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VX-668

Phase 1

Alpha-1 Antitrypsin Deficiency | Small molecule | Rare Disease |Vertex Pharmaceuticals Incorporated|Last Updated: Mar 15, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment114

FDA Designations

No designations recorded

Clinical trial landscape

VX-668 · 1 trial · 1 indication

Phase 1 1
NCT05727800A Phase 1, First-in-human Study of VX-668Alpha-1 Antitrypsin Deficiency
COMPLETED114 Analytics
PHASE1COMPLETED
A Phase 1, First-in-human Study of VX-668
Alpha-1 Antitrypsin DeficiencyUnlock trial analytics

Study Endpoints

Primary Endpoints

Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Day 20

Secondary Endpoints

Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-668
Day 1 up to Day 20
Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-668
Day 1 up to Day 20
Part A and B: Urine Concentration of VX-668
Part A Cohort A3: Days 1 and 5; Part B Cohort B3: Days 1, 10, and 11
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part AEXPERIMENTALParticipants grouped in different cohorts will receive a single ascending dose of VX-668.
Placebo Part APLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-668.
Part BEXPERIMENTALParticipants grouped in different cohorts will receive multiple doses of VX-668.The dose levels will be determined based on the data from Part A.
Placebo Part BPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-668.

Interventions

NameTypeDescription
VX-668DRUGSuspension for oral administration.
PlaceboDRUGSuspension for oral administration.
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Eligibility Criteria

Age Range18 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites5

Key Inclusion Criteria: * Participants of age between 18 to 55 years (inclusive) * 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 * Nonsmoker or ex-smoker for at least 3 months before screening Key Exclusion Criteria: * Any c...

Countries:United States
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Frequently asked questions about VX-668

What is VX-668 used for?

VX-668 is an investigational small molecule being developed for the treatment of Alpha-1 Antitrypsin Deficiency, a rare genetic condition. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes VX-668?

VX-668 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company is conducting clinical research on this investigational drug for Alpha-1 Antitrypsin Deficiency.

What phase is VX-668 in?

VX-668 is in Phase 1 clinical development. A first-in-human study of the drug has been completed, and it remains an investigational agent that has not received regulatory approval for any use.

What clinical trials is VX-668 in?

VX-668 has one completed Phase 1 clinical trial registered as NCT05727800. This first-in-human study enrolled 114 participants in the United States, included healthy volunteers, and was randomized, double-blind, and placebo-controlled.

Is VX-668 FDA approved?

VX-668 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for Alpha-1 Antitrypsin Deficiency. The completed Phase 1 trial was a first-in-human study, and the drug has not yet progressed to later-stage trials or regulatory review.