A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants

NCT07022119 | Completed Phase 1 FDA Drug

• 1 other identifier: VX24-407-002
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of VX-407 in healthy participants.

Conditions

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Outcome Measures

Primary Outcomes (2)

• Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  From Enrollment up to Day 10

• Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  From Enrollment up to Day 23

Secondary Outcomes (8)

• Part A: Maximum Observed Concentration (Cmax) of VX-407 in Plasma

  From Day 1 up to Day 6

• Part B: Cmax of VX-407 in Plasma

  Days 1, 7, and 14 up to Day 19

• Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to Infinity (AUC0-inf) of VX-407 in Plasma

  From Day 1 up to Day 6

• Part B: Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-407 in Plasma

  Days 1, 7, and 14 up to Day 19

• Part A: Time Taken for VX-407 to Reach Maximum Concentration (tmax)

  From Day 1 up to Day 6

Study Arms (4)

Part A: Single Ascending Dose

EXPERIMENTAL

Participants will be randomized to receive a single dose of VX-407.

Drug: VX-407

Placebo Part A

PLACEBO COMPARATOR

Participants will be randomized to receive placebo matched to VX-407.

Drug: Placebo

Part B: Multiple Ascending Dose

EXPERIMENTAL

Participants will be randomized to receive multiple doses of VX-407.

Drug: VX-407

Placebo Part B

PLACEBO COMPARATOR

Participants will be randomized to receive placebo matched to VX-407.

Drug: Placebo

Interventions

VX-407 DRUG

Suspension for oral administration.

Part A: Single Ascending Dose; Part B: Multiple Ascending Dose

Placebo DRUG

Suspension for oral administration.

Placebo Part A; Placebo Part B

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m\^2)
• A total body weight of greater than (\>) 50 kg
• Nonsmoker or ex-smoker for at least 3 months before screening

You may not qualify if:

• History of febrile illness or other acute illness that has not fully resolved within 14 days before the first dose of study drug
• Any condition possibly affecting drug absorption

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (1)

ICON Lenexa

Lenexa, Kansas, 66219, United States

Location

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2025
• First Posted: June 15, 2025
• Study Start: June 12, 2025
• Primary Completion: February 24, 2026
• Study Completion: February 24, 2026
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)