaborators: Diabetes Research Institute Foundation and Bascom Palmer Eye Institute

*Status as of April 15, 2024, on https://clinicaltrials.gov.

A Safety, Tolerability, and Efficacy Study of VX-880 in Participants with T1D

This interventional study, currently recruiting participants with type 1 diabetes who have impaired awareness of hypoglycemia and a history of severe hypoglycemia, is exploring the safety and efficacy of VX-880. Administered through infusion into the hepatic portal vein, this single-group study aims to evaluate the tolerability and potential reduction in severe hypoglycemic events over a period of up to five years, monitoring participants for adverse effects and improvements in glucose stability.

Status: RECRUITING*

Study Type: Interventional

Population: T1D with impaired awareness of hypoglycemia and severe hypoglycemia

  • 18 – 65 years of age
  • T1D diagnosis for > 5 years
  • ≥ two episodes of documented severe hypoglycemia in the past 12 months
  • Stable treatment for T1D
  • Consistent use of continuous glucose monitor for ≥3 months before screening and willingness to use it for the duration of the study

Intervention: VX-880 infused into the hepatic portal vein

Comparison: None – this is a single group study

Outcome: This study will assess the safety and tolerability as assessed by adverse events and the proportion of participants free of severe hypoglycemic events

Timing: The study will run for up to 5 years

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT04786262

Contact: Call 305-243-5321 or email islet@med.miami.edu

Sponsor: Vertex Pharmaceuticals Incorporated

*Status as of April 15, 2024, on https://clinicaltrials.gov.

A Safety, Tolerability, and Efficacy Study of VX-264 in Participants with T1D

This interventional study is actively recruiting participants aged 18 to 65 with type 1 diabetes to evaluate the safety, tolerability, and efficacy of VX-264, involving the transplantation of allogeneic human stem cell-derived islets into a device. As a single group study, it aims to monitor adverse events and assess changes in pancreatic function.

Status: RECRUITING*

Study Type: Interventional

Population: T1D

  • 18 – 65 years of age
  • T1D diagnosis for ≥ 5 years
  • Stable treatment for T1D
  • Consistent use of continuous glucose monitor for ≥4 weeks before screening and willingness to use it for the duration of the study

Intervention: VX-264 (transplantation of allogeneic human stem cell-derived islets)

Comparison: None – this is a single group study

Outcome: The primary outcome measures are safety and tolerability as assessed by the number of adverse events up to 24 months and change in peak C-peptide during mixed-meal tolerance tests at baseline and Day 90.

Timing: Up to 24 months

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT05791201

Contact: Call 305-243-5321 or email islet@med.miami.edu

Sponsor: Vertex Pharmaceuticals Incorporated

*Status as of April 15, 2024, on https://clinicaltrials.gov.

Long Term Follow up of Recipients of Functional Islet Allografts

Ongoing study with patients with type 1 diabetes who previously received islet transplants at the University of Miami, aiming to monitor long-term safety and effectiveness of the transplants through sustained islet function assessments.

Status: RECRUITING*

Study Type: Observational

Population: Patients with T1D

  • 18-65 years of age
  • received at least one islet transplant at the Diabetes Research Institute at the University of Miami, Miller School of Medicine on the following protocols: 2000/0329; 2000/0196; 2004/0205; 2000/024; 2006/0200; 2006/0508; 2006/0210.

Comparison: There is no comparator treatment

Outcome: The purpose of this study is to collect additional follow-up safety and efficacy information from subjects with graft function after completion in the original study. The primary endpoint is duration of sustained islet allograft function as determined by evidence from mixed meal tolerance tests of C-peptide production.

Timing: The endpoints will be assessed over three years

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT01999374

Contact: Call 305-243-5321 or email islet@med.miami.edu

Sponsor and Principal Investigator: Rodolfo Alejandro MD, University of Miami

*Status as of April 15, 2024, on https://clinicaltrials.gov.

Long Term Surveillance of Islet Transplant Recipients Following Complete Graft Loss

Ongoing study with patients with type 1 diabetes who experienced complete islet graft loss after transplantation at the University of Miami. The study aims to monitor the presence and changes in panel reactive antibodies for up to ten years after discontinuing immunosuppression therapy.

Status: RECRUITING*

Study Type: Observational

Population: Patients with T1D

  • 18-75 years of age
  • received at least one islet transplant (in the absence of any other organ transplant) at the Diabetes Research Institute at the University of Miami, Miller School of Medicine

Comparison: There is no comparator treatment

Outcome: After complete islet graft loss is determined, patient’s maintenance immunosuppression will be discontinued, and they will be monitored for antibodies (panel reactive antibodies [PRA]). The primary objective is to determine the rate of presence of PRA in patients 3 years after failed islet transplantation and monitor the persistence of elevated PRA levels at years 3, 6, and 9. Timing, frequency, and level of change in PRA will be monitored after all immunosuppression is discontinued.

Timing: The participants will be monitored for 10 years

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT02000687

Contact: Call 305-243-5321 or email islet@med.miami.edu

Sponsor and Principal Investigator: Rodolfo Alejandro MD, University of Miami

*Status as of April 15, 2024, on https://clinicaltrials.gov.

Prevention of de Novo Allosensitization in Islet Transplant Recipients Following Complete Graft Loss

Study for patients with type 1 diabetes who have experienced complete loss of islet grafts after transplantation at the University of Miami. This trial tests whether Myfortic® monotherapy for two years can prevent the development of panel reactive antibodies (PRA) following the discontinuation of regular maintenance immunosuppression, with follow-up monitoring over three years.

Status: RECRUITING*

Study Type: Interventional

Population: Patients with T1D

  • 18-70 years of age
  • received at least one islet transplant at the Diabetes Research Institute at the University of Miami, Miller School of Medicine.

Intervention: After complete islet graft loss is determined, maintenance immunosuppression will be discontinued and participants will be treated with Myfortic® monotherapy for two years.

Comparison: There is no comparator treatment

Outcome: The primary objective is to determine the presence of panel reactive antibodies (PRA) in patients after Myfortic® monotherapy is discontinued

Timing: The participants will be monitored for three years

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT01999361

Contact: Call 305-243-5321 or email islet@med.miami.edu

Sponsor and Principal Investigator: Rodolfo Alejandro MD, University of Miami

*Status as of April 15, 2024, on https://clinicaltrials.gov.

Diabetes Prevention Program Outcomes Study (DPPOS)

The Diabetes Prevention Program Outcomes Study (DPPOS) is an ongoing, long-term interventional study that follows participants from the original Diabetes Prevention Program, which demonstrated that lifestyle changes and metformin can delay the onset of type 2 diabetes in overweight or obese American adults at high risk.

Status: ACTIVE BUT NOT RECRUITING*  

Study Type: Interventional

Population: Diverse population of overweight or obese American adults at high risk of T2D

  • over 25 years of age
  • Participation as a volunteer in the Diabetes Prevention Program (DPP)

Intervention: The original DPP study showed that lifestyle changes or metformin could effectively delay the development of T2D in a diverse population of overweight or obese American adults at high risk of T2D in the short term and long term.

Comparison: Randomized to masked placebo during DPP and offered Intensive Lifestyle Group Session and DPPOS Group Lifestyle in DPPOS.

Outcome: The goal of DPPOS was to study whether the relatively short-term benefits of delaying T2D demonstrated in the DPP would translate into long-lasting impact. DPPOS had the following major goals, to determine the effect of DPP interventions on: 1) durability of T2D development; 2) early microvascular disease; and 3) atherosclerosis and CVD risk factors.

Timing:  Up to 29 years

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT00038727

Study Chair: David M. Nathan MD, Massachusetts General Hospital

Contact: call 301-881-9260 or email dppmail@bsc.gwu.edu

Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases

*Status as of April 15, 2024, on https://clinicaltrials.gov.

A Study to Investigate the Efficacy and Safety of Finerenone Versus Placebo, in Addition to Standard of Care, in People with Chronic Kidney Disease And T1D (FINE-ONE)

The FINE-ONE study is actively recruiting individuals with type 1 diabetes and chronic kidney disease (CKD) to evaluate the efficacy and safety of finerenone, a mineralocorticoid receptor antagonist already approved for use in CKD with type 2 diabetes. This interventional trial will compare finerenone to a placebo, both administered in addition to standard of care, focusing on changes in kidney disease progression.

Status: RECRUITING*  

Study Type: Interventional

Population: T1D and a clinical diagnosis of CKD

  • ≥18 years of age
  • HbA1c at Screening <10%
  • K+ ≤ 4.8 mmol/L at Screening
  • Stable angiotensin-converting enzyme inhibitor or angiotensin receptor blocker treatment.

Intervention: The study treatment finerenone works by blocking mineralocorticoid receptors. An increased stimulation of these proteins is thought to damage the kidneys and the heart. By lowering their stimulation, finerenone reduces the risk of kidney disease progressively getting worse. Finerenone is approved for doctors to prescribe to people with CKD and T2D.

In this study, researchers want to learn if finerenone works better than placebo in reducing the participants’ kidney disease from getting worse when given in addition to standard of care treatment.

Comparison: Placebo plus standard of care treatment

Outcome: The primary outcome measure will be change in urinary albumin-to-creatinine ratio, to investigate the effect of finerenone on kidney disease. Safety will also be assessed throughout the study by the number of AE.

Timing:  Up to 7.5 months

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT05901831

Contact: Call 305-243-4485 or email cblaschke@miami.edu

Sponsor: Bayer

*Status as of April 15, 2024, on https://clinicaltrials.gov.

Phase 2b Trial Comparing HDV-Insulin Lispro to Insulin Lispro in Adults with T1D Receiving Insulin Degludec (OPTI-2)

The OPTI-2 trial is currently recruiting adults with type 1 diabetes to compare the effects of Hepatocyte-Directed Vesicle-insulin lispro (HDV-L) with regular insulin lispro when used alongside insulin degludec as basal insulin. This Phase 2b interventional study aims to explore whether targeting insulin delivery directly to the liver can enhance glycemic control by reducing nocturnal hypoglycemia without compromising HbA1c levels. Participants will also be required to use a continuous glucose monitor (CGM) throughout the 7-month study period.

Status: RECRUITING*

Study Type: Interventional

Population: T1D

  • ≥18 years to 79 years of age
  • C-peptide <0.6 nmol/L
  • Using insulin for ≥ 6 months
  • willing to use study-provided insulin as the only bolus insulin and insulin degludec as the basal insulin
  • willing to use CGM device throughout the study
  • screening HbA1c ≥ 6.5% and ≤ 0%
  • daily insulin dose ≤25 U/kg/day

Intervention: The goal of this study is to see whether directing insulin to the liver using Hepatocyte-Directed Vesicles-insulin lispro (HDV-L) will improve the low blood sugar that sometimes happens when injecting insulin in patients with T1D.

Comparison: Lispro alone

Outcome: The primary objective of the study is to determine if the addition of HDV to insulin lispro results in an improvement in glycemic control as defined by decreasing evidence of nocturnal hypoglycemia while maintaining or improving HbA1c. Other outcomes related to glycemic control will also be evaluated.

Timing:  Up to 7 months

Additional information and inclusion criteria: https://clinicaltrials.gov/study/NCT06238778

Contact: Call 305-243-4485 or email cblaschke@miami.edu

Sponsor: Diasome Pharmaceuticals, Inc.

*Status as of April 15, 2024, on https://clinicaltrials.gov.

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