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VX-264

Phase 1

Type 1 Diabetes | Small molecule | Metabolic |Vertex Pharmaceuticals Incorporated|Last Updated: Jun 4, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment7

FDA Designations

No designations recorded

Clinical trial landscape

VX-264 · 1 trial · 1 indication

Phase 1 1
NCT05791201A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 DiabetesType 1 Diabetes
ACTIVE NOT_RECRUITING7 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes
Type 1 DiabetesUnlock trial analytics

Study Endpoints

Primary Endpoints

Part A and 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 24 months
Part B: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)
From Baseline and at Day 90
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
VX-264EXPERIMENTAL -

Interventions

NameTypeDescription
VX-264DRUGAllogeneic human stem cell-derived islets.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites16

Key Inclusion Criteria: Clinical history of T1D with greater than or equal to (\>=) 5 years duration Participant is on a stable diabetic treatment Consistent use of continuous glucose monitoring (CGM) for at least 4 weeks before Screening and willingness to use CGM for the duration of the study Ke...

Countries:United StatesCanadaGermanyItalyNetherlandsSwitzerlandUnited Kingdom
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Frequently asked questions about VX-264

What is VX-264 used for in Type 1 Diabetes?

VX-264 is an investigational small molecule being developed for the treatment of Type 1 Diabetes. It is currently in Phase 1 clinical development, meaning it has not yet been approved by regulatory authorities. The drug is being studied to evaluate its safety, tolerability, and efficacy in participants with this condition.

Who makes VX-264?

VX-264 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company is conducting a Phase 1 clinical trial to assess the drug's safety, tolerability, and efficacy in participants with Type 1 Diabetes.

What phase is VX-264 in?

VX-264 is in Phase 1 clinical development. It is an investigational drug, meaning it is not yet approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is designed to evaluate the safety, tolerability, and efficacy of VX-264 in participants with Type 1 Diabetes.

What clinical trials is VX-264 in?

VX-264 is being studied in a Phase 1 clinical trial with the identifier NCT05791201. This trial is titled 'A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes' and is currently active but not recruiting. The study has an enrollment of 7 participants and is being conducted in multiple countries including the United States, Canada, Germany, Italy, Netherlands, Switzerland, and the United Kingdom.

Is VX-264 FDA approved?

No, VX-264 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for the treatment of Type 1 Diabetes. The drug is being studied in a clinical trial to evaluate its safety, tolerability, and efficacy, but it has not yet received regulatory approval for commercial use.