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

Vertex Pharmaceuticals

Status and phase: Enrolling; Phase 2

Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Treatments: Drug: VX-407

Study type: Interventional

Funder types: Industry

Identifiers

NCT07161037

2024-517393-13-00 (Other Identifier)

VX24-407-101

Details and patient eligibility

About

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.

Enrollment: 24 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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/Exclusion criteria will apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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24 participants in 1 patient group

VX-407

Experimental group

Description:

Participants will receive VX-407 for up to 52 weeks.

Treatment:

Drug: VX-407