What is VX-407 studying for Autosomal Dominant Polycystic Kidney Disease (ADPKD)?

VX-407 is being studied to evaluate its effect on height-adjusted total kidney volume (htTKV) in people with ADPKD who have certain PKD1 gene variants. The study also looks at the safety, tolerability, and how the body processes the drug. This research is sponsored by Vertex Pharmaceuticals Incorporated to better understand this treatment option.

Who may be eligible for this Autosomal Dominant Polycystic Kidney Disease (ADPKD) clinical trial?

Adults aged 18 to 65 with a diagnosis of ADPKD and specific kidney imaging classifications may be eligible. Participants also need to have an estimated glomerular filtration rate (eGFR) of at least 25 mL/min/1.73 m² and be able to follow study visits. Please review eligibility criteria for more details.

Is there any cost to participate in this Autosomal Dominant Polycystic Kidney Disease (ADPKD) clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan for any travel costs themselves.

Actively Recruiting

Phase 2

Age: 18Years - 65Years

All Genders

ID07161037

A Phase 2a, Open-label Study Evaluating VX-407 for Autosomal Dominant Polycystic Kidney Disease With PKD1 Gene Variants

Led by Vertex Pharmaceuticals Incorporated · Updated on 2026-05-13

Participants Needed

Research Sites

23 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating VX-407 in adults aged 18 to 65 with Autosomal Dominant Polycystic Kidney Disease ADPKD who have certain PKD1 gene variants. The study aims to assess the effect of VX-407 on height-adjusted total kidney volume htTKV, as well as its safety, tolerability, and how the body processes the drug. This is an open-label, single-arm Phase 2a trial sponsored by Vertex Pharmaceuticals Incorporated. Participants will receive VX-407 tablets orally for up to 52 weeks. There is only one treatment group in this study, and all participants will receive this investigational drug. The study includes a treatment period lasting about one year, during which researchers will monitor the effects of VX-407 on kidney volume and collect data on the drugs behavior in the body. During the study, participants will have MRI scans to measure htTKV from the start through week 52. Safety and tolerability will be tracked by noting any adverse events up to week 54. Blood samples will be taken to evaluate the drugs plasma concentration and overall exposure over time. Participants are expected to comply with scheduled visits and study procedures throughout their involvement, which lasts a little over one year.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years - 65Years

All Genders

Eligibility Criteria

You may qualify if you...

A pre-existing diagnosis of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Age between 18 and 65 years
Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E confirmed by abdominal MRI
Estimated glomerular filtration rate (eGFR) ≥ 25 mL/min/1.73 m²
Willing and able to comply with scheduled visits and study procedures

You will not qualify if you...

History of kidney disease other than ADPKD that could affect its natural history
History of solid organ or bone marrow transplantation or nephrectomy
Currently on or planning to start renal replacement therapy within 12 months
Other protocol-defined exclusion criteria as per study guidelines

Research Team

Medical Information

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