RECRUITING

Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants

Conditions

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of the study is to evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV), safety, tolerability, and pharmacokinetics (PK) of VX-407.

Official Title

A Phase 2a, Open-label, Single-arm Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-407 in Subjects With Autosomal Dominant Polycystic Kidney Disease Who Have a Subset of PKD1 Gene Variants

Quick Facts

Study Start:2025-09-26

Study Completion:2027-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07161037

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* A pre-existing diagnosis of ADPKD as defined in the protocol * Willing and able to comply with scheduled visits and other study procedures * Participants with ADPKD with Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E confirmed by abdominal MRI obtained during screening * Estimated glomerular filtration rate (eGFR) greater than or equal to (≥) 25 milliliter per minute (mL/min)/1.73 square meter (m\^2)	* 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 within 12 months of screening

Inclusion Criteria

Exclusion Criteria

* A pre-existing diagnosis of ADPKD as defined in the protocol
* Willing and able to comply with scheduled visits and other study procedures
* Participants with ADPKD with Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E confirmed by abdominal MRI obtained during screening
* Estimated glomerular filtration rate (eGFR) greater than or equal to (≥) 25 milliliter per minute (mL/min)/1.73 square meter (m\^2)

* 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 within 12 months of screening

Contacts and Locations

Study Contact

Medical Information

CONTACT

617-341-6777

medicalinfo@vrtx.com

Study Locations (Sites)

Alabama Kidney Research

Alabaster, Alabama, 35007

United States

Nephrology & Hypertension Associates, PC

Middlebury, Connecticut, 06762

United States

Renal Associates

Columbus, Georgia, 31904

United States

DaVita Kidney Care - Las Vegas

Las Vegas, Nevada, 89107

United States

Collaborators and Investigators

Sponsor: Vertex Pharmaceuticals Incorporated

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-09-26

Study Completion Date2027-12-31

Study Record Updates

Study Start Date2025-09-26

Study Completion Date2027-12-31

Terms related to this study

Additional Relevant MeSH Terms

Autosomal Dominant Polycystic Kidney Disease (ADPKD)