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

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

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

Study Overview

• Status: Recruiting
• Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)
• Intervention / Treatment: Drug: Midazolam; Drug: VX-407; Drug: Placebo

Detailed 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.).

• Study Type: Interventional
• Enrollment (Estimated): 95
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Medical Information; Phone Number: 617-341-6777; Email: medicalinfo@vrtx.com

Study Locations

• United States
  • Kansas
    • Lenexa, Kansas, United States, 66219
      • Recruiting
      • ICON Lenexa

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part A: Single Ascending Dose (SAD); Participants will be randomized to receive a single dose of different dose levels of VX-407.	Drug: VX-407; Solution or Suspension for oral administration.
Placebo Comparator: Part A: Placebo; Participants will be randomized to receive placebo matched to VX-407.	Drug: Placebo; Solution or Suspension for oral administration.
Experimental: Part B: Multiple Ascending Dose (MAD); 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; Solution or Suspension for oral administration.
Placebo Comparator: Part B: Placebo; Participants will be randomized to receive multiple doses of placebo matched to VX-407.	Drug: Placebo; Solution or Suspension for oral administration.
Experimental: Part C (Optional): Drug-Drug Interaction; 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: Midazolam; Syrup for oral administration.; Drug: VX-407; Solution or Suspension for oral administration.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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
Part C: Maximum Observed Plasma Concentration (Cmax) of MDZ in Absence and Presence of VX-407	On Day 1, and Day 15
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 Outcome Measures

Outcome Measure	Time Frame
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: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	From Enrollment up to Day 24

Collaborators and Investigators

• Sponsor: Vertex Pharmaceuticals Incorporated

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2024
• Primary Completion (Estimated): April 1, 2025
• Study Completion (Estimated): April 1, 2025

Study Registration Dates

• First Submitted: March 28, 2024
• First Submitted That Met QC Criteria: March 28, 2024
• First Posted (Actual): April 3, 2024

Study Record Updates

• Last Update Posted (Actual): April 26, 2024
• Last Update Submitted That Met QC Criteria: April 25, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Abnormalities, Multiple; Kidney Diseases, Cystic; Ciliopathies; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Anesthetics, Intravenous; Anesthetics, General; Anesthetics; Tranquilizing Agents; Psychotropic Drugs; Hypnotics and Sedatives; Adjuvants, Anesthesia; Anti-Anxiety Agents; GABA Modulators; GABA Agents; Midazolam
• Other Study ID Numbers: VX23-407-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No