This is an open label, two to three center study to evaluate the clinical efficacy and safety of 1 dose level of 2-hydroxypropyl-β-cyclodextrin (2-HPβCD) given intravenously in adult patients with type 2 diabetes with diabetic kidney disease (DKD) and proteinuria.
This is a Phase 2a, open label, two to three center study to evaluate the clinical efficacy and safety of 1 dose level of 2-hydroxypropyl-β-cyclodextrin (2-HPβCD) given intravenously in adult patients with type 2 diabetes with diabetic kidney disease (DKD) and proteinuria. The study will be conducted at 2 - 3 sites in the United States of America (USA) and will screen a sufficient number of subjects to complete 8 participants meeting the inclusion/exclusion criteria outlined. The study consists of up to a 4-week screening period, a 12-week treatment period and a 4-week follow-up period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
2HPβCD is a 7 D-glucopyranosyl derivation of cyclodextrin (CD) that entraps and passively removes intracellular cholesterol from the kidney. It is also believed to promote active cholesterol removal through up-regulation of cholesterol efflux transporters ABCA1 and ABCG1.
Clinical Advancement Center, PLLC
San Antonio, Texas, United States
Percent change in 24-hour urinary albumin to creatinine ratio (UACR)
Percentage change in urinary albumin to creatinine ratio (UACR) from baseline (day 1) to Week 12.
Time frame: From Day 1 (baseline) to Week 12 (end of treatment)
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Number of adverse events (AEs), serious adverse events (SAEs), discontinuations over the 12-week treatment period
Time frame: 12 Weeks
Absolute change in 24-hour urinary albumin to creatinine ratio (UACR)
Absolute change in 24-hour urinary protein to creatinine ratio (UPCR) from baseline (Day 1) to Week 12 (end of treatment)
Time frame: From Day 1 (baseline) to Week 12 (end of treatment)
Absolute change in 24-hour urinary protein to creatinine ratio (UPCR)
Absolute change in log transformed 24 hour urinary protein to creatinine ratio (UPCR) from Day 1 (baseline) to Week 12 (end of treatment)
Time frame: From Day 1 (baseline) to Week 12 (end of treatment)
Population trough pharmacokinetics of 2-hydroxypropyl-β-cyclodextrin (2-HPβCD)
Trough plasma concentration of 2-hydroxypropyl-β-cyclodextrin (2-HPβCD) on Day 1, Day 29, Day 57, and Day 85, compared with pre-dosing concentrations on Day 1 and Day 4
Time frame: Day 1, Day 29, Day 57, Day 85
Effects of 2-HPβCD on hearing based on changes in pure tone air conduction threshold from Day 1 to Week 16, and percent changes in tinnitus, and sense of fullness or stuffiness in the ears based on a Hearing Monitoring Questionnaire
Changes in pure tone air conduction threshold based on audiometry testing from Day 1 to Week 16. Percent changes in the presence of tinnitus (ringing in the ears), and a sense of fullness or stuffiness in the ears based on responses to a Hearing Monitoring Questionnaire from Day 1 to Week 16
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Time frame: From Day 1 to Week 16