Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease

Vertex Pharmaceuticals

Status: Enrolling

Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Study type: Observational

Funder types: Industry

Identifiers

NCT06747572

VX24-KDO-901

Details and patient eligibility

About

The purpose of this study is to estimate the prevalence, demographic, and clinical characteristics of PKD1/2 gene variant groups in the ADPKD population.

Enrollment: 400 estimated patients

Sex: All

Ages: 12 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Estimated glomerular filtration rate (eGFR) greater than or equal to (≥) 30 milliliter per minute (mL/min)/1.73 m^2
• Willing and able to comply with scheduled visits and other study procedures
• A pre-existing diagnosis of ADPKD as defined in the protocol

Key Exclusion Criteria:

• History of kidney disease other than ADPKD that in the opinion of the investigator would independently impact the natural history of ADPKD
• History of solid organ or bone marrow transplantation or nephrectomy
• Ongoing renal replacement therapy or planning to start renal replacement therapy less than or equal to (≤)12 months from the Genotyping Visit in Part A

Other protocol defined Inclusion/Exclusion criteria will apply.

Trial design

400 participants in 2 patient groups

Part A

Description:

Eligible participants will complete a single visit to complete a blood sample collection for PKD1/2 genotyping.

Part B

Description:

Participants who have completed all assessments in Part A and have a subset of variants in PKD1 will complete 1 year of follow-up.