A Phase 1 Dose Escalation Study of VX-973 in Healthy Adults

A Phase 1, Randomized, Double-blind, Placebo-controlled, Single- and Multiple-dose Escalation Study Evaluating the Safety, Pharmacokinetics, and Pharmacodynamics of VX-973 in Healthy Adults

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of VX-973 in healthy participants.

Study Overview

• Status: Completed
• Conditions: Pain
• Intervention / Treatment: Drug: Midazolam; Drug: Placebo; Drug: VX-973

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 (Actual): 30
• Phase: Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • Harrow, United Kingdom
    • Parexel International

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 more than (>) 50 kg
• Participants of non-childbearing potential
• 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

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part A: Single Ascending Dose; Participants will be randomized to receive a single dose of VX-973 under fasted conditions.	Drug: VX-973; Suspension for oral administration.
Placebo Comparator: Placebo Part A; Participants will be randomized to receive placebo matched to VX-973.	Drug: Placebo; Suspension for oral administration.
Experimental: Part B: Multiple Ascending Dose; Participants will be randomized to receive multiple doses of VX-973 under fasted conditions. The dose and frequency will be determined based on Part A. Midazolam is planned to be administered as single oral doses of 2 mg first alone on Day -1 and then co-administered with study drug on Day 27.	Drug: Midazolam; Syrup for oral administration.; Drug: VX-973; Suspension for oral administration.
Placebo Comparator: Placebo Part B; Participants will be randomized to receive placebo matched to VX-973. Midazolam is planned to be administered as single oral doses of 2 mg first alone on Day -1 and then co-administered with placebo matched to VX-973 on Day 27.	Drug: Midazolam; Syrup for oral administration.; Drug: Placebo; 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 Day 1 up to Day 31
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	From Day 1 up to Day 43

Secondary Outcome Measures

Outcome Measure	Time Frame
Part A: Maximum Observed Plasma Concentration (Cmax) of VX-973	From Day 1 up to Day 30
Part B: Maximum Observed Plasma Concentration (Cmax) of VX-973	From Day 1 up to Day 43
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-973	From Day 1 up to Day 30
Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-973	From Day 1 up to Day 43
Part B: Maximum Observed Plasma Concentration (Cmax) of Midazolam in the Absence and Presence of VX-973	Days -1 and 27: Pre-dose up to 24 hours Post Midazolam dose
Part B: Area Under the Concentration Versus Time Curve (AUC) of Midazolam in the Absence and Presence of VX-973	Days -1 and 27: Pre-dose up to 24 hours Post Midazolam dose

Collaborators and Investigators

• Sponsor: Vertex Pharmaceuticals Incorporated

Study record dates

Study Major Dates

• Study Start (Actual): May 10, 2023
• Primary Completion (Actual): April 4, 2024
• Study Completion (Actual): April 4, 2024

Study Registration Dates

• First Submitted: April 24, 2023
• First Submitted That Met QC Criteria: May 17, 2023
• First Posted (Actual): May 19, 2023

Study Record Updates

• Last Update Posted (Actual): May 6, 2024
• Last Update Submitted That Met QC Criteria: May 3, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: 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: VX22-973-001; 2022-003301-30 (EudraCT Number)

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
• product manufactured in and exported from the U.S.: Yes