A Study to Determine the Prevalence and Clinical Characteristics Associated With PKD1 Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Overview
- Phase
- N/A
- Intervention
- Part B
- Conditions
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Enrollment
- 401
- Locations
- 45
- Primary Endpoint
- Part A: Percentage of PKD1 Gene Variant Groups in the ADPKD Population
- Status
- Active, Not Recruiting
- Last Updated
- 10 days ago
Overview
Brief Summary
The purpose of this study is to estimate the prevalence, demographic, and clinical characteristics of PKD1/2 gene variant groups in the ADPKD population.
Investigators
Eligibility Criteria
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
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.
Arms & Interventions
Part B
Participants who have completed all assessments in Part A and have a subset of variants in PKD1 will complete 1 year of follow-up.
Part A
Eligible participants will complete a single visit to complete a blood sample collection for PKD1/2 genotyping.
Outcomes
Primary Outcomes
Part A: Percentage of PKD1 Gene Variant Groups in the ADPKD Population
Time Frame: Genotyping Visit on Day 1 of Part A (Part A duration: 1 Calendar Day)
Part A: Weight of Participants Within Each PKD1 Gene Variant Group
Time Frame: Genotyping Visit on Day 1 of Part A (Part A duration: 1 Calendar Day)
Part A: Height of Participants Within Each PKD1 Gene Variant Group
Time Frame: Genotyping Visit on Day 1 of Part A (Part A duration: 1 Calendar Day)
Part A: Body Mass Index (BMI) of Participants Within Each PKD1 Gene Variant Group
Time Frame: Genotyping Visit on Day 1 of Part A (Part A duration: 1 Calendar Day)
Part A: Age of Participants at ADPKD Diagnosis Within Each PKD1 Gene Variant Group
Time Frame: Genotyping Visit on Day 1 of Part A (Part A duration: 1 Calendar Day)