A Study to Evaluate the Role of NOX4 and Related Biomarkers in Autosomal Dominant Polycystic Kidney Disease

Overview

About this study

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a devastating genetic disorder that causes numerous cysts in the kidneys, and remains a leading cause of renal failure. However, the only FDA-approved therapy for ADPKD (tolvaptan) is limited to slowing-down disease progression, and has associated side effects. Furthermore, the mechanisms that contribute to cyst formation and further damage to the kidney are still uncertain. A better understanding of these processes may assist in development of new therapies with fewer adverse effects and improve the quality of life of these patients.

Another critical problem in the care of patients with ADPKD is that the rate of disease progression varies widely among individuals and markers of renal function don’t change until late stages, representing a major challenge for following these patients, identifying a treatment response or predicting the progression of the disease. Therefore, there is a pressing need for identifying early biomarkers of disease severity/progression and additional targets for therapeutic interventions.

Oxidative stress is the imbalance between the production of harmful free radicals and antioxidants and a major contributor of renal damage in other kidney diseases, but its role in ADPKD is unclear. This study will explore for the first time the role of oxidative stress damage in a preclinical model of ADPKD and related biomarkers at early stages of the disease. The findings resulting from this study are likely to have important clinical implications by: advancing the understanding of the mechanisms of renal damage in ADPKD, identifying novel early biomarkers, and highlighting additional processes that could be target for therapeutic intervention.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Autosomal Dominant Polycystic Kidney Disease (ADPKD) (based on Ravine et al. criteria).
  • Class 1 A-E according to imaging classification.
  • Male and female subjects, 15- 40 years of age.
  • Estimated GFR > 70 mL/min/1.73 m^2 (CKD-EPI).
  • Ability to provide written, informed consent.

Exclusion Criteria:

  • Class 2 according to imaging classification.
  • Concomitant systemic disease affecting the kidney.
  • Diabetes mellitus.
  • Predicted urine protein excretion in > 1 g/24 hrs.
  • Use of antioxidants; i.e., vitamins, Nrf2 activators.
  • Abnormal urinalysis.

Eligibility last updated 10/13/21. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Maria Irazabal Mira, M.D., Ph.D.

Closed for enrollment

Contact information:

Maria Irazabal Mira M.D., Ph.D.

(507) 293-3232

IrazabalMira.Maria@mayo.edu

More information

Publications

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CLS-20513355

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