Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)
Purpose
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.
Condition
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Eligibility
- Eligible Ages
- Between 18 Years and 65 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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)
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
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental VX-407 |
Participants will receive VX-407 for up to 52 weeks. |
|
Recruiting Locations
University of Alabama at Birmingham- Nephrology Research Clinic
Birmingham 4049979, Alabama 4829764 35233
Birmingham 4049979, Alabama 4829764 35233
More Details
- Status
- Recruiting
- Sponsor
- Vertex Pharmaceuticals Incorporated