Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Conditions
Brief summary
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.
Interventions
DRUGVX-407
Tablets for oral administration.
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion 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) 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 within 12 months of screening Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| htTKV on MRI Over Time | Baseline up to End of Study (Week 52) |
Secondary
| Measure | Time frame |
|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 54 |
| Maximum Observed Plasma Concentration (Cmax) of VX-407 | From Day 1 up to Week 52 |
| Area Under the Concentration Versus Time Curve (AUC) of VX-407 | From Day 1 up to Week 52 |
Countries
Belgium, Canada, France, Germany, Spain, United Kingdom, United States
Contacts
CONTACTMedical Information
Outcome results
None listed