Clinical trial

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

Name

VX23-407-001

Description

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

Trial arms

Trial start

2024-04-01

Estimated PCD

2025-04-01

Trial end

2025-04-01

Status

Recruiting

Phase

Early phase I

Treatment

VX-407

Solution or Suspension for oral administration.

Arms:

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

Placebo

Solution or Suspension for oral administration.

Arms:

Part A: Placebo, Part B: Placebo

Midazolam

Syrup for oral administration.

Arms:

Part C (Optional): Drug-Drug Interaction

Size

Primary endpoint

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

Eligibility criteria

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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 95, 'type': 'ESTIMATED'}}

Updated at

2024-04-26

1 organization

3 products

1 indication

Organization

Vertex Pharmaceuticals

Product

Placebo

Indication

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Product

VX-407

Product

Midazolam