type 1 diabetes mellitus
Diabetes clinical trials span both type 1 (autoimmune beta-cell destruction) and type 2 (insulin resistance) disease, with growing focus on the overlapping spectrum including latent autoimmune diabetes in adults (LADA) and monogenic forms. Research priorities include beta-cell preservation in newly diagnosed type 1 patients, weight-loss-independent glucose control, and prevention of cardiovascular and renal complications.
Current trials evaluate GLP-1/GIP dual agonists (tirzepatide analogs), oral insulin formulations, closed-loop artificial pancreas systems, immunotherapy for type 1 delay, SGLT2 inhibitors in non-diabetic heart failure, and stem-cell-derived islet transplantation. Lifestyle intervention trials also remain an active category.
Many trials separate type 1 and type 2 cohorts; baseline HbA1c, duration of disease, and eGFR are common eligibility criteria.
Disease Burden & Epidemiology
Diabetes mellitus affects approximately 537 million adults worldwide as of 2021 — roughly 1 in 10 adults globally — and projections from the International Diabetes Federation estimate 783 million cases by 2045 if current trends continue. Type 2 diabetes accounts for 90–95% of cases and is strongly linked to obesity, physical inactivity, and aging populations. Type 1 diabetes, an autoimmune condition typically diagnosed in childhood and adolescence, affects approximately 8.4 million people globally, with incidence rising 2–3% annually in high-income countries for reasons that remain incompletely understood. Diabetes is the leading cause of blindness, kidney failure requiring dialysis, and non-traumatic lower-limb amputation in adults in most developed countries. Cardiovascular disease kills approximately half of all people with diabetes. Economic costs are staggering: the International Diabetes Federation estimated global diabetes-related health expenditure at $966 billion in 2021. The rise of GLP-1 receptor agonists and SGLT2 inhibitors — both validated through landmark randomized controlled trials — has fundamentally changed the metabolic disease treatment paradigm and created an active second-generation drug development pipeline.
Key Research Trends & Landmark Studies
The UKPDS trial, conducted over more than 20 years, established intensive glycemic control as beneficial for microvascular complications in type 2 diabetes and remains a landmark in the field. The EMPA-REG OUTCOME and CANVAS trials demonstrated that SGLT2 inhibitors reduce cardiovascular mortality in patients with established atherosclerotic disease, establishing this drug class as the standard of care in high-risk type 2 diabetes. The LEADER and SUSTAIN-6 trials subsequently showed cardiovascular and renal benefits for GLP-1 receptor agonists liraglutide and semaglutide, culminating in the FLOW trial demonstrating that semaglutide reduces the risk of kidney disease progression by 24%. For type 1 diabetes, the Teplizumab MPC-031-004 trial produced the first-ever disease-modifying result: anti-CD3 immunotherapy delayed clinical diagnosis by a median of 2 years in at-risk individuals, leading to FDA approval of teplizumab (Tzield) for stage 2 type 1 diabetes prevention. The CREATE trial is currently evaluating stem-cell-derived islet transplants without immunosuppression using encapsulation technology β€” potentially transformative for type 1 patients.
Patient Guide: How to Find & Join a Trial
People with type 1 or type 2 diabetes can participate in trials at nearly any stage of disease, from prevention in at-risk individuals to management of advanced complications. Before searching for trials, know your key parameters: HbA1c (most recent, within 3 months), eGFR (kidney function), duration of diabetes, current medications, and any complications such as retinopathy or neuropathy — these determine eligibility for the majority of trials. Type 1 patients with recent diagnosis (within 1–2 years) may qualify for beta-cell preservation immunotherapy trials. Patients with type 2 diabetes and high cardiovascular risk or established kidney disease should specifically search for SGLT2 and GLP-1 combination trials in their treatment line. Technology-focused trials testing automated insulin delivery systems (closed-loop or hybrid closed-loop) typically recruit through endocrinology centers with dedicated diabetes technology programs.