P2a Open Label Study to Evaluate 2-HPβCD in Subjects With Diabetic Kidney Disease

NCT06489340 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: VAR200-0301
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Diabetic Kidney Disease

Outcome Measures

Primary Outcomes (2)

• 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.

  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

  12 Weeks

Secondary Outcomes (4)

• Absolute change in 24-hour urinary albumin to creatinine ratio (UACR)

  From Day 1 (baseline) to Week 12 (end of treatment)

• Absolute change in 24-hour urinary protein to creatinine ratio (UPCR)

  From Day 1 (baseline) to Week 12 (end of treatment)

• Population trough pharmacokinetics of 2-hydroxypropyl-β-cyclodextrin (2-HPβCD)

  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

  From Day 1 to Week 16

Study Arms (1)

2HPβCD

EXPERIMENTAL

Study drug will be administered by IV infusion during the treatment period.

Drug: 2HPβCD

Interventions

2HPβCD DRUG

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.

Also known as: 2-hydroxypropyl-β-cyclodextrin

2HPβCD

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Capable of giving and has provided a signed Informed Consent Form (ICF).
• Male or female age 18 to 75 years inclusive, at the time of signing the informed consent.
• Women of childbearing potential (WOCBP) and male subjects who are partners of WOCBP must agree to use an acceptable form of contraception during the study and for 30 days following the last dose of study drug.
• Clinical diagnosis of type 2 diabetes as per guidelines.
• Clinical diagnosis of diabetic kidney disease in the opinion of the principal investigator, or renal biopsy proven diabetic kidney disease without evidence of additional pathologic findings of alternative diagnosis.
• At screening, based on two 24-hour urine collections, geometric mean of two urinary albumin creatine ratios (UACR) ≥ 400 mg/g and ≤ 3500 mg/g.
• At screening, eGFR equal or greater than 30 and less than 90 mL/min/1.73 m\^2.
• Body mass index (BMI) ≤ 40.0 kg/m\^2.
• If on diabetes and anti-hypertensive medications:
• Angiotensin converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) inhibitors dose must be stable for 3 months before screening.
• Sodium-glucose co-transporter 2 (SGLT2) or GLP-1 receptor agonist or long-acting insulin dose must be stable for at least 3 months prior to screening.
• All other diabetes and anti-hypertensive medications must be at a stable dose for at least 3 months prior to screening.
• Hemoglobin A1c (HbA1c) ≤10.0% at screening.
• Willing to comply with IV administration of the study drug for 12 weeks and all protocol procedures during the study.

You may not qualify if:

• Has a solitary kidney.
• Has a positive drug screen.
• Known kidney disease other than diabetic kidney disease.
• End stage renal disease (ESRD) (i.e., peritoneal dialysis, hemodialysis, or history of kidney transplantation).
• Acute kidney injury or dialysis within the last 3 months before the screening visit.
• Uncontrolled diabetes as defined by HbA1c \>10 at screening.
• Uncontrolled hypertension with systolic blood pressure (SBP) \>140 mmHg or diastolic blood pressure (DBP) \>90 mmHg during screening.
• Unstable cardiovascular disease or history of myocardial infarction or arterial thromboembolic events within 3 months prior to screening or severe or unstable angina, New York Heart Association (NYHA) Class III or IV disease, or a QTc interval \>480 msec.
• Patients on IV medication containing cyclodextrin.
• Patients on steroids, except for those on low-dose topical steroids (per PI discretion) or intranasal or inhaled steroids.
• Surgery within the past 3 months prior to the first study drug administration determined by the Investigator to be clinically relevant.
• Known malignancy that is progressing or has required active treatment within the past 3 years. Any exceptions must be approved by the Medical Monitor.
• a. Note: Participants with basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ (e.g., breast carcinoma, cervical cancer in situ) that have undergone potentially curative therapy are not excluded.
• Known history of Human Immunodeficiency Virus (HIV) infection (HIV 1/2 antibodies).
• Known active Hepatitis B (defined as Hepatitis B surface antigen \[HBsAg\] reactive) or known active Hepatitis C virus (defined as HCV RNA \[qualitative\] is detected) infection.

Sponsors & Collaborators

• ZyVersa Therapeutics, Inc.: lead

Study Sites (1)

Clinical Advancement Center, PLLC

San Antonio, Texas, 78212, United States

Location

MeSH Terms

Conditions

Diabetic Nephropathies

Condition Hierarchy (Ancestors)

Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Diabetes Complications; Diabetes Mellitus; Endocrine System Diseases

Study Officials

• Pablo Pergola, M.D PHD

  Clinical Advancement Center, PLLC

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 18, 2024
• First Posted: July 5, 2024
• Study Start: June 11, 2025
• Primary Completion (Estimated): August 11, 2026
• Study Completion (Estimated): August 11, 2026
• Last Updated: November 25, 2025

Record last verified: 2025-09

Locations

US (1)