MCF Cystic Kidney and Liver Disease Biobank

Overview

About this study

The purpose of this study is to

evaluate the potential of PAPP-A as a therapeutic target in ADPKD, we collaborated with Calico and performed the following studies under a sponsor research agreement (SRA) using an anti-PAPP-A monoclonal antibody (anti-PAPP-A) developed by Calico. These studies demonstrated an improvement in total kidney volume (TKV), reduction in cystic and tumor burden, and beneficial effects in survival, frailty index, and renal function.

Thus, our published studies and the preclinical studies performed under the Calico SRA confirm that the IGF/PAPP-A pathway is a therapeutic target in ADPKD, and that anti-PAPP-A treatment is beneficial in this mouse model of cystic disease. However, the specific cell types that express PAPP-A in ADPKD kidneys is unknown and their identification may provide valuable insights into the mechanism of action of the antibody.

We hypothesize that PAPP-A is highly induced in ADPKD kidneys and drives the cystic growth mainly through IGF dependent manner. We further propose that PAPP-A expression will correlate with the cystic disease in progressively growing ADPKD kidneys. The study will help to understand role of PAPP-A in cystic progression in ADPKD and to identify the cell type/pathway relevant to the IGF/PAPP-A axis and validate the human relevance of this pathway in ADPKD; which will further help us to clinically validate the relevance of PAPP-A/IGF pathway in the ADPKD patients.

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:

Patients undergoing surgery for polycystic kidney and liver disease or its complications or for a different renal or hepatic disease and wishing to participate in research studies of cystic liver and kidney disease.
Patients with polycystic kidney and/or liver disease, undergoing percutaneous cyst aspiration or aspiration with sclerotherapy and wishing to participate in research studies of cystic liver and kidney disease.
Patients without polycystic kidney or liver disease undergoing nephrectomies or partial hepatectomies for other pathologies.
Deceased patients with polycystic kidney and/or liver disease undergoing autopsy.

Exclusion Criteria:

Individuals who do not comprehend English (i.e., participants must be able to read and sign a consent form without the assistance of an interpreter).
Individuals who are unable to sign consent (e.g., mentally challenged, those declared legally incompetent).
Individuals regarded as belonging to a vulnerable population (e.g., prisoners).
Patients aged 18 years or older.
Individuals with HIV or hepatitis infection.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 4/20/23. 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
Jacksonville, Fla. Mayo Clinic principal investigator Eduardo Chini, M.D., Ph.D.	Open for enrollment	Contact information: Sonu Kashyap Ph.D. (904) 953-0798 Kashyap.Sonu@mayo.edu

Mayo Clinic Location

Status

Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Eduardo Chini, M.D., Ph.D.

Open for enrollment

Contact information:

Sonu Kashyap Ph.D.

(904) 953-0798

Kashyap.Sonu@mayo.edu

More information

Publications

Publications are currently not available

Clinical Trials