MedPath

Uric Acid Lowering Trial in Youth Onset T2D

Phase 2
Recruiting
Conditions
Diabetic Kidney Disease
Hyperuricemia
Diabetes Mellitus, Type 2
Type2 Diabetes
Diabetic Nephropathies
Diabetes
Type 2 Diabetes Mellitus
Diabetes Complications
Interventions
Registration Number
NCT03899883
Lead Sponsor
University of Colorado, Denver
Brief Summary

Adolescents and young adults with youth-onset type 2 diabetes (T2D) are disproportionally impacted by hyperuricemia compared to non-diabetic peers and youth with type 1 diabetes (T1D). In fact, 50% of males with youth-onset T2D have serum uric acid (SUA) greater than 6.8 mg/dl. The investigators also recently demonstrated that higher SUA conferred greater odds of developing hypertension and diabetic kidney disease (DKD) in youth with T2D over 7 years follow-up. Elevated SUA is thought to lead to cardiovascular disease (CVD) and DKD by inflammation, mitochondrial dysfunction and deleterious effects on nephron mass. While there are studies demonstrating beneficial effects of uric acid (UA) lowering on vascular health in the general population, there are no studies in youth-onset T2D. Youth-onset T2D carries a greater risk of DKD and CVD compared to adult-onset T2D and T1D.

Accordingly, a clinical trial evaluating UA lowering therapies is needed in youth-onset T2D. Krystexxa (pegloticase), a uricase, effectively lowers SUA and therefore holds promise as a novel therapy to impede the development of CVD and DKD in youth-onset T2D. This proposal describes a pilot and feasibility trial evaluating the effect of UA lowering by pegloticase on markers of CVD and DKD in ten (n=10) youth aged 18-25 with youth-onset T2D (diagnosed \<21 years of age) over 7 days. The overarching hypothesis is that pegloticase improves marker of cardiorenal health by lowering UA.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
Male
Target Recruitment
10
Inclusion Criteria
  • Men
  • Ages 18-25
  • Youth-onset T2D (diagnosis <21 years)
  • serum uric acid ≥ 5 mg/dl
Exclusion Criteria
  • Glucose-6-phosphate (G6P) deficiency
  • Allergies to seafood or iodine
  • MRI contraindications (severe claustrophobia, non-MRI compatible implantable devices, weight ≥ 450 lbs)
  • HbA1C ≥ 12%
  • Recent (1 month prior) diagnosis of diabetic ketoacidosis (DKA) or hyperosmolar hyperglycemia
  • Congestive heart failure
  • History of multiple and/or severe allergies or anaphylactic reactions
  • Uric acid lowering medications (ie: allopurinol, febuxostat)
  • Pegvisomant, pegvaliase, peginterferon alfa 2b, peginterferon alfa 2a, pegfilgrastim, pegaspargase, pegaptanib, pegademase and certolizumab pegol
  • Participation in another investigational study within 2 weeks prior to study

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
PegloticasePegloticase 8 MG/ML [Krystexxa]Single administration of pegloticase (8 mg IV in 250 mL 0.9% normal saline)
Primary Outcome Measures
NameTimeMethod
Cardiovascular Markers5 min

Measured by Systolic Blood Pressure (SBP), Diastolic Blood Pressure (DBP), Mean Arterial Pressure (MAP)

Pulse Wave Velocity (PWV)2 hours (x2 study visits)

Measured by Aortic MRI renal MRI (4D Flow)

Wall Shear Stress (WSS)2 hours (x2 study visits)

Measured by Aortic MRI renal MRI (4D Flow)

Renal Blood Flow1 hour (x2 study visits)

Measured by 4D Flow renal MRI

Glomerular Filtration Rate4 hours (x2 study visits)

Measured by Iohexol Clearance in Plasma

Albumin Excretion Rate (AER)4 hours (x2 study visits)

Measured by albumin and creatinine concentrations in urine

Secondary Outcome Measures
NameTimeMethod
Calculated parameters of intrarenal hemodynamic function1 hour

Measured by using existing renal hemodynamic calculations

Change in serum uric acid (sUA)1 hour

Measured by baseline sUA compared to sUA one week later

Trial Locations

Locations (1)

Children's Hospital Colorado

🇺🇸

Aurora, Colorado, United States

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