Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants
Not Applicable
Recruiting
- Conditions
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- Interventions
- Registration Number
- NCT07161037
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 24
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/Exclusion criteria will apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description VX-407 VX-407 Participants will receive VX-407 for up to 52 weeks.
- Primary Outcome Measures
Name Time Method htTKV on MRI Over Time Baseline up to End of Study (Week 52)
- Secondary Outcome Measures
Name Time Method 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
Trial Locations
- Locations (4)
Alabama Kidney Research
🇺🇸Alabaster, Alabama, United States
Nephrology & Hypertension Associates, PC
🇺🇸Middlebury, Connecticut, United States
Renal Associates
🇺🇸Columbus, Georgia, United States
DaVita Kidney Care - Las Vegas
🇺🇸Las Vegas, Nevada, United States
Alabama Kidney Research🇺🇸Alabaster, Alabama, United States