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

Autosomal Dominant Polycystic Kidney Disease (ADPKD) Clinical Trial

Official title:

A Phase 1, Randomized, Double-blind, Placebo-controlled, First-in-human Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Doses of VX-407 in Healthy Subjects

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06345755
• Other study ID #: VX23-407-001
• Status: Recruiting
• Phase: Phase 1
• Start date: April 17, 2024
• Est. completion date: April 2025

Study information

• Verified date: March 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: Medical Information
• Phone: 617-341-6777
• Email: medicalinfo[@]vrtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 95
• Est. completion date: April 2025
• Est. primary completion date: April 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

Key Inclusion Criteria:

• 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

Key Exclusion Criteria:

• 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

Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Autosomal Dominant Polycystic Kidney Disease (ADPKD)
• Kidney Diseases
• Polycystic Kidney Diseases
• Polycystic Kidney, Autosomal Dominant

Intervention

Drug:
• VX-407
Solution or Suspension for oral administration.
• Placebo
Solution or Suspension for oral administration.
• Midazolam
Syrup for oral administration.

Locations

Country	Name	City	State
United States	ICON Lenexa	Lenexa	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	Part C: Maximum Observed Plasma Concentration (Cmax) of MDZ in Absence and Presence of VX-407		On Day 1, and Day 15
Primary	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
Secondary	Part A: Maximum Observed Plasma Concentration (Cmax) of VX-407		From Day 1 up to Day 6
Secondary	Part B: Maximum Observed Plasma Concentration (Cmax) of VX-407		Days 1, 7, and 14
Secondary	Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-407		From Day 1 up to Day 6
Secondary	Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-407		Days 1, 7, and 14
Secondary	Part C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		From Enrollment up to Day 24