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VX-433

Phase 1

Narcolepsy Type 1 (NT1) | Small molecule | Neurology |Vertex Pharmaceuticals Incorporated|Last Updated: Jun 8, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment118

FDA Designations

No designations recorded

Clinical trial landscape

VX-433 · 1 trial · 1 indication

Phase 1 1
NCT07584434A Phase 1, First-in-human Study of VX-433Narcolepsy Type 1 (NT1)
RECRUITING118 Analytics
PHASE1RECRUITING
A Phase 1, First-in-human Study of VX-433
Narcolepsy Type 1 (NT1)Unlock trial analytics

Study Endpoints

Primary Endpoints

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 6
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 18
Part C: Maximum Observed Plasma Concentration (Cmax) of Midazolam in the Absence and Presence of VX-433
From Day 1 up to Day 22
Part C: Maximum Observed Plasma Concentration (Cmax) of Bupropion and Hydroxybupropion in the Absence and Presence of VX-433
From Day 1 up to Day 22
Part C: Area Under the Concentration Versus Time Curve (AUC) of Midazolam in the Absence and Presence of VX-433
From Day 1 up to Day 22
Part C: Area Under the Concentration Versus Time Curve (AUC) of Bupropion and Hydroxybupropion in the Absence and Presence of VX-433
From Day 1 up to Day 22

Secondary Endpoints

Part A: Maximum Observed Plasma Concentration (Cmax) of VX-433
From Day 1 up to Day 6
Part B: Maximum Observed Plasma Concentration (Cmax) of VX-433
From Day 1 up to Day 18
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-433
From Day 1 up to Day 6
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part A: Single Ascending Dose (SAD)EXPERIMENTALParticipants will be randomized to receive a single dose of VX-433.
Part A: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-433.
Part B: Multiple Ascending Dose (MAD)EXPERIMENTALParticipants will be randomized to receive multiple doses of VX-433.
Part B: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-433.
Part C: Drug Drug InteractionEXPERIMENTALParticipants will receive a single dose of midazolam and bupropion on Day 1 in Dosing Period 1, followed by VX-433 administration for 17 days (Days 5 through 21) and then midazolam and bupropion on Day 18 in Dosing Period 2. Part C will be open-label.

Interventions

NameTypeDescription
VX-433DRUGSuspension for Oral Administration
PlaceboDRUGSuspension for Oral Administration
MidazolamDRUGSyrup for Oral Administration
BupropionDRUGCapsule for Oral Administration
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Eligibility Criteria

Age Range18 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: * Body mass index (BMI) of 18.0 to 30.0 kilogram per meter square (kg/m2), inclusive * A total body weight of more than (\>) 50 kg * Nonsmoker or ex-smoker for at least 3 months before screening with current nonsmoking status confirmed by urine or blood cotinine at screening...

Countries:United States
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Recent Changes (Last 90 Days)

LOWJun 8, 2026NCT07584434Status: NOT_YET_RECRUITING → RECRUITING
LOWJun 8, 2026NCT07584434Status: NOT_YET_RECRUITING → RECRUITING
LOWJun 8, 2026NCT07584434Status: NOT_YET_RECRUITING → RECRUITING

Frequently asked questions about VX-433

What is VX-433 used for in Narcolepsy Type 1?

VX-433 is an investigational small molecule being developed for the treatment of Narcolepsy Type 1 (NT1). It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The drug is being studied in a first-in-human trial to evaluate its safety and tolerability in healthy volunteers and participants with NT1.

What does VX-433 target?

VX-433 is a small molecule developed by Vertex Pharmaceuticals for Narcolepsy Type 1. Its specific molecular target has not been disclosed in available clinical trial information. The drug is being investigated for its potential to address the underlying pathophysiology of NT1, though the exact mechanism of action remains under study.

Who makes VX-433?

VX-433 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. Vertex is conducting a Phase 1 clinical trial of VX-433 for Narcolepsy Type 1 in the United States.

What phase is VX-433 in?

VX-433 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The ongoing Phase 1 study is a first-in-human trial designed to assess the safety, tolerability, and pharmacokinetics of VX-433 in healthy volunteers and individuals with Narcolepsy Type 1.

What clinical trials is VX-433 in?

VX-433 is being evaluated in a single Phase 1 clinical trial registered as NCT07584434. This is a first-in-human, randomized, double-blind, placebo-controlled study with an estimated enrollment of 118 participants. The trial is currently recruiting in the United States and includes healthy volunteers aged 18 years and older.

Is VX-433 the same as any other drug?

VX-433 is a distinct investigational compound developed by Vertex Pharmaceuticals. No alternative names for VX-433 have been reported in clinical trial registrations. It is being studied specifically for Narcolepsy Type 1 and is not known to be identical to any other marketed or investigational drug.