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

NCT06345755 | Completed Phase 1 FDA Drug

• 1 other identifier: VX23-407-001
• Study Type: interventional
• Target: 159
• Locations: 2 countries
• Sites: 2

Brief Summary

The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters 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 (12)

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

  From Day 1 up to Day 6

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

  Days 1, 7, and 14

• Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-407

  From Day 1 up to Day 6

• Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-407

  Days 1, 7, and 14

• Part C: Area Under the Concentration Versus Time Curve (AUC) of MDZ in Absence and Presence of VX-407

  On Day 1, and Day 15

Study Arms (6)

Part A: Single Ascending Dose (SAD)

EXPERIMENTAL

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

Drug: VX-407

Part A: Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Part B: Multiple Ascending Dose (MAD)

EXPERIMENTAL

Participants will be randomized to receive multiple doses of different dose levels of VX-407. The dose levels will be determined based on the data from Part A.

Drug: VX-407

Part B: Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Part C: Drug-Drug Interaction

EXPERIMENTAL

Participants will be administered Midazolam (MDZ) in the presence or absence of VX-407. The dose levels will be determined based on the data from Part B.

Drug: VX-407; Drug: Midazolam

Part D

EXPERIMENTAL

Participants will be randomized to receive VX-407 in 1 of 3 treatment sequences with 3 dosing periods to assess the relative bioavailability of VX-407 formulations and the effect of food on the pharmacokinetics of VX-407.

Drug: VX-407

Interventions

VX-407 DRUG

Solution or Suspension for oral administration.

Part A: Single Ascending Dose (SAD); Part B: Multiple Ascending Dose (MAD); Part C: Drug-Drug Interaction

Placebo DRUG

Solution or Suspension for oral administration.

Part A: Placebo; Part B: Placebo

Midazolam DRUG

Syrup for oral administration.

Part C: Drug-Drug Interaction

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 (2)

ICON Lenexa

Lenexa, Kansas, 66219, United States

Location

Altasciences Montreal

Montreal, Canada

Location

Related Publications (1)

• Cortinovis M, Perico N, Remuzzi G. The Need for Novel Therapeutic Directions in Autosomal Dominant Polycystic Kidney Disease Patient Care. Clin J Am Soc Nephrol. 2025 Dec 5. doi: 10.2215/CJN.0000000975. Online ahead of print.

  PMID: 41348481 DERIVED

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Interventions

Midazolam

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

Intervention Hierarchy (Ancestors)

Benzodiazepines; Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

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: March 28, 2024
• First Posted: April 3, 2024
• Study Start: April 17, 2024
• Primary Completion: January 27, 2025
• Study Completion: June 13, 2025
• Last Updated: July 14, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); CA (1)